Thomas R. Insel, M.D., Acting Director
IOM Forum on Drug Discovery, Development, and Translation
June 4, 2012
Associated Press, May 21, 2003: “Democratic
presidential candidate Joe Lieberman on Wednesday
called for a $150 billion, 10-year federal initiative to
quickly bring cures for diseases to the marketplace. . . “
S.2104, the American Center for Cures Act of 2005 (introduced 12/14/2005) Stand alone bill for a Center within NIH; authorization of
$5,000,000,000
S.2988, the Accelerating Cures Act of 2008 (introduced 5/7/2008)
Biotechs Starved by Financial Collapse
Different Set of Reviewers (i.e. VC and Patients)
Broaden Eligible Grantees
“X Prize” Follow-on to ARRA
“DARPA-like Authorities” (Sen. Stevens [R-AK])
Specter Bill Would:
create an entity outside the NIH
authorize use of interagency agreements with the Center
for Scientific Review performing peer review
prescribe the expertise necessary for members of the initial
review groups
manage the program by a high level Board
increase the authorization for NIH to $40B
authorize $1 billion in FY10-11
Established in NIH Office of the Director
Authorized budget for FY 2010: $500 M Specifically appropriated “new money”
Creates New Award Mechanisms Cures Acceleration Partnership Awards: up to $15 M,
requiring matching funds ($1 for every $3 awarded by NIH) Cures Acceleration Grants: $15 M per award Cures Acceleration Flexible Research Awards: DARPA-like
research authority, up to 20 percent of CAN
CAN Review Board 24 member advisory board; Patient Advocates, VC Advise NIH Director about barriers to successful translation
of basic science into clinical applications
April 16, 2010
Honorable Tom Harkin
Chairman
Subcommittee on Labor-HHS-Education
Committee on Appropriations
United States Senate
Washington, DC 20510
Honorable Thad Cochran
Ranking Member
Subcommittee on Labor-HHS-Education
Committee on Appropriations
United States Senate
Washington, DC 20510
Dear Chairman Harkin and Ranking Member Cochran:
We respectfully request that the Senate Appropriations Subcommittee on Labor, Health and Human Services, Education, and Related Agencies include funding for the newly-enacted Cures Acceleration Network in the Fiscal Year (FY) 2011 Labor-HHS appropriations bill.
Abigail Alliance for Better Access to Developmental Drugs
AIDS Institute
Alexion Pharmaceuticals
Alliance for Aging Research
Alliance for Regenerative Medicine
Alpha-1 Association
Alpha-1 Foundation
ALS Association
ALS Therapy Development Institute
Alseres
Alzheimer's Association
American Institute for Medical and Biological Engineering
American Parkinson Disease Association
Association for Clinical Research Training
Association for Patient-Oriented Research
Association of Academic Health Science Libraries
AVAC: Global Advocacy for HIV Prevention
Batten Disease Support and Research Association
Benign Essential Blepharospasm Research Foundation
BIO Ventures for Global Health
Biotechnology Industry Organization
Californians4cures
CANN - Community Access National Network
Celiac Disease Center at Columbia University
Ceregene
Citizens United for Research in Epilepsy
Clinical Research Forum
Coalition of Heritable Disorders of Connective Tissue
Colon Cancer Alliance
COPD Foundation
Council for American Medical Innovation
Cutaneous Lymphoma Foundation
Cystic Fibrosis Foundation
Digestive Disease National Coalition
Dystonia Advocacy Network
Dystonia Medical Research Foundation
Easter Seals
FasterCures
Foundation for Sarcoidosis Research
Friends of Cancer Research
Genetic Alliance
Global Healthy Living Foundation Harlem United Community AIDS Center, Inc Huntington’s Disease Society of America Infectious Diseases Society of America International AIDS Vaccine Initiative International Foundation for Functional Gastrointestinal Disorders International Myeloma Foundation Interstitial Cystitis Association Juvenile Diabetes Research Foundation Kakkis EveryLife Foundation Leukemia & Lymphoma Society Lung Cancer Alliance Medical Library Association Michael J. Fox Foundation for Parkinson's Research Muscular Dystrophy
Association National Disease Research Interchange National Health Council National Minority AIDS Council National MPS Society National Multiple Sclerosis Society National Parkinson Foundation National PKU Alliance National Tay-Sachs & Allied Diseases Association National Venture Capital Association NephCure Foundation New York Stem Cell Foundation Parent Project Muscular Dystrophy Parkinson Alliance and Parkinson’s Unity Walk Parkinson's Action Network Parkinson's Disease Foundation PKD Foundation PXE International Rett Syndrome Research Trust Scleroderma Foundation Society for Clinical and Translational Science Spinal Muscular Atrophy Foundation SRI International Student Society for Stem Cell Research The Christopher and Dana Reeve Foundation Tuberous Sclerosis Alliance
