NIH, industry and non-profits join forces to speed validation of disease targets
Goal is to develop new treatments earlier, beginning with Alzheimer's, type 2 diabetes, and autoimmune disorders
The National Institutes of Health, 10 biopharmaceutical companies and several nonprofit organizations today launched an unprecedented partnership to transform the current model for identifying and validating the most promising biological targets of disease for new diagnostics and drug development.
The Accelerating Medicines Partnership (AMP) aims to distinguish biological targets of disease most likely to respond to new therapies and characterize biological indicators of disease, known as biomarkers. Through the Foundation for the NIH (FNIH), AMP partners will invest more than $230 million over five years in the first projects, which focus on Alzheimer's disease, type 2 diabetes, and the autoimmune disorders rheumatoid arthritis and systemic lupus erythematosus (lupus).
A critical and groundbreaking element of the partnership is the agreement that the data and analyses generated will be made publicly available to the broad biomedical community. The three- to five-year, milestone-driven pilot projects in these disease areas could set the stage for broadening AMP to other diseases and conditions.
"Patients and their caregivers are relying on science to find better and faster ways to detect and treat disease and improve their quality of life," said NIH Director Francis S. Collins, M.D., Ph.D. "Currently, we are investing a great deal of money and time in avenues with high failure rates, while patients and their families wait. All sectors of the biomedical enterprise agree that new approaches are sorely needed."
"The good news is that recent dramatic advances in basic research are opening new windows of opportunity for therapeutics," continued Dr. Collins. "But this challenge is beyond the scope of any one of us and it's time to work together in new ways to increase our collective odds of success. We believe this partnership is an important first step and represents the most sweeping effort to date to tackle this vital issue."
As a result of technological revolutions in genomics, imaging, and more, researchers have been able to identify many changes in genes, proteins, and other molecules that predispose to disease and influence disease progression. While researchers have identified thousands of such biological changes that hold promise as biomarkers and drug targets, only a small number have been pursued. Choosing the wrong target can result in failures late in the development process, costing time, money, and ultimately, lives. Currently, developing a drug from early discovery through U.S. Food and Drug Administration approval takes well over a decade and has a failure rate of more than 95 percent. As a consequence, each success costs more than $1 billion.
"The AMP rallies scientific key players of the innovation ecosystem in a more unified way to address one of the key challenges to Biopharma drug discovery and development," said Mikael Dolsten, M.D., Ph.D., President of Worldwide Research and Development at Pfizer. "This type of novel collaboration will leverage the strengths of both industry and NIH to ensure we expedite translation of scientific knowledge into next generation therapies to address the urgent needs of Alzheimer's, diabetes and RA/lupus patients."
AMP has been more than two years in the making, with intense interactions between scientists in the public and private sectors, progressive refinement of the goals, strategy development support from the Boston Consulting Group, and scientific project and partnership management by the FNIH. Through this effort, AMP partners have developed research plans and are sharing costs, expertise, and resources in an integrated governance structure that enables the best informed contributions to science from all participants.
The research highlights for each disease area are:
- Identify biomarkers that can predict clinical outcomes by incorporating an expanded set of biomarkers into four major NIH-funded clinical trials, which include industry support, designed to delay or prevent disease.
- Conduct large-scale, systems biology analyses of human patient brain tissue samples with Alzheimer's disease to validate biological targets that play key roles in disease progression, and increase understanding of molecular networks involved in the disease, to identify new potential therapeutic targets.
Type 2 diabetes
- Build a knowledge portal of DNA sequence, functional genomic and epigenomic information, and clinical data from studies on type 2 diabetes and its heart and kidney complications. The portal will include existing data and new data from studies involving 100,000–150,000 individuals. The rich collection of curated and collated information in this portal will provide an opportunity to identify the most promising therapeutic targets for diabetes from the growing mountain of potentially relevant data.
- Focus on DNA regions that might be critical for the development or progression of type 2 diabetes and search for natural variations in targeted populations that might predict the likelihood of success of drug development aimed at these targets.
Rheumatoid arthritis and lupus
- Collect and analyze tissue and blood samples from people with rheumatoid arthritis and lupus to pinpoint biological changes at the single cell level, to allow comparisons across the diseases and provide insights into key aspects of the disease process.
- Identify differences between rheumatoid arthritis patients who respond to current therapies and those who do not, and provide a better systems-level understanding of disease mechanisms in RA and lupus.
Highly collaborative steering committees with representation from public- and private-sector partners will be established for each disease area to oversee the research plans. The steering committees will be managed by FNIH under the direction of an AMP executive committee comprised of leaders from NIH, industry, the FDA, and patient advocacy organizations.
More information about the program and the disease research plans can be found here: http://www.nih.gov/amp.
