our journey towards curing childhood cancer...relationships more complex and on a greater scale than...

Our journey towards curing childhood cancer

ANNUAL REVIEW 2016 HIGHLIGHTS

OUR VISION & PURPOSE 2

EXECUTIVE DIRECTOR’S MESSAGE 4

THE BUILDING BLOCKS OF OUR SUCCESS 6

CHAIRMAN’S MESSAGE 7

OUR RESEARCH 8

CELEBRATING OUR EMERGING RESEARCHERS 10

CHANGING THE FUTURE FOR CHILDREN WITH BRAIN CANCER 12

ZERO CHILDHOOD CANCER 14

RESEARCH HIGHLIGHTS 18

FUNDRAISING HIGHLIGHTS 20

ISLA’S STORY 22

FINANCIAL HIGHLIGHTS 24

OUR GLOBAL COLLABORATIONS 26

TABLE OF CONTENTS

OUR VISION& PURPOSE


Our vision

To save the lives of all children with cancer and eliminate their suffering.

Our purpose

Every year more than 950 Australian children and adolescents will be diagnosed with cancer. Every week nearly three Australian children and adolescents will die of cancer. Our organisation exists solely to put an end to childhood cancer.

We don’t just hope to do it.

We will do it.

It’s not if. It’s when.

2016 has been an extraordinarily successful year for the Institute, a year in which we celebrated the 40th anniversary of the Institute’s foundation and reflected on the progress we have made towards one day curing every child with cancer.

This year, we delivered on a number of key strategic goals and set in train multiple new initiatives. We are now reaping the benefits of the platforms, programs and partnerships established over the last few years, and this year set new records for both research grants and fundraising. All this puts us in a better position than ever to translate our research into improving outcomes for children just like Brydee (pictured).

Heading towards zero childhood cancer

Our primary focus over the past 12 months has been implementing Zero Childhood

Cancer, the national child cancer personalised medicine program I announced in my report last year. In partnership with Sydney Children’s Hospital, Randwick, we are close to completing the pilot phase of a clinical trial.

The pilot study has allowed us to put in place the

infrastructure and procedures to receive, process and analyse samples of tumours of dozens of children with high-risk cancers in preparation for next year’s national clinical trial. It is exciting to be working with every children’s hospital in the country and with leading research institutes in Australia and internationally.

EXECUTIVE DIRECTOR’S

MESSAGE

FROM OUR EXECUTIVE DIRECTORPROFESSOR MICHELLE HABER AM

In May, we were thrilled to have the Prime Minister, The Hon Malcolm Turnbull MP, and the Federal Health Minister, The Hon Sussan Ley MP, visit the Institute to pledge $20 million from the Federal Government to roll out the Zero Childhood Cancer program nationally. $5.9 million of this funding will come directly to the Institute to help support our capital equipment and essential research infrastructure costs. The balance of this significant contribution will help fund the infrastructure required to deliver the program nationally in partnerships with hospitals and research institutes around Australia. This pledge was followed by a visit from His Excellency General the Honourable Sir Peter Cosgrove AK MC (Retd) in September, to open the Australian Cancer Research Foundation (ACRF) Child Cancer Personalised Medicine Centre, the program’s operational headquarters.

An additional $4 million committed by the Australian Lions Childhood Cancer Research Foundation and Lions Club International Foundation will allow us to carry out whole genome sequencing of 400 children participating in the program, in collaboration with the Garvan Institute of Medical Research.

Our progress over the past twelve months is unprecedented in the Institute’s history, involving research programs, partnerships and clinical relationships more complex and on a greater scale than we have ever undertaken before.

Collaborations continue to flourish

In December, NSW Health Minister Jillian Skinner MP officially launched Paediatrio, a joint venture between our Institute, the Sydney Children’s Hospitals Network and Children’s Medical Research Institute, that aims to make NSW a world leader in paediatric research. Personalised cancer care is a priority focus area for Paediatrio, and its inaugural Chair, Kathryn Greiner AO is a passionate and committed advocate of our work.

Through this venture and others, we are working more closely than ever with our clinical partners at the Kids Cancer Centre (KCC) at Sydney Children’s Hospital, Randwick, now under the leadership of Associate Professor Tracey O’Brien. In parallel, we continue to enjoy a very productive relationship with the UNSW Australia (University of New South Wales, Australia), increasingly through our joint initiative, the UNSW Centre for Childhood Cancer Research.

It is exciting to be working with every children’s hospital in the country and with leading research institutes in Australia and internationally.”

Our collaborations with research teams all over Australia, and indeed all over the world, are flourishing, and our standing in the international research community continues to grow. A highlight of the year came in June, when I was honoured to convene the 2016 Advances in Neuroblastoma Research symposium, one of the largest child cancer research conferences in the world.

Building research capacity

With the help of capital funding awarded by the NSW State Government, we redeveloped our laboratory and office spaces to appropriately staff the Zero Childhood Cancer program and allow for expansion. Further funding from the Federal Government will allow us to redevelop other laboratory areas in 2017, creating even greater research capacity. To address our growing technology needs, we also restructured both our Technology and Project Management teams.

Our research capacity was further strengthened this year by our grant

success with a record $15 million in new funding. Professor Maria Kavallaris, who received accelerated promotion to National Health and Medical Research Council (NHMRC) Principal Research Fellow, began her prestigious Program Grant this year with Professor Richard Lock in precision nanomedicine. Also gratifying was our 40% success rate in the NHMRC Project Grant scheme (compared with a 15% sector average) and the renewal of a second Program Grant in neuroblastoma research.

Adding to the achievements of our senior researchers, our early career scientists have also done the Institute proud, attracting significant competitive research grants from NHMRC and other funding agencies. In addition, two of our emerging leaders, Drs David Ziegler and Tao Liu, were this year promoted through UNSW to Associate Professor.

Success = our people + your support

While our researchers continue to deliver at the highest level, our Executive has continued to operate as a close-knit and effective team, and our Board remains extremely supportive. Add to this the outstanding work of our support staff, and it is easy to see that the Institute’s success is all about its people.

Of course, none of us could continue our work without the Institute’s many supporters. To cover the true costs of medical research, we need to match every dollar of grant funding with funding from the community.

Our successes so far, and our future successes, rest on this support. I do hope you will stay with us on this most exciting of journeys, towards a future we all want to see: one that is free from childhood cancer.

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Brydee, aged 2 is currently in treatment for leukaemia4 5

This year was extremely successful for Children’s Cancer Institute and one, I believe, that represents a watershed in terms of where we can now move to.”David Smith, Chairman

Children’s cancer translational research is moving forwards in leaps and bounds, positively impacting treatments for kids with cancer, and we are set to play a bigger part than ever. This year was extremely successful for Children’s Cancer Institute and one, I believe, that represents a watershed in terms of where we can now move to.

It’s an exciting time. The Institute is redefining its organisational strategy for the next five years. Our achievements and growth make us a very different organisation to the one we were in 2011. We have come a long way.

Core to our continued success are our many partnerships and research collaborations. A great example of this is our relationship with the State and Federal governments, and all the collaborators linked to the Zero Childhood Cancer Program nationally. A key example is our membership in the Cooperative Research Centre (CRC) for Cancer Therapeutics (CTx). CTx represents the pre-eminent drug development

pipeline in Australia. We are well advanced in working with CTx to develop a small molecule, a potential future clinical agent, that sensitises cancer cells to chemotherapy.

Our financial results were very strong this year. They have established a solid footing for us to progress with the agenda we have set, due in no small part to the efforts of the Institute’s Fundraising team. This year the team again achieved extraordinary things, eclipsing their 2015 efforts. Over the past four years, our fundraising income has doubled to an extraordinary $16.5 million. A big contributor to this success is the effort of our community fundraisers, many of whom have had a personal experience of childhood cancer, and of course our individual donors. Much of this activity is only possible because of the many volunteers who donate their time, energy and skills to help us deliver these results. It is only through this generous support that we can fully leverage funds received from government and infrastructure funding.

In terms of our people, the Institute’s annual staff survey again showed an extraordinary level of staff engagement, and demonstrates that our staff truly believe in our mission to cure every child of cancer. This is a credit to the inspiring leadership of Professor Michelle Haber and the management team.

I would like to acknowledge the continued excellent results of our researchers and the talent of the Institute’s staff as a whole. We should all be incredibly proud of what they have achieved. The Board is firmly focussed on supporting the Institute in its next phase of growth and development with our new strategic plan a significant focus.

It is the quality of our people and our partners that will see the outcomes we are all striving for delivered on, and our vision to save the lives of all children with cancer fulfilled.


THE BUILDING BLOCKS OF OUR SUCCESS

FROM OUR CHAIRMAN

50%

“

Brilliance

The brightest minds, using cutting-edge technology in a world-class environment

Conviction

The knowledge that what we do will solve the problem

Hunger

The dogged determination to make it happen

Translation

Driving our discoveries into improved treatments for kids with cancer

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OUR RESEARCH

Children’s Cancer Institute is the only Medical Research Institute in Australia wholly dedicated to childhood cancer. To be able to find a cure for childhood cancer, we need to first understand it. That’s why our research starts with finding the causes and unravelling the nature of the different types of cancer.

The next step in our process is to test whether our findings will have the potential to become actual treatments for children with cancer. Utilising sophisticated robotic technology and pre-clinical model systems, our state-of-the-art labs provide the infrastructure needed to help us find new treatments as quickly as possible.

Our focus is on translational research, and we have an integrated team of clinicians and scientists who make sure our research has relevance and is used collaboratively throughout Australia and internationally so we can move closer to our goal of one day curing every child.

We are leading the development of the Zero Childhood Cancer national child cancer personalised medicine program for children with the most aggressive cancers, in partnership with the Sydney Children’s Hospitals Network. It will revolutionise the way treatment decisions are made with the aim of improving survivorship for those children at highest risk from their disease.

Testing our results1

Going into the clinic3 The future of childhood

cancer research4

How we go from the lab bench to a child’s bedsideOur focus is on translational research, making sure our discoveries are progressed into actual treatments for kids with cancer as quickly as possible. We seek to discover new treatments specifically designed for children, to develop safer and less toxic drugs and treatment protocols that will minimise side-effects and ultimately give children with cancer the best chance of a cure with the highest possible quality of life.

Starting with the basics 2


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CELEBRATING OUR EMERGING RESEARCHERSAt Children’s Cancer Institute, we’re committed to fostering the next generation of childhood cancer research leaders who will help us get closer to one day curing every child. Through providing state-of-the-art laboratory facilities, a supportive research environment and established research leaders as mentors, we are committed to building the pipeline of our emerging research talent and ensuring the continued research excellence of our Institute.

Amelia ParkerPhD StudentIn 2016, former summer-student Amelia Parker completed her PhD in our Tumour Biology and Targeting Program, led by Professor Maria Kavallaris. Her research into cell skeletons (cytoskeletons) and their complex roles in cancer focussed on a protein called βIII tubulin found in high levels in cancer cells.

Now officially Dr Parker, Amelia said she wouldn’t have believed how much she’d learn in four years at Children’s Cancer Institute. Highlights included publishing journal papers, doing science outreach, and presenting her research alongside

the world’s best PhD students to Nobel laureates. Amelia has enjoyed working in an environment where everyone is committed to delivering world-class translational research, and received lots of support from research mentors, support staff and fellow PhD students.

“Going into a PhD, you’re looking to learn a lot - something I did over and above what I was expecting. It’s very rewarding.”

That’s why you’re doing the job - to improve the lives of kids with cancer.”

“As a student, you have to learn how to discuss and defend your ideas to people who’ve been doing research for decades. It can be daunting starting out but, with their help, you learn skills and gain confidence. It’s a nice feeling to think you can stand on your own two feet.”

Like most science PhD graduates, Amelia’s excited about heading overseas for a postdoctoral fellowship, an almost-essential step in what is a global career.

Where does Amelia see herself in 20 years?

“Hopefully out of a job because childhood cancer will be cured. That’s why you’re doing the job - to improve the lives of kids with cancer.”

“Dr Han ShenPostdoctoral ResearcherHan Shen joined our Targeted Therapy group eager to make a difference for children with brain cancer, particularly Diffuse Intrinsic Pontine Glioma (DIPG). Originally from Beijing and a medical doctor, Han’s clinical work showed him the desperate need for treatments for children with this terrible cancer.

DIPG is the most aggressive and deadly childhood cancer. Deemed ‘terminal on diagnosis’, it has no cure, no effective treatment and no hope of survival. In 30 years, almost nothing has changed for DIPG patients in terms of treatment or outcome. That’s why our research is so important.

“This brain cancer has no current cure. Radiation is the only standard treatment but my goal is to change that,” Han said.

This brain cancer has no current cure. Radiation is the only standard treatment but my goal is to change that.”Han was thrilled to receive two major grants this year – from brain cancer charity The Cure Starts Now and the National Health and Medical Research Council (NHMRC). Han found

that an approved drug for children called dichloroacetate (DCA) helps radiation kill DIPG cells in animal models. Next he’ll work with senior researchers including group leader, Associate Professor David Ziegler, to translate these early findings into clinical treatments.

“We want to test this drug in combination with other drugs and radiation. The results so far are very promising.”

“The project aims to ultimately change the standard therapy for DIPG. DCA has been used safely for other diseases in children. That could shorten the time to translate it into the clinic and bring us closer to a cure,” he said.

“

Dr Dan CarterProject LeaderWhen Dan Carter joined the Institute fresh from a PhD in 2011, he sheepishly says his track record was nothing special. Despite this, Professor Glenn Marshall and Dr Belamy Cheung saw his potential to contribute to vital research toward finding a cure for childhood cancer. They nurtured his progress, something he is very grateful for.

That confidence was well-founded. This year brought Dan project grants (from NHMRC and from Cancer Australia, the latter co-funded by The Kid’s Cancer Project) and a Cancer Institute NSW fellowship that build on his successful research.

“While grant funding brings huge potential to grow my career, the bigger point is I’m able to do my best to discover something new to contribute toward a cure for childhood cancer,” he said.

I’m able to discover something new to contribute towards a cure for childhood cancer.”Dan’s research focusses on neuroblastoma, which claims the lives of more children under 5 than any other cancer. He uses an innovative technique called single-cell sequencing to study individual neuroblastoma cells to explore their genetic diversity, identify what makes them cancerous and see how they resist treatment.

“The biggest problem in all cancers is that patients don’t respond or only respond temporarily to cancer drugs. If we knew why, we could come up with more effective, better tolerated therapies.”

Dan says the Institute is a great place for young researchers passionate about making discoveries that can eventually be translated into the clinic for kids like Violet.

“It’s an environment that inspires all of us to do the best work we can. Everyone in the Institute has that motivation to help children with cancer,” he said.

“


Violet, aged 8, has recently completed 2

years of treatment for neuroblastoma. 10 11

Han, Amelia and Dan (l-r) in the Children’s Cancer Institute labs.

Brain tumours are among the deadliest childhood cancers and Diffuse Intrinsic Pontine Glioma (DIPG) is one of the most aggressive. Due to its sensitive location, surgery and even a biopsy is not possible. Patients do not respond to chemotherapy. Radiation, the standard therapy, only briefly delays tumour progression. Effective treatments are desperately needed so in the future families like Amy’s do not have to experience the pain of having a child taken by DIPG.This year, our Targeted Therapy Group made significant research progress and was awarded several grants.

Australia’s first DIPG cell cultures

Clinician-researcher Associate Professor David Ziegler is Senior Staff Specialist and Head of Clinical Trials, Kids Cancer Centre at Sydney Children’s Hospital, Randwick and Targeted Therapy Group Leader at Children’s Cancer Institute. He leads research that focusses on developing innovative therapies to rapidly translate to the clinic. A key area is investigating new treatment strategies for DIPG and other malignant childhood brain tumours. We are the Australian hub of the international DIPG registry, a global network of researchers and clinicians.

The search for new DIPG treatments is hampered by the lack of available cancer tissue so we initiated a national DIPG tumour donation protocol. Through this, we have collected tumour samples in the rare cases a biopsy is performed, and enabled parents to donate their child’s tumour for research after a child has died. Children’s

Cancer Institute has the only laboratory program in Australia dedicated to DIPG research and finding new treatments. The program owes its existence to the incredible generosity of parents wanting to help other families.

We are the Australian hub of the international DIPG registry, a global network of researchers and clinicians.”To date, 18 tumour samples have been used to grow a panel of DIPG neurosphere cultures - balls of cancer cells which help us investigate DIPG’s biology and test potential treatments.

Potential new treatments

In 2015, we found a drug called CBL137 was very effective in our laboratory research against neuroblastoma and, more recently, that it holds promise for DIPG. A clinical trial will open in 2018 at the largest US paediatric oncology institutions and at Sydney Children’s Hospital, Randwick to test CBL137 as a single agent in DIPG and neuroblastoma patients. Further research will show if we can predict which children with DIPG will respond best to CBL137 and whether it works better in combination with other drugs.

In 2016, we received a grant to explore another potential DIPG treatment, fenretinide. Fenretinide is among the few safest, most effective of 3500 drugs tested against our DIPG neurospheres, the largest drug screen ever performed for DIPG. We will gather data needed to

progress fenretinide, potentially in combination with other drugs, into clinical trials. This grant was awarded through the Priority-driven Collaborative Cancer Research Scheme and co-funded by Cancer Australia, Cancer Council NSW and The Kids’ Cancer Project.

A new project funded by the National Health and Medical Research Council (NHMRC) will explore whether we can kill DIPG cells more effectively with radiation by targeting glucose metabolism. Glucose gives tumours the energy to grow. We will see if cutting off DIPG cells’ energy supply, by treating them with dichloroacetate and other selected drugs, makes them more radiation-sensitive.