Malcolm Gladwell, Annals of Innovation, “The Treatment,”
The New Yorker, May 17, 2010, p. 69
ABSTRACT: ANNALS OF INNOVATION about the process of developing cancer drugs. In the world of cancer research, there is something called a Kaplan-Meier curve, which tracks the survival of patients in the trial of an experimental drug. In its simplest form, it consists of two lines. The first follows the patients in the “control arm,” the second the patients in the “treatment arm.” In most cases, those two lines are virtually identical. But every now and again the patients in the treatment arm will live longer than the patients in the control arm, and the two lines on the Kaplan-Meier will diverge. Tells about Safi Bahcall, the C.E.O. of Synta Pharmaceuticals, a small biotechnology company located outside Boston. In the summer of 2006, Synta had two compounds in development. One was a cancer drug called elesclomol. The other was an immune modulator called apilimod. Phase 2 testing of elesclomol resulted in surprising data indicating that the compound might be an effective treatment for metastatic melanoma, a form of skin cancer that was notoriously resistant to treatment. Describes Emil Freireich's research into drugs to treat acute lymphoblastic leukemia (ALL) in the nineteen-fifties and sixties. Working with a group that included Tom Frei, of the National Cancer Institute, and James Holland, of the Roswell Park Cancer Institute, Freireich started treating ALL patients with a combination of drugs. They discovered that they didn't need a magic bullet—a superdrug that could stop all cancer in its tracks. A drug that worked a little bit could be combined with another that worked a little bit and another that worked a little bit, and the combination could turn out to be spectacular. Tells about Bahcall's partner, a cell biologist named Lan Bo Chen. Drug hunters like Chen fall into one of two broad schools: “rational design,” in which the scientist starts with the disease and works backward, or “mass screening,” in which a scientist begins with a drug candidate and searches for diseases it might attack. Chen belonged to the latter school. Describes the drug-screening process Chen went through to discover elesclomol. Discusses the complexities of drug testing: there are a variety of ways in which a drug can be applied and therefore, a variety of ways in which it can be tested against a variety of different cancers in different stages. Tells about the Phase 2 trial that produced the encouraging elesclomol results for melanoma.
From 1996 to 1999 The U.S. food
and Drug Administration approved 157
new drugs. In the comparable period a
decade later—that is, from 2006 to
2009—the agency approved 74. Not
among them were any cures, or even
meaningfully effective treatments, for
Alzheimer's disease, lung or
pancreatic cancer, Parkinson's
disease, Huntington's disease, or a
host of other afflictions that destroy
lives.
Breakthroughs and Breakdown
Drugs that changed lives, for better or
worse
Desperately Seeking Cures How the road from promising scientific breakthrough to real-world remedy has become all but a dead end.
By Sharon Begley and Mary Carmichael |
NEWSWEEK
Published May 14, 2010
From the magazine issue dated May 31, 2010
In a first-of-a-kind collaboration between academia and industry,
Pfizer Inc. will give scientists at Washington University School of
Medicine in St. Louis unprecedented access to information regarding
more than 500 pharmaceuticals and pharmaceutical candidates in a
partnership that focuses on discovering new uses for existing
compounds.
Under the five-year, $22.5 million agreement announced Monday,
May 17, Pfizer will provide access for Washington University
scientists to view extensive research data on a large array of Pfizer
pharmaceutical candidates that are or were in clinical testing.
Washington University, Pfizer announce groundbreaking
research collaboration
As part of an innovative collaboration, scientists with Pfizer
Inc.'s Indications Discovery Unit are moving to the Center of
Research Technology and Entrepreneurial Expertise
(CORTEX-pictured above), adjacent to Washington
University School of Medicine.
May 17, 2010 By: Caroline Arbanas
“To catalyze the generation of innovative methods
and technologies that will enhance the development,
testing, and implementation of diagnostics and
therapeutics across a wide range of human diseases
and conditions.”
