Ramping up its efforts in drug discovery, the National Institutes of Health (NIH) today unveiled  what it called an unprecedented $230 million, 5-year partnership with 10 drug companies aimed at finding new treatments for Alzheimer’s disease, diabetes, rheumatoid arthritis, and lupus.
In a room at the Washington, D.C., National Press Club packed with representatives from industry, patient groups, and federal officials, NIH Director Francis Collins described the Accelerating Medicines Partnership (AMP). The goal is to cut down on the more than 95% failure rate for drug candidates. As a result, it now takes some 10 years and more than $1 billion to develop a successful drug. By combining forces, companies and academics hope to cut costs and speed things up by sorting through a wealth of new genomic and molecular data on disease biology to find the most promising new drug targets. “This is a job too big for any single group,” Collins said.
The 10 companies, which include giants like GlaxoSmithKline, Merck, Eli Lilly, and Sanofi, will put up about one-half of the $230 million; NIH is providing the other half. AMP, which will also involve patients groups, will be overseen by the Foundation for the NIH. Although the foundation already oversees various similar-sounding public-private partnerships , this one will be more comprehensive, participants say. Pfizer President of Worldwide Research & Development Mikael Dolsten explained: “What we would like to have is a … GPS for human disease.”
Collins said NIH spent more than 2 years in “intense discussions” with companies to put together the partnership, and it has personal significance. As director of NIH’s genome institute, Collins helped lead the massive sequencing effort that produced the first draft of the human genome in 2001. The Human Genome Project has since been criticized for failing to appreciably improve medical care or drive down costs. For example, researchers have found more than 1000 genetic variants pointing to proteins that predispose some people to disease. But only about a half-dozen promising drug targets have come out of this effort, Collins notes.
The problem, Collins says, is not a lack of candidates but the challenge of sorting through “a deluge of information.” AMP will do this by bringing together academic scientists and industry for several 3- to 5-year pilot projects. Participants will share data openly. (In some cases, access will be restricted to researchers in order to protect patient privacy.) For companies, the payoff will come later, when they develop compounds to block the newly identified drug targets.
The projects include:
Alzheimer’s disease—At $130 million, this project will receive the lion’s share of AMP funding. It will look for new biomarkers for disease progression, such as imaging data or molecules in blood and spinal fluid. The biomarkers will be studied as part of four NIH-funded clinical trials that are testing ways to delay or prevent the disease. The project will also analyze existing brain tissue samples from Alzheimer's patients to validate biological targets, develop network models for late-onset Alzheimer's, and screen compounds against novel targets.
Type 2 diabetes—This $58 million project will pool genetic and clinical data on up to 150,000 patients, some contributed by companies, and share this information through a web portal. One goal is to understand in more detail the complications of diabetes, such as kidney failure, Dolsten says. The project will also search for rare individuals with unusually severe or mild disease who could be studied to validate targets.
Rheumatoid arthritis and lupus—A major goal of the $42 million project will be to analyze single cells from newly collected tissue samples, particularly from the synovium (the tissue lining joints), to better understand these autoimmune diseases at a molecular level and search for new drug targets.
AMP follows on other NIH initiatives in the past few years to accelerate drug development, such as the creation 2 years ago of a new National Center for Advancing Translational Sciences (NCATS). For example, NCATS has launched a project  in which companies work with academic researchers to find new uses for abandoned drugs. NCATS is not directly involved with AMP; instead, three disease-specific NIH institutes will fund the projects, Collins said.
Later this year, the institutes will release requests for proposals to seek academic partners. The investigators who win the grants will then become part of joint disease steering committees. If AMP is successful, NIH hopes it will expand to other diseases.