Personalised medicine for childhood brain cancer

Our DIPG research also focusses on developing individualised treatment for all children with high-risk brain tumours as part of the Zero Childhood Cancer national child cancer personalised medicine program. During 2016, in the program’s pilot stage, we enrolled 15 brain tumour patients, around half of all patients in this pilot study. It is children with this terminal cancer, which is high-risk at diagnosis and for which no successful treatments exist, who arguably stand to benefit most from personalised medicine.

We can only continue our research thanks to all our supporters - supporters just like Amy’s family who continue to work tirelessly to raise funds for our research, so one day we can find a cure for every child.

CHANGING THE FUTURE FOR CHILDREN WITH BRAIN CANCER “

I believe we can do for brain cancer what has already been done for leukaemia. Once, the survival rate for leukaemia was zero; today it’s 85%.”

“Associate Professor David Ziegler

Senior Staff Specialist & Head of Clinical Trials, Kids Cancer Centre at Sydney Children’s Hospital, Randwick

Targeted Therapies Group Leader - Children’s Cancer Institute


At age 9, Amy was diagnosed with a brain tumour called Diffuse Intrinsic Pontine Glioma (DIPG). Tragically, Amy passed away just 16 months after her diagnosis on 31st March 2009.

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The Zero Childhood Cancer national child cancer personalised medicine program is the most ambitious childhood cancer research initiative ever undertaken for children with cancer in Australia. It offers fresh hope to children with aggressive cancers who are at the highest risk of treatment failure. Zero Childhood Cancer stands to revolutionise the way childhood cancer is treated.

The challenge in curing all children of cancer is that every child and every cancer is unique. Zero Childhood Cancer aims to develop a genomic profiling and drug testing platform that can comprehensively analyse cancer cells from individual children with the most aggressive cancers, leading to recommendations of personalised cancer therapy with the potential to improve outcomes and reduce treatment-related toxicities. The aim is that the initial recommendation

for tailored treatment will be completed within 8 weeks from the receipt of patient samples, meaning we can personalise treatment to improve survivorship and reduce side effects in real time.

Led by Children’s Cancer Institute in partnership with Sydney Children’s Hospitals Network, Zero Childhood Cancer is truly national. Program collaborators include leading research institutes throughout Australia, USA and Europe, and all paediatric oncology centres in Australia, forming a seamless translational pipeline to bring research closer to patients than ever.

Vital support

In 2016, the Prime Minister and Health Minister committed $20 million from the Federal Government to roll out the Zero Childhood Cancer program nationally. $5.9 million of this funding will come directly to the Institute to help support our capital equipment and essential research infrastructure costs. The balance of this significant contribution will help fund

the infrastructure required to deliver the program nationally in partnerships with hospitals and research institutes around Australia.

It will support an entirely new level of profiling, analysis and therapeutic targeting of individual children’s disease, including immunological, protein, genomic and biological analyses. This is vital to deliver the benefits of Zero Childhood Cancer to all children throughout the country with the highest-risk cancers.

Pilot study

The Zero Childhood Cancer program pilot study, run in partnership with clinicians from the Kids Cancer Centre, Sydney Children’s Hospital, Randwick, commenced in July 2015.

Initially 12 children were expected to participate. However, more than 35 children have been enrolled due to strong demand for a precision medicine approach from clinicians and families.

We’ve established the necessary infrastructure and procedures

An unprecedented collaboration:

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Nixon, now aged 2, was born with cancer and began chemotherapy

at just 2 weeks old.


to receive, process and analyse samples of patients’ tumours, Genomic and laboratory testing against drugs a child’s cancer is most likely to respond to.

On track

Zero Childhood Cancer is ambitious and complex, and we are particularly pleased to report that we are on track with milestones.

This year, following full operational, organisational structure, and strategic reviews, we established governance and team structures to ensure the successful delivery of

the program. Steering and Research Management Committees have been established to guide the implementation of Zero Childhood Cancer, overseeing research management, program direction, organisational structure and reporting; and we are implementing a newly formed preclinical drug testing core team to ensure efficient development and delivery of laboratory drug screening.

We’ve worked closely with our partners to develop and submit the national study protocol for ethics approval; and we continued to seek

new funding sources to support the ongoing operational requirements to deliver the program.

In 2017 we aim to open the Zero Childhood Cancer national clinical trial involving children’s hospitals across Australia. The trial will run until 2020, with up to 400 children participating. By the trial’s end, our goal is to have shown that personalised medicine improves treatment and survivorship for children with the highest risk of treatment failure, a big step in our vision of one day curing 100% of children with cancer.

Participating children’s hospitals

Participating medical research institutes

Zero Childhood Cancer is a joint iniative of Children’s Cancer Institute, Kids Cancer Centre at Sydney Children’s Hospital Randwick and Sydney Children’s Hospital Network.

Zero Childhood Cancer Collaboration Map

Germany

USA

Zero Childhood Cancer is an innovative and translational program that offers the very real opportunity to research and implement a revolutionary new approach for childhood cancer treatment. But it is complex and expensive.

To enable the national rollout of Zero Childhood Cancer, we need at least $58 million of funding to ensure the required equipment, facilities and people are in place. These funds must be raised on top of current research requirements. To date, Children’s Cancer Institute has successfully secured commitments of $42 million from government bodies and funding partners.

The Zero Childhood Cancer fundraising campaign is a joint initiative of Children’s Cancer Institute and Sydney Children’s Hospital Foundation. It is a national, landmark campaign that has harnessed unprecedented collaboration between two of the nation’s leading children’s charities.

The campaign aims to raise $12 million, by engaging the generosity of community-spirited corporates and compassionate philanthropists Australia-wide, and we were delighted to welcome Mark Lazberger, Chief Executive Officer, Colonial First State Global Asset Management as our campaign chair and Dame Marie Bashir AD CVO, the previous Governor of NSW as patron of this fundraising campaign.

It is a national, landmark campaign that has harnessed unprecedented collaboration between two of the nation’s leading children’s charities.”2017 marks the official kick-off of this dedicated Zero Childhood Cancer fundraising campaign and we look forward to sharing our progress as the campaign evolves. More information can be found at:

zerochildhoodcancer.org.au

Zero Childhood Cancer Capacity CampaignThe Zero Childhood Cancer program will involve all paediatric oncology units across Australia who treat the 950 children and adolescents who are diagnosed with cancer every year. These hospitals will work collaboratively with a range of key Medical Research Institutes both nationally and internationally who are part of Zero Childhood Cancer.

Funding Partners:

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RESEARCH HIGHLIGHTS


Drug Discovery Centre The ACRF Drug Discovery Centre is a unique facility that enables high throughput screening of potential drugs against cancer and healthy cells, to find the drugs that kill the cancer but do not damage the healthy cells. The sophisticated robotic equipment screens drugs much faster than manual methods - testing more drugs, more quickly and producing more detailed data. This year the Centre acquired new high content cell imaging technology and began developing an exciting platform for personalised medicine called in vitro drug sensitivity testing.

Cancer & Stem Cell Biology groupOur Cancer & Stem Cell Biology team has collaborations with several biotechnology companies in Australia and the US with whom this group is developing new clinical agents specifically targeting leukaemic stem cells. The goal is treatment of a leukaemia subtype with poor prognosis - acute myeloid leukaemia (AML).

Leukaemia Biology programScientists in our Leukaemia Biology program completed preclinical research that revealed an Australian-developed, clinically-approved adult leukaemia therapy called Venetoclax could successfully target high-risk leukaemia subtypes in infants or children. These scientists are lead authors on the subsequent Australian study published in prestigious US haematology journal Blood. Based on data from this study, the drug is now set to go into a paediatric clinical trial with the goal of improving treatments for children.

Tumour Biology & Targeting programThe Tumour Biology & Targeting program found that a cell cytoskeleton protein called stathmin affects the movement of tumour cells into and out of blood vessels. Blocking stathmin significantly reduces the ability of neuroblastoma cells to spread through the body, a finding that paves the way for new treatments for children with neuroblastoma.

Minimal Residual Disease groupThe Minimal Residual Disease group, in collaboration with Kids Cancer Centre at Sydney Children’s Hospital, Randwick, completed enrolment for the international Study 9 clinical trial (AIEOP-BFM ALL 2009). With NHMRC funding support, they performed initial Minimal Residual Disease (MRD) tests for the 333 Australian children diagnosed with acute lymphoblastic leukaemia in NSW and SA. With over 5000 patients worldwide on the trial, it will provide important data to guide treatment with the hope that more children will survive with fewer side effects.

Molecular Carcinogenesis programThe cancer-causing gene or oncogene MYCN is found in many cancers. To learn how it drives tumorigenesis, scientists in the Molecular Carcinogenesis Program conducted single-cell gene expression analysis of neuroblastoma cells. They discovered there is a strong association between MYCN and deregulation of cell division, creating opportunities for a new combination therapy.

Experimental Therapeutics & Molecular Diagnostics programs In collaboration with the Cancer Therapeutics Cooperative Research Centre (CTx), which translates Australian research discoveries into new cancer drugs, the Experimental Therapeutics and Molecular Diagnostics programs are optimising inhibitors of Multidrug Resistance Protein 1 (MRP1). The goal is that these new agents, developed from the group’s original research, will be used in the clinic to increase effectiveness of current standard-of-care drugs, particularly for high-risk neuroblastoma.

Histone Modification groupScientists in our Histone Modification group found that proteins histone methyltransferase DOT1L and presenter WDR5 are over-expressed in human neuroblastoma cells. They are the first scientists to discover that DOT1L plays a critical role in MYCN-mediated neuroblastoma, making it a potential therapeutic target for some of the more aggressive neuroblastoma types.

Targeted Therapy groupThe Targeted Therapy group has established a bank of 18 tumour samples from children with Diffuse Intrinsic Pontine Glioma (DIPG), a terminal brain cancer with no successful treatment. The program is the largest in Australia and owes its existence to the incredible generosity of parents wanting to help other families. Donated DIPG cells from autopsies have been grown in cultures to investigate this cancer’s biology and test new therapies.

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The impact of childhood cancer is devastating, not only for the child but for their siblings and parents too. From the first moment of diagnosis their world will be changed forever.

From our many years of research we know that childhood cancer has different causes and effects from adult cancer, so treatments designed for adults do not necessarily work for children. That is why our research is so important and thanks to your support, this year we have moved forward towards our goal of one day curing every child. In 2016, in partnership with the Kids Cancer Centre at Sydney Children’s Hospital, Randwick we successfully ran the pilot study for the new personalised medicine program, Zero Childhood Cancer, which will progress to a national clinical trial involving all Australian children’s hospitals in 2017.

While Children’s Cancer Institute is fortunate to receive substantial grant support from government

and funding bodies, this alone is not enough to fund our pioneering research. For every grant-funded dollar we receive, we need to match this with a dollar from our fundraising efforts.

For every grant-funded dollar we receive, we need to match this with a dollar from our fundraising efforts.”

Over the last four years our fundraising income has doubled to $16.5 million.

Our fundraising success is dependent on the people that support us, including our donors, corporate partners, trusts and foundations, community fundraisers, event participants, bequestors and volunteers.

In 2016 we benefited from significant income thanks to

generous contributions from bequestors - ordinary people who are helping us achieve extraordinary things by leaving a gift in their will. These generous bequestors are supporting our future medical research and giving families hope for a future in which we will be able to cure every child of cancer.

Perhaps the greatest gift anyone can give our research is a donation in their will to help change the future for with kids with cancer.

We’ve made great progress. But we won’t stop until the job is done and with your help we can get there much sooner. We are driven by the absolute belief that dedicated research will one day cure childhood cancer. With your support, we believe that our talented team of scientists will one day find the cure for every child.

Together, we are curing childhood cancer. It’s not if. It’s when.

Dare the BossDuring Childhood Cancer Awareness Month in September, we ran our second Dare the Boss campaign – a unique corporate team initiative that showed companies Australia-wide how to put the ‘fun’ back in ‘fundraising’. Bosses all over the country were dared by their staff to do all kinds of hair-raising activities, including skydiving, walking over glass, swimming with sharks, abseiling down cliff faces and many more challenging, bizarre and fantastic things!

Determined to prove they could take on any challenge for kids with cancer, these fearless leaders stepped up – supported by their creative colleagues who activated a huge range of extra initiatives to rally behind our cause. The 2016 Dare the Boss campaign raised over $250,000 for our research and proved to be an engaging concept for us to grow year on year with our corporate partners in the future.

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Build for a CureTo celebrate International Childhood Cancer Awareness Month in September 2016, Children’s Cancer Institute, McDonald Jones Homes and McCloy Group joined forces to undertake an extraordinary challenge – building a house and fully furnishing it in just 21 days. Everything required to build the house was donated including the land, all materials, trades, furnishings and even the real estate agent fees. Gold Logie winner Scott Cam came on-site to make sure everything went to plan and meet some of our most important visitors, the families of kids with cancer.

On the 23rd of October, the Lake Macquarie house was successfully sold with funds raised from the campaign totalling $727,000. All proceeds are helping fund our research that will one day find a cure for every child with cancer – children just like Nixon (pictured ).

FUNDRAISING HIGHLIGHTS

Why I giveWe often hear from our supporters who share their stories with us, about why they want to help us cure childhood cancer. The message below is from Sylvia, who has been donating to us since 2012, and has decided to generously leave Children’s Cancer Institute a gift in her will.

Healthy and happy children are one of the most important legacies that we can leave for future generations. I feel proud

that as a donor and a bequestor I can contribute to furthering the research that will one day bring a cure for all children with cancer.”

“- Sylvia, NSW


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Nixon, now aged 2 and pictured here at the Build for a Cure house, was born with cancer and began chemotherapy at just 2 weeks old.

Byron was diagnosed withleukaemia at 17 months old.Despite relapsing after his initialtreatment Byron is now seven years old and is doing well.

How our research is helping children like Isla, as told by her dad, Darrin:The 9th of September 2014 is a date my family will never forget – that was the day our lives changed forever because of childhood cancer.

My wife Sarah had taken our four-year-old daughter Isla to the doctor because she had been snoring and had pain in her knees for about 4 weeks. Doctors put it down to enlarged adenoids and growing pains and other than these minor complaints, Isla was a happy, healthy child. It was a Tuesday afternoon and I was driving home from work when Sarah rang to say the doctor wanted to see us both as soon as possible. We both went in and the doctor said to us: “Isla has leukaemia, you have to go to Sydney Children’s Hospital at Randwick tonight, they’re expecting you.”

Twelve hours later, Isla’s treatment for cancer had started at the Kid’s Cancer Centre. I remember Sarah asking the doctor, “Is she going to die?” and the doctor said “Sarah, they’ve come leaps and bounds with the treatment,” and we remember thinking, that’s not a “no”, we wanted a guarantee – we still want a guarantee, and that’s the hardest part.

Isla had test after test, agonisingly waiting on results with so many questions. What type of leukaemia does Isla have? Has it spread? How long is her treatment and what is the cure rate? Finally answers came. Isla was diagnosed with T–cell ALL which is an aggressive form of leukaemia. The life Isla knew and loved was now gone, to be replaced with an adult world of hospitals, pain and sickness and we were left wondering how do you tell a four-year-old what cancer is?

We desperately tried to maintain a normal life. After about a month, my wife and I returned to work as police officers working opposite shifts so one of us could be with Isla at all times and the other with

our other daughters as much as possible. The first 9 months of treatment were especially scary, intense and traumatic, both emotionally and physically for our family. Emotionally, Isla had a hard time when she lost her hair for the first time. She refused to have it cut when it first started falling out and was extremely self-conscious of her appearance. She finally accepted it for what it was when one day she was having treatment and every kid in the oncology ward was as bald as badgers.

Isla wasn’t a fan of the general anaesthetics or having her chemo port accessed to allow the chemo to be administered. We felt we were betraying her each time we held her down so the procedures could be done. As parents we are the ones that protect our children, not subject them to pain and trauma, and although we knew it was for the best, it’s impossible to explain to a four-year-old.

Isla’s diagnosis had a profound, lasting effect on our whole family”Our hearts broke each of the 21 times we saw Isla lying motionless on her bed in the operating theatre when she went under and we had to leave her. It was frightening when Isla started vomiting blood due to ulcers all the way through her body and she ended up in hospital for over a month and on a morphine drip for a week. But the mind is an amazing thing and thankfully Isla doesn’t remember a lot of the traumatic parts of her treatment.

Isla has 4 older sisters, Jordan, Hannah, Ainsley and Lily. Isla’s diagnosis had a profound lasting effect on our whole family; each of the girls has done something special as a result. Last year Jordan raised over $6000 to support other kids going through cancer, and after seeing Isla in hospital Hannah secretly enrolled in a nursing

degree and is hoping to specialise in paediatrics. When Isla lost her hair, Ainsley shaved her head to make Isla feel better and donated her hair to make wigs for cancer patients. At last year’s Children’s Cancer Institute Diamond Ball Lily donated a bike we were given, to help other kids with cancer. Sarah and her boss recently completed a 90m abseil in the Blue Mountains that raised over $8000 for the Institute’s Dare the Boss campaign and every year I participate in Endure for a Cure, the Institute’s signature cycling event where our team raised over $15,000 for research – I believe raising funds is so important because every dollar is getting them closer to the cure.

Unfortunately there are still 3 children a week who pass away because of cancer. That’s why we need to continue the research so we can get 100% cure rate for all children, for all cancers.

Just as the 9th of September is a day we want to forget, the 1st of November 2016 is one we will always celebrate - as that was the day Isla finished her treatment! Isla still has a long way to go and our family are realistic, but optimistic. We will live in fear of every bruise, every ache and pain dreading the leukaemia has come back, but we have hope that she is cured.

