Evoluzione dell’iniziativa di

programmazione congiunta per

le malattie neurodegenerative

Adriana Maggi

Vice-chair JPND

……since 2008

JPND a pilot initiative for Joint

Programming in Europe

A major societal challenge for the coming years

Joint programming on

neurodegeneration – why?

30% of healthy elderly subjects already have

Alzheimer pathology

Cognitively normal subject aged 72 years

with Alzheimer pathology (red)

To mobilise the best researchers from all fields, to develop the sharing of best

practices and know how

Taking the disease

into account in all its dimensions

• A scientific challenge • Genomics

• Experimental models

• Cell biology

• …

• A medical challenge • Clinical research

• Translational research

• Therapeutics research

• …..

• An ethical and social challenge

Organisation

• Management Board • 25 countries represented

• Mandated to act

• Chair Prof Philippe Amouyel (France)

• Executive Board • Vice-Chair Prof Adriana Maggi (Italy)

• Enda Connolly (Ireland)

• Edvard Beem (The Netherlands)

• Mogens Horder (Denmark)

• Scientific Advisory Board • 15 Members (5 in each domain)

• Chosen for scientific excellence

2011- 12 – Activities

The delivery of the SRA

• 1. Mapping exercise of National and European programmes

• 2. Strategic prioritisation

• three thematic workshops (basic/clinical/healthcare)

• Stakeholder discussions

• fourth workshop to integrate outputs, incorporate stakeholder

perspectives

• 3. Consultation exercise (website publication)

• 4. Delivery of the SRA, taking into account

• Outputs from scientific workshops

• Consultation

• Discussion at MB (to incorporate funder, political & policy

considerations)

JPND mapping

€1.62bn investment – 374 programmes, 1766 smaller projects

254 bioresources captured

Survey Section Entries Which represents:

Organisational Details 69 Organisations from 20 countries

Smaller Investments 1756 €0.27 billion - 59 agencies from 18 countries

+ EC inc. ERC covering 1756 projects

Major Programmes 368 €1.3 billion - 39 funding agencies

(12 countries + EC inc. ERC)

Centres of Excellence 111 14 countries

Research Networks 32 7 countries + Europe-wide

Population Cohorts 72 15 countries

Case Control Studies 22 9 countries

Disease Registries 21 9 countries + Europe-wide

DNA, Tissue & Cell line Banks 81 15 countries

Animal Models 37 10 countries

Bio/Neuro Infrastructures 21 9 countries + Europe-wide

ND research spend by disease

area (annualised)

HD

3.3%

SCA

0.3%

SMA

0.9%

ND in general 30.4%

AD and other dementias 45.3%

MND

3.7% Prion

4.5%

PD

11.5%

Implementation- test case

1. Launched by JPND - Centres of Excellence Network in

Neurodegeneration Research (COEN) on Feb 2011

Participating Countries: Germany, UK, Ireland, Canada, Belgium,

Italy

Topic : Biomarkers, Imaging, Animal models

Commitment: for 4 M€

8 Projects retained on Sept 2nd 2011

2. Launched by JPND on May 2011 Participating Countries: 21

Topic: Neurodegenerative Diseases - a call for European research

projects for the optimisation of biomarkers and harmonisation of their

use between clinical centres”

Commitment for 16 M€

4 projects retained on 7th Dec 2011

March 2012 Launch of the JPND

Research Strategic Agenda

Available on JPND Website

• http://www.neurodegenerationresearch.eu

• http://www.jpnd.eu

• Full Research Strategy

• Executive Summary in 13 Languages

• Reports summarising responses received from consultations

Further knowledge is needed regarding:

Causes of specific ND

Factors that determine people’s risk and resilience

Triggering events leading to illness

Ageing process

Characterise ‘at-risk’ populations;

genetic, epigenetic and environmental risk factors

Identify environmental and behavioural modulators

Scientific Priorities: Origins of neurodegenerative

disease

Scientific Priorities: Disease mechanisms and models

• Increase understanding of underlying

disease mechanisms to:

• Underpin the development of new diagnostic

and therapeutic approaches

• Identify appropriate time-windows for

intervention

• Establish novel cell-based and animal models

• Elucidate the biological and environmental

basis of behavioural and psychological

symptoms in ND

• Refine and update current diagnostic criteria

• Better define the various forms and subtypes of ND, including the

stages before clinical symptoms emerge

• Provide new/improved diagnostic tools for earlier detection

• Establish new biomarkers to:

• Provide links between human and animal-based studies

• Provide measures of disease progression, prognosis and treatment

effects

• Standardise and harmonise tools and assessments to ensure

comparability of results and support cross-centre studies

Scientific Priorities: Disease definitions and diagnosis

Scientific Priorities: Treatments and prevention

• Promoting connectivity between studies in animal/cell models and

patients

• Improve selection (or stratification) of subjects entering clinical trials

• Further develop psychosocial interventions, paying attention to the

promotion of social inclusion and carer involvement

• Establish cohorts of patients with preclinical ND to provide a platform

for the future testing of interventions to prevent or slow disease

• Pursue longer-term approaches that promote regenerative strategies

and develop novel systems for the delivery and targeting of

therapeutics to specific sites in the brain and nervous system

Scientific Priorities: Healthcare and social care

• Evaluate equity of access to, and cost-effectiveness

of, pathways to diagnosis, treatment, care & support

• Identify factors impacting on disability and health-

related QoL in ND, including comorbidity, nutrition,

and interaction with family, carers, environment, and

health/social care systems

• Relate short-term studies to real-world settings

• Improve outcome measures to better reflect patient

& carer perspectives

• Addressing specific burden of ND in strategies for

assisted living

• Research in palliative and end-of-life care

Enabling Activities

• Knowing our research capability • European and National ND research activity mapped