About the Foundation for the National Institutes of Health (FNIH): FNIH creates and manages alliances with public and private institutions in support of the mission of the NIH, the world's premier medical research agency. The Foundation, also known as the FNIH, works with its partners to accelerate key issues of scientific study and strategies against diseases and health concerns in the United States and across the globe. The FNIH organizes and administers research projects; supports education and training of new researchers; organizes educational events and symposia; and administers a series of endowments supporting a wide range of health issues. Established by Congress in 1996, the FNIH is a not-for-profit 501(c)(3) charitable organization. For additional information about the FNIH, please visit http://www.fnih.org .
The Office of the Director, the central office at NIH, is responsible for setting policy for NIH, which includes 27 Institutes and Centers. This involves planning, managing, and coordinating the programs and activities of all NIH components. The Office of the Director also includes program offices which are responsible for stimulating specific areas of research throughout NIH. Additional information is available at http://www.nih.gov/icd/od.
About the National Institutes of Health (NIH): NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit www.nih.gov.
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Johnson & Johnson
American Diabetes Association
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Foundation for the NIH
Geoffrey Beene Foundation
Rheumatology Research Foundation
What others are saying about the Accelerating Medicines Partnership:
National Institute of Diabetes and Digestive and Kidney Diseases:
"The knowledge portal we're developing in partnership with industry has the potential to make the promise of genomics a reality when it comes to new therapies," said Griffin Rodgers, M.D., M.A.C.P., National Institute of Diabetes and Digestive and Kidney Diseases.
National Institute of Arthritis and Musculoskeletal and Skin Diseases:
"Collectively, autoimmune diseases have a significant impact on millions of Americans," said Stephen I. Katz, M.D., Ph.D., Director of the National Institute of Arthritis and Musculoskeletal and Skin Diseases. "This is the right moment to leverage new scientific knowledge and emerging technologies to improve targeted therapies for these patients."
National Institute on Aging:
"The NIA is pleased that this partnership will facilitate both basic studies to identify new targets to pursue for Alzheimer's disease therapies, as well as studies to add novel biomarkers to Alzheimer's clinical trials to assess response to treatments," said Richard J. Hodes, M.D., Director, National Institute on Aging. Such efforts, we believe, should accelerate the development of interventions that can make a real difference for people with this devastating disease."
"AbbVie is committed to developing advanced therapies to address unmet medical need in Alzheimer's and autoimmune diseases and we look forward to sharing our expertise in these areas," said Jim Sullivan, Ph.D., Vice President, Pharmaceutical Discovery, AbbVie. "This unique collaboration between the public and private sectors will aid discovery in understanding the molecular pathways driving disease progression and improve the ability to treat these serious health concerns."
"Through this partnership, we expect to identify new strategies for attacking some of the most challenging and costly diseases facing society, such as Alzheimer's disease," said Douglas E. Williams, Ph.D., Biogen Idec's Executive Vice President of Research and Development. "It's an exciting opportunity for us to collaborate across government, patient advocacy and industry to better understand the biology of disease and more effectively test our treatment hypotheses, helping us to get better medicines to patients faster."
"Today's announcement recognizes the importance of creative collaborations in R&D that will speed the development of new medicines for serious diseases," said Francis Cuss, Chief Scientific Officer, Bristol-Myers Squibb. "By combining the NIH's leadership in scientific research and the biopharmaceutical industry's expertise in translational research, we see this collaboration as a way to expand our individual research efforts to achieve a broader understanding of some of the most challenging disease areas, with the goal of getting improved treatment options to patients faster."
"This initiative allows industry's best and brightest scientific minds in industry and academia the opportunity to solve some of the mysteries behind society's currently most devastating diseases, with the goal of delivering medicines to patients who are waiting," said Jan Lundberg, Ph.D., Chief Scientific Officer at Eli Lilly and Company.
"We have learned from experience that diseases such as Alzheimer's are too difficult and complicated to be solved by any one organization," said Lon Cardon, Ph.D., Senior Vice President of Alternative Discovery & Development at GSK. "This is the type of global health issue that needs the resources and scientific know-how that pharma, government, academia, and non-profits can harness together under the proven success of NIH coordination. The Accelerating Medicines Program allows each of us to play to our strengths with the combined goal of more efficiently and effectively discovering new medicines for patients."
Janssen Research and Development (part of the pharmaceutical companies of Johnson & Johnson):
"We believe that working collaboratively is critical to speeding the development of cures that benefit people all around the world," said Bill Hait, M.D., Ph.D., Global Head, Janssen Research and Development. "We participate in a number of initiatives with industry, government, and NGOs to improve the ways in which new drugs are discovered, clinical trials are conducted, clinical data is shared, and healthcare is delivered. We know the model works. That's why we're very pleased to join this broad-based partnership to accelerate the development of new medicines by transforming the current model for identifying and validating the most promising biological targets of disease."