We have that hope because of research at Children’s Cancer Institute. We have been amazed by the determination, compassion and passion by everyone at the Institute to achieve a 100% cure rate for children’s cancer. By supporting the vital work of the Children’s Cancer Institute you also support us and Isla, and every child with cancer.

“Isla, aged 4, during one of her many hospital admissions.

“

Darrin and Sarah with their daughters Ainsley, Isla and Lily (l-r).

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ISLA’S STORY ANNUAL REVIEW 2016 HIGHLIGHTS

Financial Report

Statement of financial positionAs at 31 December 2016

31 Dec 2016 ($) 31 Dec 2015 ($)

ASSETS Current Assets Cash and cash equivalents 10,165,813 6,791,871 Restricted cash 51,745 Trade and other receivables 802,280 1,007,415 Other assets 1,845,358 1,975,399 Other financial assets 250,883 4,249,023

Total Current Assets 13,064,334 14,075,453

Non-current Assets Other assets 13,858,186 15,558,186 Available-for-sale financial assets 4,938 11,458 Property, plant and equipment 7,010,204 3,728,743 Intangible assets 707,441 603,871

Total Non-current Assets 21,580,769 19,902,258

Total Assets 34,645,103 33,977,711LIABILITIES

Current Liabilities Trade and other payables 2,183,265 2,080,675Provisions 2,067,090 1,952,265

Total Current Liabilities 4,250,355 4,032,940

Non-current Liabilities Provisions 157,765 159,045

Total Non-current Liabilities 157,765 159,045Total Liabilities 4,408,120 4,191,985 NET ASSETS 30,236,983 29,785,726 EQUITY Reserves 4,862,032 6,805,223Retained surplus 25,374,951 22,980,503

TOTAL EQUITY 30,236,983 29,785,726

Statement of comprehensive incomeFor the year ended 31 December 2016 2016 ($) 2015 ($) 2016 (%)

REVENUE Research 22,990,845 23,133,540 58% Fundraising 16,450,430 13,638,238 42% Other 183,454 144,321 -

39,624,729 36,916,099 100%EXPENSES Research and scientific activities 31,712,302 25,109,932 81%Fundraising 5,163,688 4,653,321 13%Support and administration 2,297,482 2,182,607 6%

39,173,472 31,945,860 100%

Total comprehensive income for the year 451,257 4,970,239

In 2016, 81 cents of every dollar spent within the Institute was spent directly on dedicated research to help cure childhood cancer.

81% Research & scientific activities to help cure childhood cancer

13% Fundraising activities to generate donations

6% Core support services & critical infrastructure costs

81%

13%

6%

Where the money goes:

FINANCIAL HIGHLIGHTS ANNUAL REVIEW 2016 HIGHLIGHTS

24

Mr Bart VogelDirector

BCom (Hons), FCAMr Vogel was appointed to the Board in 2007. He is the Chairman of Infomedia Limited and a non-executive director of Macquarie Telecom and BAI Communications.

During his 35 year executive career Bart was the CEO of Asurion Australia, and Lucent Technologies in Australia and Asia Pacific, and was a partner of Bain & Company, a global management consulting firm. His corporate advisory work has included 8 years as a partner at Deloitte. Mr Vogel Chairs the Audit and Risk Management Subcommittee and is a member of the University Relations Subcommittee.

Professor Les White AMDirector

AM DSc MBBS FRACP MHA AFACHSMEmeritus Professor Les White was appointed as the inaugural NSW Chief Paediatrician in September 2010, a position he held until his retirement in 2016.

Les was Executive Director of Sydney Children’s Hospital from 1995 to 2010, following a clinical and academic career, with emphasis on childhood cancer. Other positions have included President of Children’s Hospitals Australasia (1999-2004) and the John Beveridge Professor of Paediatrics (2005-2010). He has over 130 publications and many more abstracts, awards, grants and invited presentations in his CV.

Les serves on 8 not-for-profit boards relating to children’s health or medical research. He was awarded a Doctorate of Science for research contributions related to childhood cancer and holds a Master of Health Administration. In 2007 he received an Order of Australia award for service to medicine, medical administration and the community in the field of paediatrics. He has championed the rights of children and young people in healthcare settings. His current interests include childhood injury, intellectual disability, health services research and the networking of paediatric services. Emeritus Professor White is a member of the Remuneration and Nominations Subcommittee and the Scientific Advisory Committee.

Mr Lindsay Partridge AMDirector

BSc. Hons. Ceramic Eng, FAICD, Dip CDMr Partridge joined the Board in June 2012, after more than a decade of philanthropic support for CCIA and other charities. Lindsay is a ceramic engineer who has worked extensively in all facets of the Building and Construction industry in Australia and the United States prior to joining the Austral Bricks division of Brickworks Ltd in 1985. He held various senior management positions at Austral Bricks before being appointed Chief Executive Officer of Brickworks Ltd in 1999 and Managing Director in 2000.

From 2001 to 2003, Lindsay was a Director of Bristile Ltd. He has also had extensive industry body involvement and was the Founding Chairman of the Australian Brick and Blocklaying Training Foundation and is a Director of Think Brick Australia. Previously he was a Director of HIA (Housing Industry Association) Youthbuild and was elected HIA State Councillor. Lindsay is a Member of Manufacturing Australia. Mr Partridge is Chair of the Fundraising Subcommittee and the Commercialisation and IP Subcommittee.

Mr David JP SmithChairman

MBA, B.Bus, GAICD, FAIMMr Smith joined the Board in February 2012 and was elected Chairman on 1 November 2012. He has over 35 years’ experience in financial services in both Australia and New Zealand. He has over 20 years’ experience in CEO positions reporting to local and international Boards with Westpac Banking Corporation, Insurance Australia Group NZ Limited, Zurich Financial Services Australia and AXIS Specialty Australia.

Mr Smith is presently Co-Founder and Executive Director of Equip Corporate Advisory, a private consultancy business working with large and medium-sized organisations in the fields of corporate strategy, organisational effectiveness, employee relations and advocacy. Mr Smith holds an MBA from the Massachusetts Institute of Technology and was a 1997 Sloan Fellow. He also holds a Bachelor of Business degree, is a graduate member of the Australian Institute of Company Directors and is a Fellow of the Australian Institute of Management. Mr Smith is a member of the Remuneration and Nominations Subcommittee and Chairs the University Relations Subcommittee.

Professor Michelle Haber AMExecutive Director

BSc (Psych) (Hons), PhD, Hon DSc (UNSW), FAHMSProfessor Haber joined Children’s Cancer Institute as a staff scientist in 1984. She was appointed Director of the Institute in June 2000 and Executive Director in June 2003. Professor Haber is known for her world-class research into the treatment of neuroblastoma and acute lymphoblastic leukaemia in children.

Professor Haber holds a conjoint appointment as Professor in the Faculty of Medicine at the University of New South Wales. She has served as President (2010-2012) and currently serves on the Steering Committee of the International Advances in Neuroblastoma Research Association. In 2007 Professor Haber was appointed a Member of the Order of Australia for services to science in the field of research into childhood cancer, to scientific education and to the community.

In 2008, she was awarded an Honorary Doctorate from the University of New South Wales for her eminent service to the cancer research community. In 2014, Professor Haber was awarded the Cancer Institute NSW’s Premier’s Award for Outstanding Cancer Researcher of the Year, and in 2015 she was appointed an Inaugural Fellow of the Australian Academy of Health and Medical Sciences.

Ms Edwina JonesDirector

BPharmMrs Jones joined the Board in 2010. Prior to her retirement, Mrs Jones was a pharmacist, working in retail pharmacy and later as a consultant pharmacist. Her daughter, Emma, is a childhood cancer survivor, having been diagnosed in 1991 with osteogenic sarcoma.

Mrs Jones and her late husband, Spike, have been long-time supporters of CCI. Spike was on the CCI board from 1993 to 1998. Mrs Jones is a member of the Fundraising Sub-Committee.

OUR BOARD OF DIRECTORS

2726

Mr Peter Stirling BensonDirector

BA (Comb. Hon), MBA, GAICDMr Stirling Benson has extensive experience in the travel, media, entertainment and health sectors, both in Australia and overseas. He was a senior executive at Diageo and British Airways prior to holding a number of CEO positions over a period of 15 years in pay television (TV1), electronic distribution of travel services (Galileo), ticketing of major events (Ticketek) and the fitness industry (Fitness First).

Peter holds a BA (Combined Honours) from University College London, an MBA from the London Business School and is a Graduate of the Australian Institute of Company Directors. Peter has been appointed adjunct professor at UTS Business School, working on their MBA program for entrepreneurs (MBAe) and is a member of the 20/20 Exchange, a mentoring organisation, where he chairs a group of CEOs.

Peter is Chairman of the Remuneration and Nominations Subcommittee and a member of the Commercialisation and IP Subcommittee.

Emeritus Professor Richard Henry AMDirectorMB BS, MD, FRACP, Dip Clin Epi

Emeritus Professor Richard Henry works as a consultant in Health and Higher Education. He had a long career in academic medicine and hospital paediatrics before his appointment as Vice-President and Deputy Vice-Chancellor (Academic) of the University of New South Wales from 2006-2012.

Emeritus Professor Henry is a Pro-Chancellor of UNSW, a Trustee of Sydney Grammar School, Chair of the Centre for Social Impact and a Board member of Legal Aid NSW. He was appointed a Member of the Order of Australia in 2007 for service to paediatric respiratory medicine as a clinician, researcher, educator and mentor, and serving in a range of roles with professional medical organisations.

Emeritus Professor Henry is a member of the Audit and Risk Management Subcommittee, the University Relations Subcommittee and the Scientific Advisory Committee.

Mr Simon TruskettDirector

BCom, LLB; GAICDMr Truskett was appointed to the Board in 2006. He is a Partner at national law firm Clayton Utz, with more than 30 years’ experience advising Australian and foreign companies on a range of corporate and securities law issues in medical, health and other industries.

Mr Truskett was the founding National Partner responsible for community involvement at Clayton Utz (called Community Connect) and a director of the Clayton Utz Foundation. He is also a former director and Chair of Lex Mundi Limited, the world’s leading association of independent law firms. Mr Truskett is a member of the Audit and Risk Management Subcommittee and the Commercialisation and IP Subcommittee.

Mr Tim YuleDirector

BCom, M.Mgt, Grad Dip App Fin Mr Yule was appointed to the Board in December 2013. He is an experienced business leader within investment banking and financial services in Australia and Asia, with previous roles at Westpac Bank, Perpetual Investments and UBS Australia, and is currently Head of Distribution at Mason Stevens.

Over the last twenty years, he has led businesses servicing institutional, corporate and retail clients in a variety of areas including fixed income, interest rate and equity derivatives, structured finance, and commodities. He has significant sales, strategy and product development experience.

His daughter has completed treatment for childhood cancer. Mr Yule is a member of the Remuneration and Nominations Subcommittee, the Fundraising Subcommittee and the Audit and Risk Management Subcommittee.

Mr Matthew Burke OAMDirector (resigned from Board 12 October 2016)Mr Burke, who joined the Board in December 2015, was a member of the Australian Wallabies Rugby Union team from 1993 to 2004, being a part of the team that won the 1999 Rugby World Cup.

Mr Burke is currently a sports presenter on Network Ten Australia and is a published author and an in-demand guest speaker. Mr Burke has held past roles with the Newcastle Falcons Rugby Union (United Kingdom), New South Wales Rugby Union and the Australian Rugby Union. He has also had involvement with charity organisations such as the Leukaemia Foundation.

Professor Rodney PhillipsDirector

FMed.Sci. MA (Oxon) MD (Melb) FRCP FRACPProfessor Rodney Phillips is an immunologist whose research has impacted the world’s understanding of HIV/AIDS and other infectious diseases. A native Australian and graduate of the University of Melbourne, he worked for 35 years in the United Kingdom, primarily at Oxford University.

He is best known for describing, for the first time, how the HIV virus evades the body’s immune defences. He has conducted many years of bedside research in communities across Asia and Africa and has an enduring interest in the significant health challenges of the third world, such as malaria, tuberculosis and AIDS.

As a medical administrator, Professor Phillips has held numerous influential roles including Vice-Dean of Medical Sciences at Oxford University and Director of the Peter Medawar Building for Pathogen Research. He took up his appointment as Dean of UNSW Medicine in mid-2015, and joined the Board of Children’s Cancer Institute in October 2015.

OUR BOARD OF DIRECTORS CONTINUED

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Mrs Anne JohnstonHead of Fundraising & MarketingAnne joined Children’s Cancer Institute in October 2012 as Head of Fundraising, with extensive experience across the commercial and not for profit (NFP) sector. She is responsible for all fundraising income streams and is driving a strategy to grow these significantly to fund vital research that will achieve the Institute’s vision of curing childhood cancer in the foreseeable future.

Under Anne’s leadership, fundraising income has more than doubled since she joined the Institute four years ago. She also directed the successful launch of the Institute’s new brand in 2014, which continues to attract increased levels of corporate sponsorship and community support.

Previously Anne was at the Starlight Children’s Foundation Australia where she had headed Fundraising and Marketing for six years.

Prior to joining the NFP sector Anne was General Manager for Oroton Handbags and Actil bed linen in Australia heading up all aspects of these businesses from product design and supply to marketing and sales. In the UK Anne was Marketing Director for a major textile company for 12 years, with considerable experience across textiles and fashion retail which commenced as a graduate in Harrods department store.

Dr Peter WejboraHead of Research Development & OperationsPeter Wejbora joined Children’s Cancer Institute in July 2011 as Associate Director, responsible for advocacy and career development. In May 2012 he was appointed Head of Research Development & Operations with responsibility for driving research collaborations, partnerships and commercialisation. Prior to this he held the position of Director, Strategic Research Investment at the Cancer Institute NSW, the first state-wide cancer control agency in Australia. In this role he oversaw the development and delivery of support programs aimed at enhancing cancer research activity in NSW as well as facilitating the rapid translation of research evidence into practice.

Peter has worked in senior research development and management roles within the university sector, first at the University of Newcastle and later at Western Sydney University, where he carried responsibility for increasing research income across all university colleges.

Mrs Elaine NeesonHead of TechnologyElaine Neeson came to Children’s Cancer Institute in March 2007, bringing more than 15 years’ experience in business analysis and project management. She was initially engaged as Project Manager for the Institute’s relocation to the Lowy Cancer Research Centre.

In July 2008 she was appointed Risk & Compliance Manger and Project Manager for the Institute, working closely with the Fundraising team to ensure compliance and operational integrity. In November 2010, Elaine was appointed Head of Fundraising Operations, making use of her specialised skills in project management, compliance management and business analysis. In May 2012, Elaine was appointed Strategic Operations Manager, with guiding responsibility for the implementation of Children’s Cancer Institute’s five-year Strategic Plan.

In November 2015, Elaine was appointed Head of Technology for the Institute, integrating the Project Management Office and the IT department to deliver a cohesive technology approach for the organisation.

OUR EXECUTIVE TEAM

Professor Michelle Haber AM Executive Director, Head of Program, Experimental Therapeutics

BSc Psych (Hons), PhD, Hon DSc (UNSW) FAHMSProfessor Michelle Haber joined Children’s Cancer Institute as a staff scientist in 1984. She was appointed Director of Children’s Cancer Institute in June 2000 and Executive Director in 2003. Michelle is known for her world-class research into the treatment of neuroblastoma and acute lymphoblastic leukaemia in children. She holds a conjoint appointment as Professor in the Faculty of Medicine at UNSW Australia. Michelle is a Past President of the International Advances in Neuroblastoma Research Association (ANRA), the peak international body for neuroblastoma research and, in 2007, was appointed a Member of the Order of Australia for services to science in the field of research into childhood cancer, to scientific education and to the community.

In 2008, Michelle was awarded an Honorary Doctorate from UNSW Australia for her eminent service to the cancer research community and in 2014 she was awarded the Cancer Institute NSW Premier’s Award for Outstanding Cancer Researcher of the Year.

In 2015, Michelle was made an Inaugural Fellow of the Australian Academy of Health and Medical Sciences (AAHMS).

Professor Murray Norris AMDeputy Director, Head of Program, Molecular Diagnostics

BSc ANU, MAppSc NSWIT, PhD UNSWProfessor Murray Norris was one of the first three scientists to staff Children’s Cancer Institute when its research laboratories opened in 1984. He is Head of the Molecular Diagnostics Program, Director of the ACRF Drug Discovery Centre and was appointed Deputy Director of the Institute in 2000.

His research focuses on utilising new molecular genetic technologies to improve the diagnosis, risk classification and treatment of childhood cancer and he has developed and implemented unique technology enabling the early prediction of relapse in children with acute lymphoblastic leukaemia. Murray has an international research reputation in childhood neuroblastoma, particularly the molecular analysis of genes and their relationship with clinical variables. Key areas of his research are new therapeutic approaches to treating cancers and the role of the MYCN oncogene in neuroblastoma tumourigenesis.

Murray’s work has been recognised by a number of awards and he has an established record of state and national grant-funded research. He is also President of the international Advances in Neuroblastoma Research Association (2016–2018), the peak body representing neuroblastoma research worldwide.

In 2015, he was awarded an Order of Australia (AM) for significant service to medical research as a molecular biologist and pioneering development of treatments for cancer in children.

Murray is also Director of the newly opened UNSW Centre for Childhood Cancer Research.