• Supportive infrastructure and platforms • integration and harmonisation of data and materials

• standardisation of guidelines, methods and tools for data collection

and analysis

• review policy frameworks to facilitate research across the full range

of healthcare structures

• Working in partnership with industry • Promote connection between and across academic and

commercial domains

• Encourage data and resource exchange

Enabling Activities

• Working with regulatory organisations • Effective translation of research through to patient benefit

• Ensure that regulation is easily understood + proportionate to risk

• Promote regulatory support networks

• International partnership beyond Europe • Unmet clinical need and societal impact of ND is a global issue

• Cooperation should be strategic and offer clear added value

• Capacity building • Networks across and between different disciplines

• Methodological hubs

Enabling Activities

• Education and Training • advice given to patients should be based upon;

• a good understanding of the disorder

• the patient needs characteristic of these conditions

• the available evidence-based options for treatment

• tailored clinical and research education and training programmes

• public health messaging

• Connection to policy makers • a framework to highlight issues for national policy consideration, and

promote compatibility between countries

• address translational gaps in policy

• Communication and outreach • promote translation into policy and practice

• ensure stakeholder communities are informed

SRA Implementation

1) Create List of priorities from SRA proposals

2) Organise thematic working groups

3) Create List of opportunities

4) Support Implementation

Next Challenges

• Implementation of the SRA

• Developing national SRA

• Funding commitment based on a 5 year

period

• Leverage effect

• Based on existing mechanisms

• Scale-up funding commitment

SRA implementation 2012 –

Topics proposed MB Meeting London May 2012

• Genetic, epigenetic & environmental risk factors

• Animal models for the identification of mechanisms underlying

ND

• Cross-disease pathway analysis

• Preventive strategies

• Evaluating healthcare policy strategies and interventions

• Systematic review of «pathways to care»

• Palliative and end-of life care

Task force

Task force

Future call

Funding forecast

• Ireland € 2.5 M

• Turkey € 1 M

• Germany € 3 M

• Norway € 1.8 M

• UK € 1 M

• France € 5 M

• Finland € 1 M

• Belgium € 1,5 M

• Spain € 0,5-1 M

• Croatia € 1 M

• Luxenbourg € 0,5 M

• Denmark € 0,5-1 M Total at June 2012 € 20 M

Austria - Italy - Netherlands - Poland -

Portugal- Sweden - Slovenia

...e l’ITALIA?

ITALIA IN SENO AL JPND

• Acquisizione della vicepresidenza

• Partecipazione SAB (Stefano Cappa)

• Partecipazione attiva ai workshop per la

definizione della agenda strategica

• Organizzazione workshop JPND-industrie

• Attiva partecipazione alle task-forces per

implementazione agenda strategica

ESERCIZIO DI MAPPATURA

Mapping of Italian research excellence in Neurodegenerative Diseases

Riunione APRE

Roma 2 marzo 2011

creazione di una lista di laboratori attivi nel settore delle malattie

neurodegenerative e dei progetti attualmente finanziati

sensibilizzazione delle principali società scientifiche: SIN –

SINDEM - SIF- SINS

definizione di una lista di priorità per la ricerca italiana

Ricerca Nel Settore Delle Malattie Neurodegenerative In Italia Verso La

Roadmap Italiana - Incontro con le Industrie interessate

MIUR Roma 13 dicembre 2011

Risultato della prima call: hanno partecipato 6 gruppi italiani come capo-fila o

come membri:

2 soli gruppi di italiani sono rientrati nel novero dei vincitori

a.) Mondino di Pavia: membro del gruppo di ricerca che ha presentato il

progetto di ricerca che ha avuto il massimo dei risultati

b.) Fatebenefratelli di Brescia

Risultato della seconda call: hanno partecipato 8 gruppi italiani di cui 6 hanno

ottenuto il finanziamento

Risultati partecipazione

italiana alle prime 2 call

I FINANZIAMENTI

MINISTERO DELLA SALUTE

MIUR

LE INDUSTRIE INTERESSATE

I Paesi Europei affrontano

Il problema delle mallatie neurodegenerative

La FRANCIA : Fondazione per l’Alzheimer

La GERMANIA: nuovo istituto di ricerca sul SN a Bonn

La SPAGNA: Istituto di ricerca e cura Regina Sophia

La GRAN BRETAGNA: interventi alla Camera dei Lord e nuovi finanziamenti

L’OLANDA: un nuovo programma di ricerca dedicato

Keep up to date

• Visit the JPND website:

• http://www.neurodegenerationresearch.eu

• http://www.jpnd.eu

• Search our Mapping Database

• Sign up to the JPND News Feed

• Follow us on Twitter: @JPNDEurope

• E-mail us: secretariat@jpnd.eu