"Our most critical health challenges require new, innovative ways to develop medicines and vaccines.
Collaborations such as this, that exchange data, share insights and generate knowledge will be important to unravelling the mysteries of the diseases that cause suffering for individuals and are a burden to our society," said Rupert Vessey, BM Bch, DPhil, Senior Vice President, Early Development and Discovery Sciences, Merck. "Merck is pleased to contribute our scientific expertise to help advance the AMP."
"No longer a collection of acute illnesses, the medical landscape now features chronic diseases, many of which are not well understood. Its ignorance is expensive. It costs society in lives, dollars, and human contributions to the therapeutic development process. This knowledge gap must be approached with a strong sense of urgency," says Dr. Elias Zerhouni, President, Global R&D at Sanofi. "We have to join forces to better understand the complex puzzle of these diseases and accelerate our capacity to bring new therapies to patients."
"The AMP is an exciting collaboration that will harness the resources, knowledge and will of the NIH and the pharmaceutical industry to discover new insights and new approaches in three important disease areas," said Jonathan Zalevsky, Ph.D., Head of the Inflammation Drug Discovery Unit within Takeda's Pharmaceutical Research Division. "Takeda is delighted to participate in this important initiative, which we believe will bring tremendous benefit to patients with unmet medical needs."
"With the Alzheimer's epidemic upon us, it is critical that we not only discover new Alzheimer's diagnostics and treatments but also reduce the time and cost of developing them," said Harry Johns, President and CEO of the Alzheimer's Association. "As a member organization of the AMP steering committee, we look forward to working with our industry partners to further develop and execute the AMP for complex diseases, such as Alzheimer's, where the desperation for new drugs is palpable. The Alzheimer's Association is the largest non-profit funder of Alzheimer's research and has been part of every major Alzheimer's research advancement over the past 30 years; we look forward to AMP helping to create future breakthroughs."
American Diabetes Association:
"The Accelerating Medicines Partnership provides an important opportunity for all who are working to improve the lives of people with diabetes," said Larry Hausner, MBA, Chief Executive Officer, American Diabetes Association. "The American Diabetes Association looks forward to collaborating with NIH, biopharmaceutical companies and other health-focused nonprofit organizations so that we may better leverage the scientific expertise and resources each of us bring to the important mission of advancing the treatment of type 2 diabetes. Our shared goal is to increase the number of new diagnostics and therapies for patients with type 2 diabetes and to reduce the time and cost of developing them, and we are proud to take part in this endeavor."
Foundation for the NIH:
"Significant strides against diseases require bold initiatives that venture into uncharted territory and confront challenging biological questions," said Maria Freire, Ph.D., President, Foundation for the NIH. "I am delighted that the Foundation for the NIH has the opportunity to be part of this trailblazing effort, managing and coordinating complex scientific alliances that can transform the speed and precision with which new medicines are developed."
Geoffrey Beene Foundation Alzheimer's Initiative:
"Alzheimer's is the dark side of longevity," said Meryl Comer, President and CEO, Geoffrey Beene Foundation Alzheimer's Initiative. "The sheer numbers of our generation who will live long enough to get this disease demands this focus on new diagnostics and therapies. We cannot afford the consequences of burdening the next generation with the economic and societal costs of Alzheimer's disease. New diagnostics and therapies for patients are our only way out."
Lupus Foundation of America:
"We are pleased to be a partner with this important collaborative effort which will bring greater focus and coordination to help solve the cruel mystery of lupus and improve the quality of life for the millions of people worldwide living with this misunderstood and unpredictable disease," said Sandra C. Raymond, President & CEO of the Lupus Foundation of America. "In recent years there have been a number of scientific findings and developments that have placed us at the brink of breakthrough, and greater collaboration among all stakeholders is needed to maximize comprehensive efforts to bring lupus under control."
"This exciting collaborative partnership not only holds promise for the identification of more validated targets for drug discovery and development in three disease states but may serve as a model for future progress in this most critical hurdle in finding new medicines for patients," said William W. Chin, M.D., Executive Vice President of Scientific and Regulatory Affairs, Pharmaceutical Research and Manufacturers of America (PhRMA).
Rheumatology Research Foundation:
"The Rheumatology Research Foundation is excited to be part of the Accelerating Medicines Partnership," said Steven Echard, IOM, CAE, Executive Director, Rheumatology Research Foundation. "The new alliance furthers our mission to advance research and training to improve the health of people with rheumatic diseases."
"This exciting new public/private initiative demonstrates the unity and commitment of the leadership of our public, private and non-profit sectors to achieve our goal of preventing and effectively treating Alzheimer's by 2025," said George Vradenburg, Chairman and Co-Founder, USAgainstAlzheimer's.
NIH News Media Branch