Mr Mark BizerayChief Financial OfficerMark Bizeray joined the Institute in April 2011 and was appointed Chief Financial Officer and Company Secretary in May 2012. He is a CPA with over 14 years’ experience managing the finance function across a range of industries including the not-for-profit sector. He has extensive experience in financial management, planning and control as well as designing and implementing business strategies and governance frameworks. Mark brings a strong combination of technical, commercial and leadership skills to the Institute.

30

Professor Michelle Haber AM Executive Director, Head of Program, Experimental Therapeutics

BSc Psych (Hons), PhD, Hon DSc (UNSW) FAHMSProfessor Michelle Haber joined Children’s Cancer Institute as a staff scientist in 1984. She was appointed Director of Children’s Cancer Institute in June 2000 and Executive Director in 2003. Michelle is known for her world-class research into the treatment of neuroblastoma and acute lymphoblastic leukaemia in children. She holds a conjoint appointment as Professor in the Faculty of Medicine at UNSW Australia. Michelle is a Past President of the International Advances in Neuroblastoma Research Association (ANRA), the peak international body for neuroblastoma research and, in 2007, was appointed a Member of the Order of Australia for services to science in the field of research into childhood cancer, to scientific education and to the community.

In 2008, Michelle was awarded an Honorary Doctorate from UNSW Australia for her eminent service to the cancer research community and in 2014 she was awarded the Cancer Institute NSW Premier’s Award for Outstanding Cancer Researcher of the Year.

In 2015, Michelle was made an Inaugural Fellow of the Australian Academy of Health and Medical Sciences (AAHMS).

Professor Murray Norris AMDeputy Director, Head of Program, Molecular Diagnostics

BSc ANU, MAppSc NSWIT, PhD UNSWProfessor Murray Norris was one of the first three scientists to staff Children’s Cancer Institute when its research laboratories opened in 1984. He is Head of the Molecular Diagnostics Program, Director of the ACRF Drug Discovery Centre and was appointed Deputy Director of the Institute in 2000.

His research focuses on utilising new molecular genetic technologies to improve the diagnosis, risk classification and treatment of childhood cancer and he has developed and implemented unique technology enabling the early prediction of relapse in children with acute lymphoblastic leukaemia. Murray has an international research reputation in childhood neuroblastoma, particularly the molecular analysis of genes and their relationship with clinical variables. Key areas of his research are new therapeutic approaches to treating cancers and the role of the MYCN oncogene in neuroblastoma tumourigenesis.

Murray’s work has been recognised by a number of awards and he has an established record of state and national grant-funded research. He is also President of the international Advances in Neuroblastoma Research Association (2016–2018), the peak body representing neuroblastoma research worldwide.

In 2015, he was awarded an Order of Australia (AM) for significant service to medical research as a molecular biologist and pioneering development of treatments for cancer in children.

Murray is also Director of the newly opened UNSW Centre for Childhood Cancer Research.

Professor Glenn Marshall AMHead of Translational Research & Molecular Carcinogenesis

MBBS, MD UNSW, FRACPProfessor Glenn Marshall’s primary preclinical research interests include investigating the mechanisms by which normal embryonal cells become cancerous, and improving the effectiveness of non-cytotoxic anti-cancer therapy in child cancer. Glenn has a longstanding clinical and translational research focus on relapse detection by Minimal Residual Disease (MRD) testing in leukaemia, and the therapy of high-risk or relapsed leukaemia. With colleagues at the Children’s Hospital Westmead, John Hunter Children’s Hospital and Ronald McDonald Learning Program, he helped initiate the Learning Pathways Project for Children with Cancer in 2007.

In addition to his research activities, Glenn is a full-time paediatric haematologist and oncologist in the Kids Cancer Centre at Sydney Children’s Hospital, Randwick.

In 2011, Glenn was appointed Head of Translational Research at Children’s Cancer Institute and became Director of a research network of all child cancer clinicians and researchers in NSW, known as the Kids Cancer Alliance, funded by the Cancer Institute NSW. In 2016 he became the Director of the Chronic Illness in Childhood academic stream of a new translational research partnership, SPHERE.

With colleagues Professors Haber and Norris he received the Premier’s Award for Excellence in Translational Cancer Research and the NHMRC “Ten of the Best” award in 2012. He was profiled in The Lancet on 25 March 2013 as a successful clinician-scientist. He was awarded an Order of Australia (AM) in 2014 for significant service to medicine field of paediatric oncology.

OUR RESEARCH LEADERS

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Professor Maria KavallarisHead of Program, Tumour Biology & Targeting

BAppSci UTS, PhD UNSWProfessor Maria Kavallaris is Head of the Tumour Biology and Targeting Program at Children’s Cancer Institute and Director of the Australian Centre for NanoMedicine at UNSW. Her research contributions are internationally regarded and include identifying the mechanisms of action and resistance to anticancer drugs, discovering new protein interactions in cancer and the development of less toxic cancer therapies using nanotechnology. Professor Kavallaris has held numerous competitive fellowships. She is a National Health and Medical Research Council (NHMRC) Principal Research Fellow and Fellow of the Australian Academy of Health and Medical Sciences (AAHMS). Her research contributions have been recognised by international and national awards and prizes including an International Agency for Research on Cancer Fellowship, an American Association for Cancer Research Women in Cancer Research Award, NHMRC Career Development Award, a Young Tall Poppy Award and an Australian Museum Eureka Prize. She is regularly invited to speak at and chair international meetings. Professor Kavallaris has served on numerous committees including the Program Committee for the Annual Meeting of the American Association for Cancer Research and the International NanoMedicine conference, and on funding review panels. She serves on the Board of the Australian Institute for Policy and Science and has played a major role in advocating medical research through public outreach. She has served as President of the Australian Society for Medical Research (ASMR) and was recognised by the NHMRC in 2014 as an Australian ‘high achiever’ in health and medical research. She serves on the NHMRC Research Committee where she contributes to high level research policy.

Professor Richard LockHead of Program, Leukaemia Biology

BSc (Hons) UC Swansea, PhD LondonProfessor Richard Lock was recruited as Head of Children’s Cancer Institute’s Leukaemia Biology Program in 1998 from the position of Associate Professor, Department of Medicine and Department of Biochemistry and Molecular Biology, University of Louisville, Kentucky, USA. Prior to his move, he had attained an international reputation in the cancer-related fields of cell cycle control, drug resistance and mechanisms of programmed cell death (apoptosis).

Since arriving at the Institute, Richard has successfully developed a clinically relevant laboratory model for the in vivo growth of human acute lymphoblastic leukaemia cells – the first such model in Australia. The model now plays a central role in the preclinical evaluation of anticancer agents and the identification of new targets for targeted therapies.

Richard’s contribution to cancer research has been reflected in his authorship of around 150 peer-reviewed papers, including several in prestigious journals such as Blood, Cancer Research, Cell Stem Cell, Clinical Cancer Research, and Cancer Cell. He is currently a National Health and Medical Research Council Senior Research Fellow, and has been awarded research grants by the National Cancer Institute (USA), The Cancer Council NSW, and the National Health and Medical Research Council.

Professor Lock is Deputy Director of the newly opened UNSW Centre for Childhood Cancer Research.

A/Prof Richard CohnClinical Research Associate, Long Term Follow-up Project

MB, BCh Rand, DCH SA, FCP (Paed) SA, FRACPAssociate Professor Richard Cohn has a senior management role as the Clinical Program Director of Medicine and Diagnostics in the Sydney Children’s Hospitals Network (Randwick) and as Head of Clinical Oncology in the Kids Cancer Centre at the Hospital. He has a senior teaching role as a Conjoint Professor in the School of Woman’s and Children’s Health, UNSW Medicine with over 160 peer reviewed scientific publications and grant funding worth over $11 million. He is recognised nationally and internationally as a leader in the field of research in the field of Cancer Survivorship. He is the Director of the Late-effects Program at Sydney Children’s Hospital, chairs the Australian and New Zealand Late-effects subcommittee of ANZCHOG and chairs a subcommittee of the International Committee for Harmonization of Health Screening Guidelines for Childhood Cancer Survivors. He is the co-director (paediatric) of the National Centre for Cancer Survivorship at UNSW, a collaboration between Paediatric and Adult Oncology. The Behavioural Sciences Unit which he established in the Kids Cancer Centre is regarded as one of the premier centres in the Southern Hemisphere for paediatric/adolescent psychosocial health research.

Dr Jenny WangGroup Leader, Cancer & Stem Cell Biology

BSc, PhD MacqDr Jenny Wang was recruited to Children’s Cancer Institute in 2011 from Harvard Medical School and Harvard Stem Cell Institute, where she undertook postdoctoral research in cancer stem cell biology (2005–2011). Since relocating Jenny has established an independent stem cell research group and was awarded CINSW Career Development Fellowship in 2012 and ARC Future Fellowship in 2013. Her research has attracted a total of $2.5 millions in competitive project grant funding as Chief Investigator A since 2013.

Genetic and epigenetic abnormalities enable cancer stem cells to hijack normal stem cell self-renewal mechanisms that multiply out of control, causing cancer. Research in Jenny’s laboratory is focused on understanding the mechanisms that regulate aberrant self-renewal and drug resistance of malignant stem cells, and developing cancer stem cell-targeted therapies that are more effective and less toxic in patients who have a high risk of relapse and poor outcome.

Dr Karen MacKenzieSenior Scientist, Personalised Medicine Program

BAppSci UTS, PhD UNSWIn 2015 Karen was appointed Senior Scientist in the Institute’s newly established Personalised Medicine (PM) Program, a joint initiative between the Institute and The Sydney Children’s Hospitals Network. Her focus within the PM Program is on the development of a platform for pre-clinical evaluation of drug efficacy for children with aggressive sarcomas. She also oversees research that is intricately aligned with the PM Program, centred on molecular targets in paediatric solid tumours.

Leading into her appointment in the PM Program in 2015, Dr MacKenzie was Group Leader at the Institute (2002-2014), supervising a successful independent research team investigating the step-wise processes that underpin cancer development in soft-tissue and blood stem cells, with the overarching goal of exploiting these mechanisms as therapeutic targets. Her research has been published in highly regarded journals, including Cancer Res, Leukemia, Oncogene, Cancer Cell and J Biol Chem. She has recently been invited to write reviews for the prestigious journals Nature Reviews Cancer and Seminars in Cancer Biology. In total, she has published 55 papers, including three book chapters.

Dr MacKenzie has supported her research at the Institute with a continual source of independent peer-reviewed project grant funds from NHMRC, Cancer Council and other agencies. She received a NHMRC Career Development Fellowship 2008-2011 and Cancer Institute NSW Career Development Fellowships 2005-2007 and 2008-2010. She contributes to professional working parties, advisory committees, conference organisation, peer review processes and higher degree student supervision.

Dr MacKenzie is also a Conjoint Senior Lecturer at UNSW Australia.

A/Prof Rosemary SuttonGroup Manager, Minimal Residual Disease

BSc (ANU), PhD (USyd)Dr Rosemary Sutton joined Children’s Cancer Institute in 2001 as a Senior Scientist, following a successful career in translational research at CSIRO Livestock Industries. She manages the only accredited Australian MRD molecular diagnostics laboratory for patients enrolled on clinical trials for acute lymphoblastic leukaemia (ALL), and her team has developed unique MRD tests for more than 2000 patients since 2002. She has grants awarded from the National Health & Medical Research Council, Sporting Chance Cancer Foundation and hospital funding to support her research and diagnostics for three current clinical trials for children with leukaemia (Interfant 06, IntReALL SR 2010 and FORUMpedSCT 2012) and the young adult trial ALL6.

Rosemary has several international collaborations for her research which focuses on both understanding the molecular genetics of leukaemia in high risk and relapsed patients and developing novel methods to measure MRD. Collectively, these projects will improve decisions about the right treatment for clinical trial patients and for personalised medicine. Rosemary has published over 70 peer- reviewed journal articles.

Dr Tao LiuGroup Leader, Histone Modification in Cancer

BMed, MMed, PhDOriginally trained as a medical practitioner specialising in neurology, Dr Tao Liu joined Children’s Cancer Institute as a Senior Research Officer in 2003. Since 2004, he has been focusing his research on the roles of histone deacetylases, histone demethylases, histone methyltransferases and long noncoding RNAs in modulating gene transcription and tumourigenesis, and the roles of histone deacetylase inhibitors and histone methyltransferase inhibitors as anti-cancer agents.

In the past five years, Tao has been awarded research grants from various external funding bodies including Cure Cancer Australia, Cancer Council NSW, Cancer Institute NSW, National Health & Medical Research Council (NHMRC, Australia) and National Institutes of Health (USA). Over the past decade, Tao has authored a number of peer-reviewed publications in scientific journals including The Lancet, Proceedings of the National Academy of Sciences USA, PLOS Genetics and Cell Death & Differentiation.

Dr Greg ArndtHead, ACRF Drug Discovery Centre

BSc Hons, PhD (UofS)Dr Greg Arndt joined Children’s Cancer Institute in 2008 to establish the ACRF Drug Discovery Centre for Childhood Cancer (DDC). He received his PhD from the University of Saskatchewan (UofS), Canada in 1993 and worked as a postdoctoral fellow at the RW Johnson Pharmaceutical Research Institute.

Prior to joining the Institute, Greg was Research Director and Project Leader with Johnson & Johnson Research Pty Ltd, where he spent 15 years in increasingly senior roles focusing on cell-based screening as a tool for developing novel therapeutics. Upon joining the DDC, Greg underwent training at the Walter & Eliza Hall Institute of Medical Research (WEHI) to gain further experience with high throughput chemical screening in an academic setting.

Greg has also had significant academic involvement as an Adjunct Senior Lecturer (2005–2007) and Senior Visiting Fellow (2007–2013) in the School of Biotechnology and Biomolecular Science, UNSW Australia. In this context, he has successfully trained several Honours and PhD students and published numerous manuscripts in high-quality journals. His research has also led to the submission of several international patent applications.

In 2014, Greg became a Project Leader within the Cancer Therapeutics Cooperative Research Centre (CTx) as part of the six-year partnership between the Institute and CTx to find novel therapeutics for children with cancer and develop a unique platform to find personalised treatments for individual childhood cancer patients.

Dr David ZieglerGroup Leader, Targeted Therapy

BSc (Med), MBBS, FRACP, MDA/Prof David Ziegler is a paediatric oncologist with expertise in neuro- oncology and early phase clinical trials. A/Prof Ziegler completed his clinical training in paediatric haematology/oncology at Sydney Children’s Hospital, Randwick. From 2005-2007 he was a Fulbright Scholar at the Dana Farber Cancer Institute, Harvard Medical School and Children’s Hospital Boston. His research focused on the preclinical development and clinical translation of novel therapies for paediatric brain tumours.

A/Prof Ziegler has been a staff specialist at the Kids Cancer Centre at Sydney Children’s Hospital, Randwick, since 2007. He has established a translational research program to develop novel therapies for children with cancer and heads the Centre’s clinical trials unit and is a conjoint Associate Professor at UNSW Australia.

A/Prof Ziegler’s preclinical research, and the Targeted Therapies program, focus on establishing tailored therapeutics for children with high-risk brain tumours and other high-risk malignancies.

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ANNUAL REVIEW 2016

AWARDS & SCHOLARSHIPSAustralian Association of Chinese Biomedical Scientists Annual Scientific Meeting, Second Place in the CanCare Research Awards Chi Yan Ooi, PhD Student

Australian Society for Medical Research (ASMR) NSW Scientific Meeting, Eppendorf Best Poster Presentation Award Kimberley Hanssen, Honours Student

Convergent Bio‐Nano Science & Technology Bursary AwardDavid Chang, PhD Student

Fellow of the Australian Academy of Health and Medical Sciences (AAHMS)Professor Maria Kavallaris

Kids Cancer Alliance PhD Top-up ScholarshipAshleigh Fordham, PhD Student

St Vincent’s Campus Research Symposium, Best Student Poster AwardKimberley Hanssen, Honours Student

TOW Research Awards, Best Poster Presentation Kimberley Hanssen, Honours Student

Winifred Dickes Rost School of Medical Sciences Honours Prize For the best performance in the final year of the Bachelor of Science or Bachelor of Medical Science degree, Faculty of Medicine, UNSWKimberley Hanssen, Honours Student

COMPETITIVE RESEARCH GRANTSAustralian Institute of Nuclear Science and EngineeringResearch Grant2015-16 Copper Metabolism as a Target for Neuroblastoma Treatment: Effect Of Dextran-Catechin. O Vittorio

Australian Research CouncilCentre of Excellence 2014-20 ARC Centre of Excellence in Convergent Bio-Nano Science and Technology. T Davis, F Caruso, C Porter, J Gooding, N Voelcker, A Whittaker, N

Bunnett, M Kendall, E Crampin, S Kent, R Parton, M Kavallaris, B Boyd, M Kearnes, K Thurecht, A Johnston, P Thordarson, T Nann, S Corrie, K Dawson, C Hawker, D Haddleton, C Alexander, M Stevens, K McLean, N Abbott, D Lee, I Greguric, J Lewis Cooperative Research Centre 2014-20 Cooperative Research Centre: Cancer Therapeutics Cooperative Research Centre “CTx”. Monash University, Griffith University, Children’s Cancer Institute, CSIRO Materials Science and Engineering, Melbourne Health , The National Cancer Centre Singapore, Peter MacCallum Cancer Centre, Walter + Eliza Hall Institute of Medical ResearchDiscovery Grants2014-16 Fine-tuning the conformations of cyclic peptides: a paradigm for optimising synthetic efficiency and biological activity. L Hunter, E Pasquier 2014-16 Structural domains of beta-tubulin and their role in microtubule dynamics and transport. M Kavallaris, L Wordeman.

