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Vol. LXVI, No. 5
February 28, 2014
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NIH, Industry and Non-Profits Partner to Speed Validation of Disease Targets

On the front page...

NIH director Dr. Francis Collins unveils AMP at National Press Club on Feb. 4.
NIH director Dr. Francis Collins unveils AMP at National Press Club on Feb. 4.
NIH, 10 biopharmaceutical companies and several nonprofit organizations launched an unprecedented partnership on Feb. 4 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, AMP partners will invest more than $230 million over 5 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).

Continued...

“AMP is tackling a major challenge in drug development, on an unprecedented level,” said NIH director Dr. Francis Collins. “As you know, amp is a unit of current, and we’re all amped up this morning about the promise of this new consortium.”

In addition to Collins (l), other speakers assemble to discuss AMP.

In addition to Collins (l), other speakers assemble to discuss AMP. “What we would like to have is a precise navigation system—a GPS for human disease,” said Dr. Mikael Dolsten (c), president of worldwide research and development at Pfizer. Lupus patient Shanelle Gabriel, a performance artist, said, “To see everyone in here teaming up up together to try to find better ways to help people like me…it means a lot.”

Unveiling AMP at the National Press Club downtown, Collins continued, “It means we are going to try to increase the odds of picking the right targets to go after for the next generation of drug development…This landmark partnership will transform the way we develop medicines. And I think no one will be happier to hear that than patients and their loved ones, for whom we really are dedicated in this effort.”

With Collins for the historic partnership announcement is former NIH’er Dr. Maria Freire, president of FNIH. The foundation is one of several non-profit partners involved with AMP.

With Collins for the historic partnership announcement is former NIH’er Dr. Maria Freire, president of FNIH. The foundation is one of several non-profit partners involved with AMP.

Photos: Bill Branson

A critical and groundbreaking element of the partnership is the agreement that the data and analyses generated will be made publicly available to the biomedical community. The 3- to 5-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 Collins. “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.

Cynthia Leoro (l), a translator and type 2 diabetes patient on hand at the AMP event, speaks with Dr. Satwant Narula, vice president of immunoscience and fibrotic diseases, discovery science and biologics at Bristol-Myers Squibb, one of the AMP partners.
Cynthia Leoro (l), a translator and type 2 diabetes patient on hand at the AMP event, speaks with Dr. Satwant Narula, vice president of immunoscience and fibrotic diseases, discovery science and biologics at Bristol-Myers Squibb, one of the AMP partners.

“The good news is that recent dramatic advances in basic research are opening new windows of opportunity for therapeutics,” he continued. “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.”

As a result of 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, Collins explained. While researchers have identified thousands of such 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.

Another NIH alum, Dr. Gary Nabel (r), now chief scientific officer for global research and development at Sanofi, was present at the briefing.
Another NIH alum, Dr. Gary Nabel (r), now chief scientific officer for global research and development at Sanofi, was present at the briefing.

Currently, developing a drug from early discovery through 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.

AMP has been more than 2 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 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.

More information about the program can be found at www.nih.gov/amp.


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