0
500
1000
1500
2000
2500
3000
3500
4000
4500
5000
Disorders with Known Molecular Basis
Source: Online Mendelian Inheritance in Man, Morbid Anatomy of the Human Genome
250 with
therapy
Past decade: 17-36 NMEs/year
Drug Discovery
10,000 Compound
s
1 Approve
d
Drug
Preclinical
Testing Clinical Trials FDA
Review Clinic
1.5
Years
6 Years 6.5 Years
250
Compounds
5
Compounds
82% fail in Phase II
Basic Laboratory
Research
Translational
Research Population
Research
Clinical
Research
Improved
Public
Health
Standard Model The Way it Should Work
Basic Laboratory
Research
Patient-oriented
Clinical Research
Population-based Clinical
Research Clinical Trials
Improved
Public Health
Clinical and Translational Science Activities
Clinical and Translational Science Awards
Rare Diseases Research and Therapeutics
Therapeutics for Rare and Neglected Diseases
Office of Rare Diseases Research
Re-engineering Translational Sciences
Tox-21 and Tissue Chip Programs
Repurposing
New Therapeutic Uses for Existing Drugs
Cures Acceleration Network (CAN)
Clinical and Translational Science Awards (CTSAs)
Support a national consortium of medical research institutions
Work together to improve the way clinical and translational research is conducted nationwide
Aim to accelerate the research translation process
Therapeutics for Rare and Neglected
Diseased (TRND) Program
Designed to re-engineer the development of new drugs for
rare and neglected diseases
Specifically intended to stimulate research collaborations for
drug discovery and development between NIH and:
Academic scientists
Nonprofit organizations
Pharmaceutical and biotechnology companies
Tox21: Toxicology in the 21st Century
A collaboration with:
NIH’s National Institute of Environmental Health Sciences
U.S. Environmental Protection Agency
U.S. Food and Drug Administration
Designed to screen a collection of 10,000 compounds composed of environmental chemicals and drugs approved for use
Looks for compounds’ potential to disrupt biological pathways that may be toxic
Tissue Chip for Drug Screening: NIH‒FDA‒DARPA
Collaboration
Aims to develop a tissue chip that mimics human
physiology to screen for safe, effective drugs
Liver, heart, lung, other cell types
Designed for multiple types of readouts
NIH and Defense Advanced Research Projects Agency
(DARPA) contribute $70M over 5 years; FDA provides
guidance
Seeking best ideas in engineering, biology, toxicology
Clinical and Translational Science Activities
Clinical and Translational Science Awards
Rare Diseases Research and Therapeutics
Therapeutics for Rare and Neglected Diseases
Office of Rare Diseases Research
Re-engineering Translational Sciences
Tox-21 and Tissue Chip Programs
Repurposing
New Therapeutic Uses for Existing Drugs
Cures Acceleration Network (CAN)
Created to advance development of “high need cures”
Reduces barriers to translation in areas the private
sector is less likely to pursue
Funded via:
Grant awards with or without partnership
Flexible Research Awards: DARPA-like authority
Not to exceed 20 percent of total appropriated funds
per fiscal year
FY 2012 budget: $10 million (FY 2013 PB: $50 million)
24 members appointed by Secretary DHHS
At least one member eminent in the following areas:
basic research, medicine, biopharmaceuticals, discovery
and delivery of medical products, bioinformatics and
gene therapy, medical instrumentation, regulatory review
and approval
At least 4 members recognized as leaders in venture
capital or private equity investing
At least 8 individuals representing disease advocacy
organizations
“$10M for CAN Board and related activities”
CAN Board encouraged to create measurable
outcomes to track success
IOM workgroup “to review, evaluate, and identify
issues related to CAN authority” and report to CAN
Board to help it identify ways to accelerate cures
IOM should include NIH, FDA, AHRQ, CDC, PTO
Patent authority, marketability, use of high
throughput analysis, regulatory timelines, and cost
structure issues related to CAN
How will CAN have greatest impact?
What tools, methods, and approaches can accelerate translational science? (FDA, CDC, AHRQ)
Best use of CAN matching and flexible research authorities? (DARPA, DTRA, BARDA)
How will CAN assist public-private partnerships? (Pharma, Biotech, VC, advocacy)
How will CAN interact with regulatory science initiative? (FDA)