Future Fellowships2013-16 Identification of novel therapeutic targets for selectively eliminating cancer stem cells in paediatric leukaemia. JY Wang2013-16 The critical role of the class III histone deacetylase SIRT2 in stabilizing N·Myc oncoprotein. T Liu

Linkage Grants2014-17 The development of tuneable materials to allow the 3D printing of cells. M Kavallaris, P Gunning 2015-18 Linkage A gold-coated magnetic nanoparticle biosensor for detecting microRNA. JJ Gooding, R Williams, M Kavallaris

Cancer AustraliaPriority-driven Collaborative Cancer Research Grant2014-17 MRP4 and MRP3 transporters: potential new targets in ovarian cancer. M Henderson, A DeFazio, C Scott

Cancer Council NSW Program Grant2016-20 Improving outcomes for children with leukaemia through molecular targeted therapies. M Norris, M Haber, G Marshall, M Kavallaris, R Lock

Project Grants2015-17 Dyskerin as a novel therapeutic target in neoplastic cells. K MacKenzie, P Gunaratne, J Fletcher, B Liu, T Bryan2015-17 Identifying and targeting a novel self-renewal signalling cascade in leukemic stem cells. JY Wang, J Wang

Cancer Institute NSWCareer Development Fellowship2016-18 Nanomedicine-based treatment to inhibit lung cancer growth and metastases. J McCarroll

Early Career Fellowships 2014-16 Dextran-Catechin conjugation with SPIONs: a potential targeted therapy against gliobastoma multiforme. O Vittorio2014-16 Targeting mitochondria and lipid metabolism as novel therapeutic approach in Diffuse Intrinsic Pontine Glioma. A Kankean2016-18 Reversing glucocorticoid resistance in paediatric acute lymphoblastic leukaemia - a novel epigenetic mechanism linked to basic DNA structure. D Jing

Equipment Grants2015-16 A High Content Imager for Identification of Drugs for Precision Cancer Medicine. M Haber, K MacKenzie, G Marshall, J Pimanda, R Lock, D Ziegler, J Byrne, G Arndt 2015-2016 High-throughput five-laser flow cytometer for cancer research and cancer drug discovery. R Lock, G Arndt, V Chen, M Haber, KL MacKenzie, KL McDonald, P Phillips, J Pimanda, C Power, JY Wang2016-17 A Shared Mass Spectrometry Facility for Discovery and Quantitative Cancer Research, Proteomics and Lipidomics. R Lock, M Kavallaris, J Wong, A Don, B Cheung, N Verrills, Y Li, N Turner

Translational Cancer Research Centre 2011-16 Kids Cancer Alliance (KCA). G Marshall, M Haber, M Norris, I Alexander, R Reddel, L Dalla-Pozza 2016-21 Kids Cancer Alliance (KCA). G Marshall, M Haber, R Reddel, J Byrne, D Ziegler, GC Cowage, C Wakefield

Translational Program Grant2015-20 Experimental therapeutics for Myc-driven childhood cancer. G Marshall, M Norris, M Haber, C Wakefield

Community of Practice Grant2016-17 Transitioning BSN biobanks to certification. G Marshall, J Byrne

Research Infrastructure Grants2015-18 Prospective biobanking of child cancer samples for precision medicine and research. G Marshall2015-18 Continuing infrastructure support for highly skilled professional staff to manage the ACRF Drug Discovery Centre for Childhood Cancer, a world-class drug discovery and high throughput chemical screening facility. M Norris, M Haber, G Arndt, G Marshall, D Ziegler, R Lock, P Hogg

French National Institute for Cancer

Project Grant2015-16 Project Grant from the SIRIC-PACA Centre of Excellence. E Pasquier

National Health and Medical Research Council

Program Grants2012-16 Improved outcomes for children with cancer through improved target identification and drug discovery: neuroblastoma as a model. M Haber, G Marshall, M Norris2016-20 Precision nanomedicine-based diagnostics and therapeutics for refractory malignancies. M Kavallaris, T Davis, R Lock, J Gooding

Project Grants2012-18 IntReALL Phase II/III Clinical Trial – Evaluation of new therapies to improve survival of children with relapsed acute lymphoblastic leukaemia. T Revesz, R Sutton, R Lock, V Saha, A von Stackelberg2014-18 Improving risk evaluation and outcomes in paediatric acute lymphoblastic leukaemia. R Sutton, L Dalla-Pozza, T Revesz 2014-16 Targeting mitochondrial metabolism in diffuse intrinsic pontine gliomas as a novel therapeutic strategy. D Ziegler, P Hogg 2014-16 The oncogenic function of a histone H3K9 demethylase and its contribution to the aggressive malignant phenotype of leukaemia. JY Wang, J Wang2015-18 Targeted Inhibition of

the FACT (Facilitates Chromatin Transcription) Complex as a Novel Therapeutic Approach in Aggressive Childhood Cancers. D Ziegler, M Haber, A Gudkov2015-17 A novel molecular target capable of abrogating neuroblastoma development. M Norris, G Perini2016-18 Functional characterization and therapeutic targeting of the novel long noncoding RNA MYCN-AS. T Liu, M Dinger2016-18 Targeting JMJD6 gene gain for the therapy of neuroblastoma. T Liu, R George

Senior Research Fellowships2014-18 Regulation & targeting of the cancer cytoskeleton. M Kavallaris2014-18 Translational research initiatives in acute leukaemia. R Lock

Targeted Call for Research into Preparing Australia for the Genomics Revolution in Health Care2016-20 Preparing Australia for Genomic Medicine: A proposal by the Australian Genomics Health Alliance. K North, A Sinclair, M Little, N Laing, J Christodoulou, H Scott, A Oshlack, Natal Thorne, M Dinger, D Hansen A Lonie, D Schofield, R Ward C Gaff S Metcalfe, D Thorburn S B Fox D Thomas, D Hilton, D MacArthur, R Gibbs Baylor, T Speed, P Waring, G Baynam D Amor, M Buckley, N Waddell, J Fletcher, J McGaughran, C Meldrum, S Grimmond, G Taylor, A Papenfuss, J Mattick, L Botten, J Pearson, T Groza, R Sinnott, P Lorgelly, J Carlin, A Wilson, C Simons T Roscioli, M Field, D White, G Marshall, M Millward A Spurdle, P Ekert, I Winship, A Dobrovic, M Haber, J Trapani, J Desai, J Beilby, D Bowtell, G McArthur, Gillian Mitchell, R Johnstone, S Forrest, B Wainwright

La Fondation de France et UNICANCER

2016-17 Postdoctoral fellowship. M Le Grand

National Institutes of Health, National Cancer Institute (US)

2015-20 Pediatric Preclinical Testing Consortium. R Lock

National Collaborative Research Infrastructure Strategy (NCRIS)

2015-17 Drug Discovery Centre Salary Support. NCRIS 2016 Funding Program, Therapeutic Innovation

Australia. GM Arndt Royal Australian and NZ College of Radiologists2016-17 Cell uptake, cytotoxicity, heating efficiency and radiosensitizing property of iron oxide nanoparticles grafted with brushed phosphorylcholine in prostate cancer. DW Chang

TRUSTS & FOUNDATIONSAnthony Rothe Memorial Trust

2015-16 A novel class of glucocorticoid-sensitising compounds in acute lymphoblastic leukaemia. R Lock2016-17 Developing a novel therapeutic strategy for leukaemia stem cell eradication. JY Wang

Apex Foundation

2013-16 Develop more targeted therapy to improve survival rates for children with neuroblastoma. T Liu

Balnaves Foundation

2008-16 Young Researcher’s Fund

Brain Foundation

2016-17 Utilising global transcriptome analysis to develop a rational therapeutic strategy targeting microRNAs in glioblastoma. W Muskovic

The Bright Blue Police Commissioner’s Fund for Sick Kids

2014-2016 Seeking a cure for neuroblastoma. T Liu

Cure Brain Cancer Foundation

2015-19 Personalised Medicine Program, the future of Childhood cancer research and treatment. M Haber2014-17 Novel targeted chemotherapeutic agents against Diffuse Intrinsic Pontine Gliomas (DIPG). M Tsoli, D Ziegler

The Cure Starts Now

2015-16 Fenretinide as a noveltherapy for DIPG. M Tsoli2016-17 Radiosensitisation of diffuse intrinsic pontine gliomas by modulating the glucose metabolism. H Shen

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ANNUAL REVIEW 2016

TRUSTS & FOUNDATIONScont.Estate of the Late R T Hall

2014-16 Precision nanomedicine-based diagnostics and therapeutics for refractory malignancies. M Kavallaris

Ian Potter Foundation

Medical Research Grant2016-17 An Automated robotic workstation to identify drugs for personalised cancer medicine. GM Arndt

Travel Grants 2016 L Franshaw2016 H Shen 2016 A Ehteda

Inner Wheel Australia

Cord Blood Research Grants2016-17 Haematopoietic stem cells from umbilical cord blood to generate cancer specific immune effectors cells. A Dolnikov2016-17 Cord Blood Stem Cells for Ex vivo Expansion of Antigen Specific Natural Killer Cells for Multi-modality Cancer Immunotherapy. S Shen

The Kids’ Cancer Project

2011-17 Stathmin regulation of microRNA expression in neuroblastoma cells. M Kavallaris2015-17 Development of TRX-E-009-1 as a therapy for Diffuse Intrinsic Pontine Gliomas. D Ziegler2016-19 Bio-banking and personalised medicine software platform for the Kids Cancer Alliance. G Marshall, J Byrne 2016-19 Zero Childhood Cancer: Personalised Medicine Program. M Haber 2016-19 Pre-clinical development of novel immune therapy for paediatric cancers. A Dolnikov, T O’Brien2016-17 Additional support of a clinical trial of polyamine inhibition as a new treatment approach for relapsed neuroblastoma. M Haber, D Ziegler

Lions Club International Foundation

2016‐19 Lions Kid’s Cancer Genome

Project ‐ Whole Genome Sequencing and analysis of tumour (≥60X) and germline (≥30X) pairs for 400 children enrolled in the Zero Childhood Cancer Program. J Mattick, GM Marshall, M Haber

Robert Connor Dawes Foundation

Research Grant2014-16 Personalised mouse models for childhood brain tumour patients. D Ziegler

Travel Grants 2016 H Shen2016 A Ehteda

Roth Charitable Foundation

2016-17 Support for the Children’s Cancer Institute Drug Discovery Centre. Roth Charitable Foundation. GM Arndt

Sporting Chance Cancer Foundation

2008-17 Minimal Residual Disease testing for high risk leukaemia patients. R Sutton

Steadfast Foundation

2016-18 Development of Nanomedicines for the Treatment of Childhood Brain Cancer. J McCarroll

Sydney Children’s Hospital Foundation

Research Grant2015-16 Ex vivo expansion of cytolitic natural killer cells in combination immune therapy for relapsed and refractory paediatric solid tumour. S Shen, T O’Brien

Tour De Cure

Scott Canner Young Researcher Grant2016-17 Developing a novel targeted therapy to eradicate leukaemic stem cells. J Lynch

PUBLICATIONS1. André N, Banavali S, Pasquier E (2016). Paediatrics: Metronomics-fulfilling unmet needs beyond level A evidence. Nature Reviews Clinical Oncology, 13(8):469-470.

2. André N, Pasquier E (2016). Metronomics during palliative care in paediatric oncology? For sure! But

handle me with care. Acta Paediatrica, International Journal of Paediatrics, 105(8):874-875.

3. Arndt GM, MacKenzie KL (2016). New prospects for targeting telomerase beyond the telomere. Nature Reviews Cancer, 16(8):508-524.

4. Attiyeh EF, Maris JM, Lock RB, Reynolds CP, Kang MH, Carol H, Gorlick R, Kolb EA, Keir ST, Wu J, Landesman Y, Shacham S, Lyalin D, Kurmasheva RT, Houghton PJ, Smith MA (2016). Pharmacodynamic and genomic markers associated with response to the XPO1/CRM1 inhibitor selinexor (KPT-330): A report from the pediatric preclinical testing program. Pediatric Blood and Cancer, 63(2):276-286.

5. Baker A, Gregory GP, Verbrugge I, Kats L, Hilton JJ, Vidacs E, Lee EM, Lock RB, Zuber J, Shortt J, Johnstone RW (2016). The CDK9 inhibitor dinaciclib exerts potent apoptotic and antitumor effects in preclinical models of MLL-rearranged acute myeloid Leukemia. Cancer Research, 76(5):1158-1169.

6. Bingul M, Tan O, Gardner CR, Sutton SK, Arndt GM, Marshall GM, Cheung BB, Kumar N, Black DS (2016). Synthesis, characterization and anti-cancer activity of hydrazide derivatives incorporating a quinoline moiety. Molecules, 21(7):916.

7. Boer JM, Van Der Veer A, Rizopoulos D, Fiocco M, Sonneveld E, De Groot-Kruseman HA, Kuiper RP, Hoogerbrugge P, Horstmann M, Zaliova M, Palmi C, Trka J, Fronkova E, Emerenciano M, Do Socorro Pombo-De-Oliveira M, Mlynarski W, Szczepanski T, Nebral K, Attarbaschi A, Venn N, Sutton R, Schwab CJ, Enshaei A, Vora A, Stanulla M, Schrappe M, Cazzaniga G, Conter V, Zimmermann M, Moorman AV, Pieters R, Den Boer ML (2016). Prognostic value of rare IKZF1 deletion in childhood B-cell precursor acute lymphoblastic leukemia: An international collaborative study. Leukemia, 30(1):32-38.

8. Brumatti G, Ma C, Lalaoui N, Nguyen N-Y, Navarro M, Tanzer MC, Richmond J, Ghisi M, Salmon JM, Silke N, Pomilio G, Glaser SP, De Valle E, Gugasyan R, Gurthridge MA, Condon SM, Johnstone RW, Lock RB, Salvesen G, Wei A, Vaux DL, Ekert PG, Silke J (2016). The caspase-8 inhibitor emricasan combines with the SMAC mimetic birinapant to induce necroptosis and treat acute myeloid leukemia. Science Translational Medicine, 8(339):339ra69.

9. Carter DR, Sutton SK, Pajic M, Murray J, Sekyere EO, Fletcher J, Beckers A, De Preter K, Speleman F, George RE, Haber M, Norris MD, Cheung BB, Marshall GM (2016). Glutathione biosynthesis is upregulated at the initiation of MYCN-driven neuroblastoma tumorigenesis. Molecular Oncology, 10(6):866-878.

10. Chang W, Brohl AS, Patidar R, Sindiri S, Shern JF, Wei JS, Song YK, Yohe ME, Gryder B, Zhang S, Calzone KA, Shivaprasad N, Wen X, Badgett TC, Miettinen M, Hartman KR, League-Pascual JC, Trahair TN, Widemann BC, Merchant MS, Kaplan RN, Lin JC, Khan J (2016). Multidimensional clinomics for precision therapy of children and adolescent young adults with relapsed and refractory cancer: A report from the center for cancer research. Clinical Cancer Research, 22(15):3810-3820.

11. Churchman ML, Evans K, Richmond J, Robbins A, Jones L, Shapiro IM, Pachter JA, Weaver DT, Houghton PJ, Smith MA, Lock RB, Mullighan CG (2016). Synergism of FAK and tyrosine kinase inhibition in Ph(+) B-ALL. Journal of Clinical Investigation Insight, 1(4):e86082.

12. Cirillo G, Curcio M, Vittorio O, Iemma F, Restuccia D, Spizzirri UG, Puoci F, Picci N (2016). Polyphenol Conjugates and Human Health: A Perspective Review. Critical Reviews in Food Science and Nutrition, 56(2):326-337.

13. Cirillo G, Curcio M, Vittorio O, Spizzirri UG, Nicoletta FP, Picci N, Hampel S, Iemma F (2016). Dual Stimuli Responsive Gelatin-CNT Hybrid Films as a Versatile Tool for the Delivery of Anionic Drugs. Macromolecular Materials and Engineering, 301(12): 1537–1547.

14. Cirillo G, Spizzirri UG, Curcio M, Hampel S, Vittorio O, Restuccia D, Picci N, Iemma F (2016). Carbon nanohybrids as electro-responsive drug delivery systems. Mini-Reviews in Medicinal Chemistry, 16(8):658-668.

15. Davis T, Doyle H, Tobias V, Ellison DW, Ziegler DS (2016). Case report of spontaneous resolution of a congenital glioblastoma. Pediatrics, 137(4):e20150868.

16. De Winter LM, Hansen WLJ, van Steenbergen HW, Geusens P, Lenaerts J, Somers K, Stinissen P, van der Helm-van Mil AHM, Somers V (2016). Autoantibodies to two novel peptides in seronegative and early rheumatoid

arthritis. Rheumatology (United Kingdom), 55(8):1431-1436.

17. Decock A, Ongenaert M, Cannoodt R, Verniers K, Wilde BD, Laureys G, Van Roy N, Berbegall AP, Bienertova-Vasku J, Bown N, Clément N, Combaret V, Haber M, Hoyoux C, Murray J, Noguera R, Pierron G, Schleiermacher G, Schulte JH, Stallings RL, Tweddle DA, De Preter K, Speleman F, Vandesompele J (2016). Methyl-CpG-binding domain sequencing reveals a prognostic methylation signature in neuroblastoma. Oncotarget, 7(2):1960-1972.

18. Dolai S, Sia KCS, Robbins AK, Zhong L, Heatley SL, Vincent TL, Hochgräfe F, Sutton R, Kurmasheva RT, Revesz T, White DL, Houghton PJ, Smith MA, Teachey DT, Daly RJ, Raftery MJ, Lock RB (2016). Quantitative phosphotyrosine profiling of patient-derived xenografts identifies therapeutic targets in pediatric leukemia. Cancer Research, 76(9):2766-2777.

19. El-Hoss J, Jing D, Evans K, Toscan C, Xie J, Lee H, Taylor RA, Lawrence MG, Risbridger GP, MacKenzie KL, Sutton R, Lock RB (2016). A single nucleotide polymorphism genotyping platform for the authentication of patient derived xenografts. Oncotarget, 7(37):60475-60490.

20. Ercole F, Mansfeld F, Kavallaris M, Whittaker M, Quinn J, Halls M, Davis T (2016). Macromolecular Hydrogen Sulfide Donors Trigger Spatiotemporally Confined Changes in Cell Signaling. Biomacromolecules, 17(1):371–383.

21. Evageliou NF, Haber M, Vu A, Laetsch TW, Murray J, Gamble LD, Cheng NC, Liu K, Reese M, Corrigan KA, Ziegler DS, Webber H, Hayes CS, Pawel B, Marshall GM, Zhao H, Gilmour SK, Norris MD, Hogarty MD (2016). Polyamine antagonist therapies inhibit neuroblastoma initiation and progression. Clinical Cancer Research, 22(17):4391-4404.

22. Fabian J, Opitz D, Althoff K, Lodrini M, Hero B, Volland R, Beckers A, Preter K, Decock A, Patil N, Abba M, Kopp-Schneider A, Astrahantseff K, Wünschel J, Pfeil S, Ercu M, Künkele A, Hu J, Thole T, Schweizer L, Mechtersheimer G, Carter D, Cheung BB, Popanda O, Deimling A, Koster J, Versteeg R, Schwab M, Marshall GM, Speleman F, Erb U, Zoeller M, Allgayer H, Simon T, Fischer M, Kulozik AE, Eggert A, Witt O, Schulte JH, Deubzer HE (2016). MYCN

and HDAC5 transcriptionally repress CD9 to trigger invasion and metastasis in neuroblastoma. Oncotarget, 7(41):66344-66359.

23. Fife CM, Sagnella SM, Teo WS, Po’uha ST, Byrne FL, Yeap YC, Ng DC, Davis TP, McCarroll JA*, Maria Kavallaris* (2016). Stathmin mediates neuroblastoma metastasis in a tubulin-independent manner via RhoA/ROCK signaling and enhanced transendothelial migration. Oncogene, 36(4):501-511. * Corresponding Author.

24. Fletcher JI, Williams RT, Henderson MJ, Norris MD, Haber M (2016). ABC transporters as mediators of drug resistance and contributors to cancer cell biology. Drug Resistance Updates, 26:1-9.

25. French JD, Johnatty SE, Lu Y, Beesley J, Gao B, Kalimutho M, Henderson MJ, Russell AJ, Kar S, Chen X, Hillman KM, Kaufmann S, Sivakumaran H, O’Reilly M, Wang C, Korbie DJ, Lambrechts D, Despierre E, Van Nieuwenhuysen E, Lambrechts S, Vergote I, Karlan B, Lester J, Orsulic S, Walsh C, Fasching PA, Beckmann MW, Ekici AB, Hein A, Matsuo K, Hosono S, Pisterer J, Hillemanns P, Nakanishi T, Yatabe Y, Goodman MT, Lurie G, Matsuno RK, Thompson PJ, Pejovic T, Bean Y, Heitz F, Harter P, Du Bois A, Schwaab I, Hogdall E, Kjaer SK, Jensen A, Hogdall C, Lundvall L, Engelholm SA, Brown B, Flanagan JM, Metcalf MD, Siddiqui N, Sellers T, Fridley B, Cunningham J, Schildkraut JM, Iversen E, Weber RP, Brennan D, Berchuck A, Pharoah P, Harnett P, Norris MD, Haber M, Goode EL, Lee JS, Khanna KK, Meyer KB, Chenevix-Trench G, DeFazio A, Edwards SL, MacGregor S (2016). Germline polymorphisms in an enhancer of PSIP1 are associated with progression-free survival in epithelial ovarian cancer. Oncotarget, 7(6):6353-6368.

26. Gorlick R, Kolb EA, Keir ST, Maris JM, Lock RB, Carol H, Reynolds CP, Kang MH, Billups CA, Collins J, Kurmashev D, Kurmasheva RT, Houghton PJ, Smith MA (2016). Initial Testing of NSC 750854, a Novel Purine Analog, Against Pediatric Tumor Models by the Pediatric Preclinical Testing Program. Pediatric Blood and Cancer, 63(3):443-450.

27. Hau E, Shen H, Clark C, Graham PH, Koh ES, Mcdonald K (2016). The evolving roles and controversies of radiotherapy in the treatment of glioblastoma. Journal of Medical Radiation Sciences, 16(1):3-15.

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PUBLICATIONS cont.28. Huang Y, Thoms JAI, Tursky ML, Knezevic K, Beck D, Chandrakanthan V, Suryani S, Olivier J, Boulton A, Glaros EN, Thomas SR, Lock RB, MacKenzie KL, Bushweller JH, Wong JWH, Pimanda JE (2016). MAPK/ERK2 phosphorylates ERG at serine 283 in leukemic cells and promotes stem cell signatures and cell proliferation. Leukemia, 30(7):1552-1561.

29. Irving JA , Enshaei A, Parker CA, Sutton R, Kuiper RP, Erhorn A, Minto L, Venn NC, Law T, Yu J, Schwab C, Davies R, Matheson E, Davies A, Sonneveld E, den Boer ML, Love SB, Harrison CJ, Hoogerbrugge PM, Revesz T, Saha V, Moorman AV (2016). Integration of genetic and clinical risk factors improves prognostication in relapsed childhood B-cell precursor acute lymphoblastic leukaemia. Blood, 128(7):911-922.

30. Jones L, Carol H, Evans K, Richmond J, Houghton PJ, Smith MA, Lock RB (2016). A review of new agents evaluated against pediatric acute lymphoblastic leukemia by the Pediatric Preclinical Testing Program. Leukemia, 30(11):2133-2141.

31. Kang MH, Reynolds CP, Kolb EA, Gorlick R, Carol H, Lock RB, Keir ST, Maris JM, Wu J, Lyalin D, Kurmasheva RT, Houghton PJ, Smith MA (2016). Initial Testing (Stage 1) of MK-8242—A Novel MDM2 Inhibitor—by the Pediatric Preclinical Testing Program. Pediatric Blood and Cancer, 63(10):1744-1752.

32. Khaw SL, Suryani S, Evans K, Richmond J, Robbins A, Kurmasheva RT, Billups CA, Erickson SW, Guo Y, Houghton PJ, Smith MA, Carol H, Roberts AW, Huang DCS, Lock RB (2016). Venetoclax responses of pediatric ALL xenografts reveal sensitivity of MLL-rearranged leukemia. Blood, 128(10):1382-1395.

33. Kim PY, Tan O, Liu B, Trahair T, Liu T, Haber M, Norris MD, Marshall GM, Cheung BB (2016). High TDP43 expression is required for TRIM16-induced inhibition of cancer cell growth and correlated with good prognosis of neuroblastoma and breast cancer patients. Cancer Letters, 374(2):315-323.

34. Lalaoui N, Hänggi K, Brumatti G, Chau D, Nguyen N-YN, Vasilikos L,

Spilgies LM, Heckmann DA, Ma C, Ghisi M, Salmon JM, Matthews GM, de Valle E, Moujalled DM, Menon MB, Spall SK, Glaser SP, Richmond J, Lock RB, Condon SM, Gugasyan R, Gaestel M, Guthridge M, Johnstone RW, Munoz L, Wei A, Ekert PG, Vaux DL, Wong WWL, Silke J (2016). Targeting p38 or MK2 Enhances the Anti-Leukemic Activity of Smac-Mimetics. Cancer Cell, 29(2):145-158.

35. Levrier C, Sadowski MC, Rockstroh A, Gabrielli B, Kavallaris M, Lehman M, Davis RA, Nels α on CC (2016). 6α-Acetoxyanopterine: A novel structure class of mitotic inhibitor disrupting microtubule dynamics in prostate cancer cells. Molecular Cancer Therapeutics, 16(1):3-15.

36. Liu PY, Atmadibrata B, Mondal S, Tee AE, Liu T (2016). NCYM is upregulated by lncUSMycN and modulates N-Myc expression. International Journal of Oncology, 49(6):2464-2470.

37. Liu PY, Sokolowski N, Guo ST, Siddiqi F, Atmadibrata B, Telfer TJ, Sun Y, Zhang L, Yu D, Mccarroll J, Liu B, Yang RH, Guo XY, Tee AE, Itoh K, Wang J, Kavallaris M, Haber M, Norris MD, Cheung BB, Byrne JA, Ziegler DS, Marshall GM, Dinger ME, Codd R, Zhang XD, Liu T (2016). The BET bromodomain inhibitor exerts the most potent synergistic anticancer effects with quinone-containing compounds and anti-microtubule drugs. Oncotarget, 7(48):79217-79232.

38. Lopes BA, Meyer C, Barbosa TC, Stadt UZ, Horstmann M, Venn NC, Heatley S, White DL, Sutton R, Pombo-de-Oliveira MS, Marschalek R, Emerenciano M (2016). COBL is a novel hotspot for IKZF1 deletions in childhood acute lymphoblastic leukemia. Oncotarget, 7(33):53064-53073.

39. Lynch JR, Yi H, Casolari DA, Voli F, Gonzales-Aloy E, Fung TK, Liu B, Brown A, Liu T, Haber M, Norris MD, Lewis ID, So CWE, D’Andrea RJ, Wang JY (2016). Gaq signaling is required for the maintenance of MLL-AF9-induced acute myeloid leukemia. Leukemia, 30(8):1745-1748.

40. Magalon K, Le Grand M, El Waly B, Moulis M, Pruss R, Bordet T, Cayre M, Belenger P, Carré M, Durbec P (2016). Olesoxime favors oligodendrocyte differentiation through a functional interplay between mitochondria and microtubules. Neuropharmacology, 111:293-303.

41. McCarroll JA, Kavallaris M, Byrne FL (2016). Analyses of Tumor Burden In Vivo and Metastasis Ex Vivo Using Luciferase-Expressing Cancer Cells in an Orthotopic Mouse Model of Neuroblastoma. Methods in Molecular Biology, 1372:61-77.

42. Murphy B, Yin H, Maris JM, Kolb EA, Gorlick R, Reynolds CP, Kang MH, Keir ST, Kurmasheva RT, Dvorchik I, Wu J, Billups CA, Boateng N, Smith MA, Lock RB, Houghton PJ (2016). Evaluation of alternative in vivo drug screening methodology: single mouse analysis. Cancer Research, 76(19):5798-5809.

43. Oliver S, Thomas DS, Kavallaris M, Vittorio O, Boyer C (2016). Efficient Functionalisation of Dextran-Aldehyde with Catechin: Potential Applications in the treatment of Cancer. Polymer Chemistry, 7:2542-2552.

44. Oliver S, Vittorio O, Cirillo G, Boyer C (2016). Enhancing the therapeutic effects of polyphenols with macromolecules. Polymer Chemistry, 7:1529-1544.

45. Parchi PD, Vittorio O, Andreani L, Battistini P, Piolanti N, Marchetti S, Poggetti A, Lisanti M (2016). Nanoparticles for tendon healing and regeneration: Literature review. Frontiers in Aging Neuroscience, 8:202.

46. Parker AL, Turner N, McCarroll JA*, Kavallaris M* (2016). βIII- tubulin alters glucose metabolism and stress response signalling to promote cell survival and proliferation in glucose-starved non-small cell lung cancer. Carcinogenesis, 37(8):787-98.

47. Pasalic L, Williams R, Siupa A, Campbell H, Henderson MJ, Chen VMY (2016). Enumeration of extracellular vesicles by a new improved flow cytometric method is comparable to fluorescence mode nanoparticle tracking analysis. Nanomedicine, 12(4):977-986.

48. Pasquier E, Andre N, Street J, Chougule A, Rekhi B, Ghosh J, Philip DSJ, Meurer M, Mackenzie KL, Kavallaris M, Banavali SD (2016). Effective management of advanced angiosarcoma by the synergistic combination of propranolol and vinblastine-based metronomic chemotherapy: a bench to bedside study. EBioMedicine, 6:87-95.

49. Richmond J, Robbins A, Evans K, Beck D, Kurmasheva RT, Billups CA, Carol H, Heatley S, Sutton R, Marshall GM,

White D, Pimanda J, Houghton PJ, Smith MA, Lock RB (2016). Acute sensitivity of preclinical models of Ph-like acute lymphoblastic leukemia to the SMAC-mimetic birinapant. Cancer Research, 76(15):4579-4591.

50. Scheijen B, Boer JM, Marke R, Tijchon E, van Ingen Schenau D, Waanders E, van Emst L, van der Meer LT, Pieters R, Escherich G, Horstmann MA, Sonneveld E, Venn N, Sutton R, Dalla-Pozza L, Kuiper RP, Hoogerbrugge PM, den Boer ML, van Leeuwen FN (2016). Tumor suppressors BTG1 and IKZF1 cooperate during mouse leukemia development and increase relapse risk in B-cell precursor acute lymphoblastic leukemia patients. Haematologica, 102(3):541-551.

51. Shahbazi J, Liu PY, Atmadibrata B, Bradner JE, Marshall GM, Lock RB, Liu T (2016). The bromodomain inhibitor jq1 and the histone deacetylase inhibitor panobinostat synergistically reduce n-myc expression and induce anticancer effects. Clinical Cancer Research, 22(10):2534-2544.

52. Sharbeen G, McCarroll J, Liu J, Youkhana J, Limbri LF, Biankin AV, Johns A, Kavallaris M, Goldstein D, Phillips PA (2016). Delineating the Role of βIV-Tubulins in Pancreatic Cancer: βIVb-Tubulin Inhibition Sensitizes Pancreatic Cancer Cells to Vinca Alkaloids. Neoplasia, 18(12):753-764.

53. Sharbeen G, Youkhana J, Mawson A, McCarroll J, Nunez A, Biankin A, Johns A, Goldstein D, Phillips P (2016). MutY-Homolog (MYH) inhibition reduces pancreatic cancer cell growth and increases chemosensitivity. Oncotarget, 8(6):9216-9229.

54. Shen S, Ning X, Symonds G, Dolnikov A (2016). Stem Cell Approach to Generate Cancer Specific Immune Effectors Cells. International Journal of Stem Cell Research & Therapy, 3(1):3-022.

55. Smith AM, Dun MD, Lee EM, Harrison C, Kahl R, Flanagan H, Panicker N, Mashkani B, Don AS, Morris J, Toop H, Lock RB, Powell JA, Thomas D, Guthridge MA, Moore A, Ashman LK, Skelding KA, Enjeti A, Verrills NM (2016). Activation of protein phosphatase 2A in FLT3+ acute myeloid leukemia cells enhances the cytotoxicity of FLT3 tyrosine kinase inhibitors. Oncotarget, 7(30):47465-47478.

56. Somers K, Chudakova DA, Middlemiss SMC, Wen VW, Clifton M, Kwek A, Liu B, Mayoh C, Bongers A, Karsa M, Pan S, Cruikshank S, Scandlyn M, Hoang W, Imamura T, Kees UR, Gudkov AV, Chernova OB, Haber M, Norris MD, Henderson MJ (2016). CCI-007, a novel small molecule with cytotoxic activity against infant leukemia with MLL rearrangements. Oncotarget, 7(29):46067-46087.

57. Sutton SK, Carter DR, Kim P, Tan O, Arndt GM, Zhang XD, Baell J, Noll BD, Wang S, Kumar N, McArthur GA, Cheung BB, Marshall GM (2016). A novel compound which sensitizes BRAF wild-type melanoma cells to vemurafenib in a TRIM16-dependent manner. Oncotarget, 7(32):52166-52178.

58. Tee AE, Liu B, Song R, Li J, Pasquier E, Cheung BB, Jiang C, Marshall GM, Haber M, Norris MD, Fletcher JI, Dinger ME, Liu T (2016). The long noncoding RNA MALAT1 promotes tumor-driven angiogenesis by up-regulating pro-angiogenic gene expression. Oncotarget, 7(8):8663-8675.

59. Teo J, McCarroll JA, Boyer C, Youkhana J, Sagnella SM, Duong HTT, Liu J, Sharbeen G, Goldstein D, Davis TP, Kavallaris M, Phillips PA (2016). A Rationally Optimized Nanoparticle System for the Delivery of RNA Interference Therapeutics into Pancreatic Tumors in Vivo. Biomacromolecules, 17(7):2337-2351.

60. Trahair TN, Lock RB, Sutton R, Sia KCS, Evans K, Richmond J, Law T, Venn NC, Irving JA, Moore S, Nievergall E, Dang P, Heatley SL, White DL, Revesz T (2016). Xenograft-directed personalized therapy for a patient with refractory acute lymphoblastic leukemia. Bone Marrow Transplant, 51(9):1279-82.

61. Tsoli M, Swarbrick MM, Robertson GR (2016). Lipolytic and thermogenic depletion of adipose tissue in cancer cachexia. Seminars in Cell and Developmental Biology, 54:68-81.

62. Unnikrishnan A, Guan YF, Huang Y, Beck D, Thoms JAI, Peirs S, Knezevic K, Ma S, Van de Walle I, de Jong I, Ali Z, Zhong L, Raftery MJ, Taghon T, Larsson J, MacKenzie KL, Van Vlierberghe P, Wong JWH, Pimanda JE (2016). A quantitative proteomics approach identifies ETV6 and IKZF1 as new regulators of an ERG-driven transcriptional network. Nucleic Acids Research, 7(30):47479-47493.

63. Vittorio O, Brandl M, Cirillo G, Kimpton K, Hinde E, Duong H, Gaus K, Yee E, Kumar N, Fleming C, Haber M, Norris M, Boyer C, Kavallaris M (2016). Dextran-Catechin: an anticancer chemically-modified natural compound targeting copper that attenuates neuroblastoma growth. Oncotarget, 7(30):47479-47493.

64. Vittorio O, Cojoc M, Curcio M, Spizzirri UG, Hampel S, Nicolette FP, Iemma F, Dubrovska A, Kavallaris M, Cirillo G* (2016). Polyphenol conjugates by immobilized laccase: the green synthesis of Dextran-Catechin. Macromolecular Chemistry & Physics, 217(13):1488-1492. * Corresponding Author.

65. Wojciechowski JP, Martin AD, Mason AF, Fife CM, Sagnella SM, Kavallaris M, Thordarson P (2016). Choice of Capping Group in Tripeptide Hydrogels Influences Viability in the Three-Dimensional Cell Culture of Tumor Spheroids. ChemPlusChem [Epub ahead of print DOI: 10.1002/cplu.201600464].

66. Xi Z, Yao M, Li Y, Xie C, Holst J, Liu T, Cai S, Lao Y, Tan H, Xu H-X, Dong Q (2016). Guttiferone K impedes cell cycle re-entry of quiescent prostate cancer cells via stabilization of FBXW7 and subsequent c-MYC degradation. Cell Death and Disease, 7(6):e2252.

67. Xue C, Yu D, Gherardi S, Koach J, Milazzo G, Gamble L, Liu B, Valli E, Russell AJ, London WB, Liu T, Cheung BB, Marshall GM, Perini G, Haber M, Norris MD (2016). MYCN promotes neuroblastoma malignancy by establishing a regulatory circuit with transcription factor AP4. Oncotarget, 7(34):54937-54951.

68. Yadav BD, Samuels AL, Wells JE, Sutton R, Venn NC, Bendak K, Anderson D, Marshall GM, Cole CH, Beesley A, Kees UR, Lock RB (2016). Heterogeneity in mechanisms of emergent resistance in pediatric T-cell acute lymphoblastic leukemia. Oncotarget, 7(37):58728-58742.

Book Chapters

1. Mansfeld FM, Davis TP, Kavallaris M (2016). Nanotechnology in Medical Research, Micro- and Nanotechnology in Vaccine Development.

2. Shen H, Hau E (2016). Resistance of Glioblastomas to Radiation Therapy. Resistance to Targeted Therapies Against Adult Brain Cancers.

4140

SEMINARS & PRESENTATIONSArndt GM, Failes TW, Mariana A, Kao F, Toscan C, Tsoli M, Andrews L, Diakiw S, Cheung BB, Cheung L, Maritz M, Morgan L, Fletcher JI, Ziegler DS, MacKenzie KL, Lock RB, Kavallaris M, Marshall GM, Norris MD, Haber M (2016). The ACRF Drug Discovery Centre: an early-stage drug discovery facility. 28th Lorne Cancer Conference, Lorne, Vic.

Bernardino R, Di Candia G, Giordani G, De Giorgio E, De Grandis L, Milazzo G, Erriques D, Ferrucci F, Norris MD, Haber M, Perini G (2016). Altering the MYCN/MAX ratio in a drosophila MYCN model leads to homeotic transformation of the eye to wing through deregulation of specific HOX genes. 17th Advances in Neuroblastoma Research Congress, Cairns, QLD.

Carter D, Cheung BB, Beck D, Beckers A, De Preter K, Speleman F, Norris M, Haber M, Swarbrick A, Marshall GM (2016). Identifying mechanisms of neuroblastoma tumorigenesis using single cell transcriptomics. 17th Advances in Neuroblastoma Research Congress, Cairns, QLD.

Cheung BB, Carter D, Murray J, Norris M, Haber M, Marshall GM (2016). Therapeutic targeting of the MYC signal by inhibition of histone chaperone FACT in neuroblastoma. Australian Society for Medical Research Annual Scientific Meeting, Gold Coast, QLD.

Cheung BB, Tan O, Koach J, Sutton S, Carter D, Diakiw SM, Au CG, Jankowski K, Trahair T, Marshall GM (2016). Identification of novel small molecule compounds to restore sensitivity to trophic factor withdrawal in MYCN-initiated death resistant cells. 17th Advances in Neuroblastoma Research Congress, Cairns, QLD.

Dagg R, Napier C, Reyolds CP, Haber M, Reddel R, Lau L (2016). A unique mechanism for the continual proliferation of neuroblastoma cells. 17th Advances in Neuroblastoma Research Congress, Cairns, QLD.

Ehteda A, Tsoli M, Chitranjan A, Chang C, Simon S, Kankean A, Shen H, Liu J, Valvi S, Franshaw L, Haber M, Gudkov A, Ziegler DS (2016). Combination of epigenetic modifiers CBL0137 and panobinostat is highly potent in vitro

and in vivo for Diffuse Intrinsic Pontine Glioma. ISPNO Conference, Liverpool, UK.

Failes TW, Morgan L, Mariana A, Wadham C, Trahair T, MacKenzie KL, Mould E, Fletcher JI, Kamili A, Tsoli M, Ziegler DS, Lock RB, Xie A, Sutton R, Norris MD, Haber M, Arndt GM (2016). In vitro drug sensitivity testing of high risk childhood patient cells for potential new drug treatments. 28th Lorne Cancer Conference, Lorne, Vic.

Flynn A, Benavides A, Vu A, Liu K, Scadden E, Attiyeh E, Haber M, Norris MD, Bassiri H, Hogarty MD (2016). Effects on tumour cells and the immune microenvironment may both contribute to the anti-tumor activities of DFMO in neuroblastoma pre-clinical models. 17th Advances in Neuroblastoma Research Congress, Cairns, QLD.

Gamble L, Murray J, Eden G, Allan S, Biktasova A, Kosciolek A, Marshall GM, Ziegler DS, Hogarty MD, Norris MD, Haber M (2016). Targeting the polyamine pathway in combination with conventional chemotherapy for the treatment of childhood neuroblastoma. 17th Advances in Neuroblastoma Research Congress, Cairns, QLD.

Gana C, Yu DMT, Murray J, Cheung L, Flemming CL, Gudkov AV, Purmal A, Fletcher JI, Norris MD, Haber M (2016). New, highly selective MRP1 inhibitors show promising preclinical activity in neuroblastoma. 17th Advances in Neuroblastoma Research Congress, Cairns, QLD.

Gao J, Somers K, Hannan KM, Liu B, Sivarajasingam S, Hannan RD, Pearson RB, Haber M, Norris MD, Henderson MJ (2016). The ABC transporter ABCE1 is a therapeutic target in neuroblastoma. 17th Advances in Neuroblastoma Research Congress, Cairns, QLD.

Gonzales-Aloy E, Norris MD, Wang JY (2016). Suppression of miR-101a contributes to leukemogenesis by regulating epigenetic and pro-survival pathways in MLL AML stem cells. American Association for Cancer Research Annual Meeting, New Orleans, USA.

Jing D, Beck D, Thoms JAI, Pimanda JE, Lock RB (2016). A novel epigenetic mechanism in glucocorticoid resistant paediatric acute lymphoblastic leukaemia. New Directions in

Leukaemia Research Meeting, Noosa, QLD.

Jing D, Lock RB (2016). A novel epigenetic mechanism associated with glucocorticoid resistance in pediatric acute lymphoblastic leukemia. BIT’s 9th Annual World Cancer Congress, Shanghai, China.

Jung MS, Russell AJ, Liu B, George J, Liu PY, Liu T, DeFazio A, Bowtell DL, Oberthuer A, London WB, Fletcher JI, Haber M, Norris MD, Henderson MJ (2016). An 18-gene Myc activity signature predicts poor clinical outcome in multiple Myc-associated cancer types. 17th Advances in Neuroblastoma Research Congress, Cairns, QLD.

Kamili A, Lockwood MJ, Wadham C, Mould E, Failes TW, Mariana A, Morgan L, Arndt GM, Reynolds CP, Norris MD, Haber M, Trahair TN, Fletcher JI (2016). Ex vivo drug screening as a strategy for personalised therapy in high-risk neuroblastoma. 17th Advances in Neuroblastoma Research Congress, Cairns, QLD.

Karsa M, Somers K, Mariana A, Failes T, Arndt G, Haber M, Norris M, Sutton R, Lock R, Henderson M (2016).Repositioning existing drugs as novel MLL-rearranged leukaemia therapies. CTx Higher Degree Research Symposium, Melbourne, Vic.

Kim P, Rahmanto AS, Tan O, Sutton S, Dilda PJ, Chung S, Norris MD, Haber M, Hogg PJ, Cheung BB, Marshall GM (2016). Combination of HDAC and mitochondrial-targeted metabolism inhibitors exhibits strong therapeutic synergy in vitro and in vivo against neuroblastoma. 17th Advances in Neuroblastoma Research Congress, Cairns, QLD.

Koach J, Murray J, Mccarroll J, Milazzo G, Perini G, Haber M, Norris M, Fletcher J, Cheung BB, Marshall GM (2016). Targeting a novel MYCN oncofactor, PA2G4, for the treatment of Neuroblastoma. 17th Advances in Neuroblastoma Research Congress, Cairns, QLD.

Koach J, Murray JE, McCarroll J, Milazzo G, Perini G, Haber M, Norris MD, Fletcher JI, Cheung BB, Marshall GM (2016). Targeting PA2G4, a novel MYCN co-factor, for the treatment of Neuroblastoma. European Association for Cancer Research, Manchester, UK.

Koach J, Murray JE, McCarroll J, Milazzo G, Perini G, Haber M, Norris

MD, Fletcher JI, Cheung BB, Marshall GM (2016). Targeting a novel MYCN onco-factor, PA2G4, for the treatment of neuroblastoma. 17th Advances in Neuroblastoma Research Congress, Cairns, QLD.

Lopes BA, Meyer C, Barbosa TC, zur Stadt U, Venn NC, Sutton R, Pombo-de-Oliveira MS, Marschalek R, Emerenciano M (2016). COBL: A Novel Hotspot For IKZF1 Deletions in Childhood Acute Lymphoblastic Leukemia. 10th Biennial Childhood Leukemia Symposium, Athens, Greece.

Mateos M, Trahair T, Quinn M, Barbaro P, Sutton R, Revesz T, Alvaro F, Dalla-Pozza L, Mechinaud F, MacGregor S, Marshall GM (2016). Evaluation of Risk of All Treatment-Related Side-Effects: The Erase Study. 10th Biennial Childhood Leukemia Symposium, Athens, Greece.

Murray JE, Valli E, Troung A, Eden G, Allan S, Webber H, Tivnan A, Tan A, Flemming C, Cheung L, Hanssen K, Carnegie-Clark A, Ruhle M, Henderson MJ, Marshall GM, Yu D, Norris MD, Fletcher JI, Haber M (2016). Suppression of multidrug resistant protein 4 inhibits neuroblastoma growth both in vitro and in vivo. 17th Advances in Neuroblastoma Research Congress, Cairns, QLD.

O’Brien R, Tran S, Maritz M, Liu B, Cheng FK, Purgato S, Yang C, Murray J, Russell AJ, Flemming CL, von Jonquieres G, Pickett HA, London WB, Haber M, Gunaratne P, Norris MD, Perini G, Fletcher JI, MacKenzie KL (2016). Elevated expression of dyskerin is a potential therapeutic target with a telomerase-independent role in Myc/N-Myc driven neuroblastoma. 17th Advances in Neuroblastoma Research Congress, Cairns, QLD.

Ooi CY, Carter DR, Liu B, Beckers A, De Brouwer S, De Preter K, Norris MD, Haber M, Speleman F, Liu T, Cheung BB, Marshall GM (2016). MicroRNA-204 suppresses neuroblastoma tumour growth through down-regulation of MYCN oncogene. 17th Advances in Neuroblastoma Research Congress, Cairns, QLD.

Ooi CY, Carter DR, Liu B, Mayoh C, Beckers A, De Brouwer S, Norris MD, Haber M, Liu T, De Preter K, Speleman F, Cheung BB, Marshall GM (2016). MicroRNA-204 is a tumour suppressor that down-regulates MYCN oncogene and suppresses tumour growth in

neuroblastoma. Australian Association of Chinese Biomedical Scientists 10th Annual Scientific Meeting, St George Research and Education Centre, Sydney, NSW.

Roundhill E, Turnbull D, Fletcher JI, Norris MD, Haber M, Burchill S (2016). Expression, trafficking and biological significance of mitochondrial MRP1 in neuroblastoma. 17th Advances in Neuroblastoma Research Congress, Cairns, QLD.

Schreiber B, Venn NC, Muskovic W, Giles JE, Law T, Trahair TN, Sutton R (2016). New dual purpose MRD markers based on ERG and IKZF1 deletions in B-cell ALL patients. New Directions in Leukemia Research, Noosa, QLD.

Shahabzi J, Liu P, Atmadibrata B, Bradner JE, Marshall GM, Lock R, Liu T (2016). Combination therapy with the bromodomain inhibitor JQ1 and the histone deacetylase inhibitor panobinostat synergistically reduce LIN28B gene and N-Myc protein expression and suppress neuroblastoma progression. 17th Advances in Neuroblastoma Research Congress, Cairns, QLD.

Shaw P, Gottlieb D, Antonenas V, Law T, Venn N, Sutton R (2016). Is speed of DLI Important To Avoid Overt Relapse After MRD Positivity Post HSCT. 10th Biennial Childhood Leukemia Symposium, Athens, Greece.

Sutton R, Parker C, Law T, Wadham C, Venn NC, Mould E, Reeves M, Love S, Hancock J, Hoogerbrugge P, Fraser C, Lock R, Revesz T, Saha V (2016). Low MRD predicts favourable outcome in High Risk Relapsed Childhood ALL. New Directions in Leukaemia Research, Noosa, QLD.

Sutton R, Venn N, Schreiber B, Muskovic W, Giles JE, Law T, Doculara L, Norris MD, Trahair T (2016). Ready-made Minimal Residual Disease Tests for ALL Patients Based on Recurrent Microdeletions. 10th Biennial Childhood Leukemia Symposium, Athens, Greece.

Sutton R, Venn N, Wadham C, Law T, Lock R, Meyer C, Marshalek R, Marshall GM, Norris MD, Trahair T (2016). Analysis of MRD Levels and Quantitative Gene Expression In Infant ALL. 10th Biennial Childhood Leukemia Symposium, Athens, Greece.

Tee A, Liu P, Milazzo G, Tan E-Y, Modal S, Atmadibrata B, Sun Y, Norris MD,

Haber M, Marshall GM, Vandesompele J, Dinger ME, Perini G, Mestdagh P, Liu T (2016). Suppressing the expression of a single novel long noncoding RNA leads to neuroblastoma regression or eradication. 17th Advances in Neuroblastoma Research Congress, Cairns, QLD.

Tee AE, Wong M, Bell J, Sun Y, Liu PY, Hüttelmaier S, Polly P, Scarlett CJ, Liu T (2016). The histone methyltransferase DOT1L induces neuroblastoma progression by regulating gene transcription. 17th Advances in Neuroblastoma Research Congress, Cairns, QLD.

Toscan C, Jing D, Failes T, Arndt GM, Rahimi M, McAlpine S, Lock R (2016). Thioimidazoline based compounds reverse glucocorticoid resistance in paediatric acute lymphoblastic leukaemia. New Directions in Leukaemia Research Meeting, Noosa, QLD.

Tyrrell V (2016). Zero Childhood Cancer Personalised Medicine Program. ANZCHOG Australian Central Nervous System Meeting, Cairns, QLD.

Tyrrell V, Arndt GM, Failes T, Fletcher J, Lock R, MacKenzie K, Mayoh C, Mould E, Sutton R, Tsoli M, Wadham C, Lau L, Trahair T, Wakefield C, Ziegler D, Tucker K, Gifford A, Anozado A, Dalla Pozza L, McCowage G, Byrne J, Saletta F, White D, Moore A, Gottardo N, Kotecha R, Hansford J, Mechinaud F, Revesz T, Tapp H, Ekert P, Fox S, Fellowes A, Kumar A, Papenfuss A, Dinger M, Kaplan W, Thomas D, Bhadri V, Boddy A, Khan J, Pfister S, Norris M, Haber M, Marshall GM (2016). Translating Research into the Paediatric Oncology Clinic: Personalised treatment for high risk cancer patients using a multidisciplinary integrative approach. 5th Annual NHMRC Symposium on Research Translation, Melbourne, Vic.

Tyrrell V, Mould E, Lau L, Wadham C, Xie A, Lock R, MacKenzie K, Ekert P, Ziegler D, Trahair T, Kumar A, Fellowes A, Haber M, Marshall GM (2016). Translating Research in to the Paediatric Oncology Clinic: A clinical trial to assess the feasibility and clinical value of a diagnostic platform for identifying personalised cancer treatment for high risk paediatric and AYA cancer patients using a multidisciplinary integrative approach. Human Genetics Society of Australasia Annual Scientific Meeting, Hobart, Tas.

ANNUAL REVIEW 2016

SEMINARS & PRESENTATIONS cont.Tyrrell V, Ziegler D (2016). Zero Childhood Cancer Personalised Medicine Program. ANZCHOG Australian Children’s Cancer Trials (ACCT) Meeting, Cairns, QLD.

Unnikrishnan A, Papaemmanuil E, Beck D, Verma A, Kumari A, Woll P, Richards L, Knezevic K, Chandrakanthan V, Thoms J, Tursky M, Huang Y, Ali Z, Olivier J, Galbraith S, Kulasekararaj A, Tobiasson M, Karimi M, Pellagatti A, Wilson S, Lindeman R, Young B, Ramakrishna R, Arthur C Stark R, Crispin P, Curnow J, Warburton P, Roncolato F, Boultwood J, Lynch K, Jacobsen S, Mufti G, Hellstrom-Lindberg E, MacKenzie K, Wong J, Campbell P, Pimanda J (2016). Integrative genomics identifies the molecular basis of resistance to azacitidine therapy in myelodysplastic syndromes. 21st Congress of the European-Hematology-Association, Copenhagen, Denmark.

Venn N, Law T, Wadham C, Mould E, Trahair TN, Sutton R (2016). Analysis of MRD and gene expression in ‘infant ALL 2006’. New Directions in Leukaemia Research, Noosa, QLD.

Vittorio O, Brandl M, Cirillo G, Kimpton K, Hinde E, Duong HTT, Boyer C, Flemming C, Yee EMH, Kumar N, Parmar A, Pascali G, Charil A, Haber M, Norris MD, Kavallaris M (2016). Dextran-Catechin conjugate: An anticancer nano-modified natural compound targeting copper metabolism in neuroblastoma. American Association of Cancer Research, New Orleans, LA, USA.

Vittorio O, Brandl M, Cirillo G, Kimpton K, Hinde E, Flemming C, Haber M, Norris MD, Parmar A, Pascall G, Charil A, Kavallaris M (2016). Dextran-Catechin conjugate targets copper metabolism in neuroblastoma. 17th Advances in Neuroblastoma Research Congress, Cairns, QLD.

Xie J, Mould E, Fordham A, Gifford AJ, Trahair T, MacKenzie KL (2016). Establishment of an orthotopic patient-derived xenograft model of relapsed inflammatory myofibroblastic tumour. Australian Sarcoma Group 2016 Annual Scientific meeting, Kinghorn Cancer Centre, Garvan Institute of Medical Research, Sydney, NSW.

Xue C, Yu D, Gherardi S, Koach J, Milazzo G, Gamble L, Liu B, Russell A, Liu T, Cheung BB, Marshall GM, Perini G, Haber M, Norris MD (2016). MYCN promotes neuroblastoma malignancy by establishing a regulatory circuit with transcription factor AP4. 17th Advances in Neuroblastoma Research Congress, Cairns, QLD.

Xue C, Yu D, Gherardi S, Koach J, Milazzo G, Gamble L, Liu B, Russell A, Liu T, Cheung BB, Marshall GM, Perini G, Haber M, Norris MD (2016). MYCN and TFAP4 promote neuroblastoma malignancy by cooperating in the regulation a subset of target genes involved in cancer cell growth and metastasis. American Association of Cancer Research, New Orleans, LA, USA.

Yi H, Leung H, Holik A, Voli F, Asselin-Labat M, Wang JY (2016). Identification of a novel epigenetic regulator that acts as a tumor suppressor in the development of MLL AML leukemic stem cells. American Society of Hematology Annual Meeting, San Diego, CA, USA.

Young E, Bartolo R, Francis D, Challis J, Brooks I, Petrovich V, Fletcher J, Marshall GM, Norris MD, Haber M, Mechinaud F, Ekert PG (2016). Molecular karyotyping in neuroblastoma – time to stop G-banding. 17th Advances in Neuroblastoma Research Congress, Cairns, QLD.

Invited presentations

Dr Greg ArndtCancer drug discovery – making it personal. Flinders Centre for Innovation in Cancer,

Flinders University, Adelaide, SA.

In vitro sensitivity testing of cancer patient cells for individualised treatment.

Queenstown Molecular Biology - Drug Discovery Satellite Meeting, Nelson, NZ.

Personalised medicine and childhood cancer. Live for Lily Foundation Butterfly Ball, Melbourne, Vic.

Dr Belamy CheungFrom bench-side to bedside, and from generalized medicine to personalised medicine. Plenary speaker, 20th Postgraduate Academic Forum, Guizhou University, China.

Novel small molecule compounds that overcome MYCN-initiated death resistance for the treatment of childhood

neuroblastoma. Plenary Speaker, Shanghai Paediatric Oncology Conference, Xin Hua Hospital, School of Medicine, Shanghai Tiao Tong University, Shangai, China.

Speaker and Chairperson, Advanced Targeted Cancer Therapies, 9th Annual World Cancer Congress, Shanghai, China.

Targeting the MYCN oncogene and the histone chaperone FACT for the treatment of neuroblastoma, Tongji University, Shanghai, China.

Professor Michelle HaberPlenary address, EMBL Australia Postgraduate Symposium, Adelaide, SA.

Inaugural Launch of the UNSW Women in Medicine Group, UNSW, Sydney, NSW.

3rd Symposium on Nuclear Science and Technology for Health, Sydney, NSW. (declined due to other commitments)

Professor Maria Kavallaris Targeting Aggressive Cancers with Nanoparticle Delivery or RNAi. Keynote Speaker, 1st International

Society for Nanomedicine Congress, Korea University, Seoul, Korea.

Nanoparticle Delivery to 3D Cell & Animal Models. Invited Speaker, First Summer School of

International Society for Nanomedicine, Seoul National University Hospital, Seoul, Korea.

Gene silencing in solid tumours: opportunities and challenges Nanomedicine: Prospects for a Brighter Future in Healthcare. Plenary Speaker, International Nanomedicine Conference, Mauritius.

Invited Speaker, 4th Macro Group Meeting, Warwick University, Coventry, UK.

Designing Polymer Therapeutics for the Treatment of Childhood Cancer. Invited Speaker, 11th

International Symposium on Polymer Therapeutics (ISPT), Centro de Investigación Príncipe Felipe, Valencia, Spain.

Microtubules: Multiple Functions in Cancer Drug Resistance, Tumourigenesis and Metastasis. Invited

Seminar, Centro de Investigaciones Biologicas, Madrid University, Madrid, Spain.

Cancer, Microtubules and Nanotherapeutics. Invited Seminar, Centre

de Recherche en Oncologie

biologique et Oncopharmacologie, INSERM, University of Marseille, Marseille, France.

Cancer, Microtubules & Nanomedicine. Invited Speaker, Houston Methodist Research Institute, Houston, USA.

Associate Professor Tao Liu The interplay between histone H3 lysine 4 methyltransferases and N-Myc. Invited Speaker, the 21st

World Congress on Advances in Oncology, Athens, Greece.

Suppressing the expression of a single novel long noncoding RNA leads to neuroblastoma regression

or eradication. Invited Speaker, Mechanisms and Mysteries in Epigenetics Conference, Garvan

Institute of Medical Research, Sydney, NSW.

Professor Richard Lock

Research Skills Seminar for SoMS Honours students: Animal Research and Ethical Considerations, UNSW, Sydney, NSW.

Postgraduate Research Student Induction: Human & animal ethics applications, Faculty of Medicine, UNSW, Sydney, NSW.

Helmholtz Center Munich, Großhadern, as guest of the scientific collaboration netwerk SFB 1243 Genetic and Epigenetic Evolution of Hematopoietic Neoplasms, Munich, Germany.

New agent testing in paediatric acute lymphoblastic leukaemia. Invited Speaker, New Directions in Leukaemia Research Conference, Sunshine Coast, QLD.

Global phosphotyrosine profiling identifies new therapeutic targets in CRLF2-rearranged Ph-like acute lymphoblastic leukaemia Symposium on Signalling Pathways and Systems biology. Invited Speaker, ComBio 2016, Brisbane, QLD.

New agent testing in paediatric leukaemia, XXI. Wilsede Meeting. Wilsede, Germany

Professor Glenn MarshallPlenary Session, American Society for Clinical Oncology, Chicago, USA (invited, but declined)

Plenary Lecture, ANZCHOG Annual Scientific Conference, Cairns, QLD.

Individualising therapy for childhood Leukaemia. Keynote speaker, Ruijin Hospital Seminar, Shanghai

Jiao Tong University, Shanghai, China.

A model for individualising therapy for child cancer. Keynote Speaker, Shanghai Pediatric Oncology

Conference, Xin Hua Hospital, Shanghai Jiao Tong University, Shangai, China.

Dr Jenny WangIdentification of novel mechanisms for self-renewal and drug resistance of malignant stem cells. Invited Speaker, Kids Cancer Alliance Workshop Emerging Technologies, University of Sydney, Sydney, NSW.

Leukaemia stem cells: drug resistance and targeting. Invited Speaker, The 24th Stem Cell Network Workshop – Stem Cells and Cancer, Darlington Centre, University of Sydney, Sydney, NSW.

Targeting leukaemia stem cells by blocking self-renewal signalling. Invited Speaker, New Directions in Leukaemia Research Conference, Sunshine Coast, QLD.

Guest Speakers at Children’s Cancer Institute Seminar Series

Professor James Camakaris Honorary Professorial Fellow, School of Biosciences; Head of the Human Neurogenetics Laboratory Melbourne Brain Centre, The University of Melbourne Melbourne, Vic.

Dr Giuseppe Cirillo Group Leader, Department of Pharmacy, Health and Nutritional Sciences The University of Calabria Calabria, Italy.

Dr Charles Farnsworth Senior Proteomics Applications Scientist, Cell Signalling Technology Danvers, Massachusetts, USA.

Prof Gail Risbridger Group Leader, Prostate Cancer Program, Peter MacCallum Cancer Centre Melbourne, Vic.

Prof Phillip Robinson Head, Cell Signalling Unit, Children’s Medical Research Institute Westmead, NSW.

Professor Brandon Wainwright Director and Group Leader, Institute for Molecular Bioscience; Genomics of Development and Disease Division; Investigator, Centre for Rare Diseases Research University of Queensland Brisbane, QLD.

Guest Speakers at Lowy Seminar SeriesDr Tito Fojo Co-Director, Adrenal Center Herbert Irving Comprehensive Cancer Center, Columbia University Medical Center New York, USA.

Dr Paul Timpson Lab Head - Invasion and Metastasis Garvan Institute of Medical Research Sydney, NSW.

Professor Angelika Eggert Director, Charité Center for Gynecology, Perinatal, Pediatric & Adolescent Medicine with Perinatal Center & Human Genetics, Charité Medical University Berlin, Germany.

Dr Annette Kuenkele Postdoctoral Scientist, Department of Paediatrics, Division of Oncology and Haematology, Charité Medical University Berlin, Germany.

Professor David Huang Laboratory Head, Cancer and Haematology Walter and Eliza Hall Institute of Medical Research Melbourne, Vic.

Associate Professor Ross Dickins Head, Dickins Laboratory, Australian Centre for Blood Diseases Monash University Melbourne, Vic.

Dr Belinda Parker Senior Research Fellow; ARC Future Fellow; Head of the Cancer Microenvironment Laboratory, College of Science, Health and Engineering La Trobe University Melbourne, Vic.

Dr Curtis Harris Chief, Laboratory of Human Carcinogenesis, Center for Cancer Research, National Cancer Institute, National Institutes of Health Bethesda, MD, USA.

Dr Samir Taoudi Laboratory Head, Molecular Medicine Cancer and Haematology Walter and Eliza Hall Institute of Medical Research Melbourne, Vic.

Dr Jack London Informatics Core Director, Kimmel Cancer Center, Thomas Jefferson University, Philadelphia, PA, USA.

ANNUAL REVIEW 2016

Aix-Marseille University Marseille, France

Cancer Research UK Children’s Cancer Group Manchester, UK

Charité Universitätsmedizin Berlin Berlin, Germany

Charles University Childhood Leukaemia Investigation Prague (CLIP) Prague, Czech Republic

Christie Hospital Manchester, UK

Deutsches Krebsforschungszentrum, DKFZ (German Cancer Research Center) Heidelberg, Germany

The Dresden Center OncoRay Dresden, Germany

Ghent University Hospital Center for Medical Genetics Ghent Ghent, Belgium

Erasmus Medical Centre Rotterdam, the Netherlands

European SIOP Neuroblastoma Group Brussels, Belgium

Leibniz Institute for Material Science Leibniz, Germany

EUROPE

Newcastle University Northern Institute for Cancer Research Newcastle upon Tyne, UK

Princess Máxima Center for Pediatric Oncology Utrecht, The Netherlands

Royal College of Surgeons in Ireland Dublin, Ireland

St John of Hope Hospital Spain

University of Bologna Bologna, Italy

University of Catanzaro School of Pharmacy Catanzaro, Italy

University of Dresden Dresden, Germany

University of Dundee Dundee, Scotland

University of Kent Kent, UK

Universita Milano-Bococca Monza, Italy

University of Schleswig-Holstein Berlin-Frankfurt-Münster-Germany Study Group (BFM-G) Kiel, Germany

OUR GLOBAL COLLABORATIONS

We can’t work alone, and we are fortunate to have collaborations with many organisations, clinicians and researchers around the world to help us in our vision of saving the lives of all children with cancer and eliminating their suffering.

Hirosaki University Hirosaki, Japan

Hiroshima University Hiroshima, Japan

Institut Pasteur Korea Seoul, Korea

National University of Singapore Singapore

Tata Memorial Hospital Mumbai, India

University of Nagoya Nagoya, Japan

ASIA

NORTH AMERICA

Children’s Hospital Boston Boston, MA, USA

Children’s Hospital of Philadelphia Philadelphia, PA, USA

Children’s Memorial Hospital Chicago, IL, USA

Children’s Oncology Group Bethesda, MD, USA

Cleveland BioLabs Inc. Buffalo, NY, USA

Emory University Atlanta, GA, USA

Harvard Medical School Boston, MA, USA

Mount Sinai School of Medicine, Black Family Stem Cell Institute New York City, USA

Nationwide Children’s Hospital Columbus, OH, USA

National Institutes of Health, National Cancer Institute, Center for Cancer Research Bethesda, MD, USA

Oncotartis Inc Buffalo, NY, USA

Queen’s Cancer Research Institute Queen’s University Kingston, ON, Canada

Roswell Park Cancer Institute Buffalo, NY, USA

Sanford-Burnham Medical Research Institute San Diego, CA, USA

St Jude Children’s Research Hospital Memphis, TN, USA

Texas Children’s Hospital Houston, TX, USA

Texas Tech University Health Sciences Center Lubbock, TX, USA

Auckland Cancer Society Research Centre Auckland, NZ

Australian and New Zealand Children’s Haematology/Oncology Group (ANZCHOG) Clayton, VIC

Australian National University, John Curtin School of Medical Research Canberra, ACT

Cancer Therapeutics (CTx) Melbourne, VIC

Centre for Cancer Biology Adelaide, SA

Centenary Institute Sydney, NSW

The Children’s Hospital at Westmead Sydney, NSW

Children’s Medical Research Institute Sydney, NSW

Compounds Australia Brisbane, QLD

CSL Limited Parkville, VIC

Flinders University Adelaide, SA

Garvan Institute of Medical Research Sydney, NSW

AUSTRALIA/NEW ZEALAND

John Hunter Children’s Hospital Newcastle, NSW

Kids’ Cancer Alliance Sydney, NSW

Lady Cilento Children’s Hospital Brisbane, QLD

Macquarie University Sydney, NSW

Monash University Melbourne, VIC

Murdoch Children’s Research Institute Melbourne, VIC

Novogen Ltd Sydney, NSW

Peter MacCallum Cancer Centre Melbourne, Vic

Prince Charles Hospital Brisbane, QLD

Princess Margaret Hospital Perth, WA

Prince of Wales Hospital Sydney, NSW

QIMR Berghofer Medical Research Institute Brisbane, QLD

Queensland Children’s Medical Research Institute Brisbane, QLD

Queensland Compound Library Facility Nathan, QLD

Royal Children’s Hospital Melbourne, VIC

Royal Prince Alfred Hospital Sydney, NSW

South Australian Health and Medical Research Institute Adelaide, SA

St Vincent’s Hospital Darlinghurst, NSW

St Vincent’s Institute of Medical Research Melbourne, VIC

Sydney Children’s Hospital, Randwick Randwick, NSW

Telethon Institute for Child Health Research Perth, WA

Therapeutic Innovation Australia Melbourne, VIC

University of Newcastle Newcastle, NSW

University of Queensland Diamantina Institute Brisbane, QLD

University of Sydney Sydney, NSW

UNSW Australia - Adult Cancer Program, Lowy

Cancer Research Centre- Australian Centre for

NanoMedicine- 3D Visualisation Aesthetics Lab,

School of Art and Design- School of Biotechnology and

Biomolecular Sciences (BABS)- School of Chemistry - School of Medical SciencesSydney, NSW

University of Western Australia Perth, WA

Western Sydney University Sydney, NSW

University of Wollongong Wollongong, NSW

Walter and Eliza Hall Institute of Medical Research Parkville, VIC

Westmead Millennium Institute Sydney, NSW

Women’s and Children’s Hospital Adelaide, SA

Instituto Nacional de Câncer, Pediatric Hematology-Oncology Program Rio de Janeiro, RJ, Brazil

SOUTH AMERICA

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For our full annual review, please visit ccia.org.au

Contact us for more information:

Children’s Cancer Institute

ABN 41 072 279 559

Lowy Cancer Research Centre, UNSW Australia

PO Box 81 Randwick NSW 2031

T 1800 685 686 | E [email protected] | www.ccia.org.au

By supporting the vital work of Children’s Cancer Institute you also support us and Isla, and every child with cancer" - Darrin, Isla's dad

“

our journey towards curing childhood cancer...relationships more complex and on a greater scale than...

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