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NIH, Pharmas, Non-Profits Partner on Molecular Drug Targets

Francis Collins

NIH director Francis Collins (NIH)

4 February 2014. National Institutes of Health (NIH), with 10 pharmaceutical companies and 8 not-for-profit organizations, are collaborating on identification of targets at the molecular level for new drugs and diagnostics. The Accelerating Medicines Partnership — a five-year, $230 million initiative — is expected to focus on Alzheimer’s disease, type 2 diabetes, and the autoimmune disorders rheumatoid arthritis and systemic lupus erythematosus (lupus).

The initiative aims to develop a new model for identifying and validating promising biological targets of disease, often called biomarkers, that are most likely to respond to new therapies. A key part of the collaboration, says NIH, is the agreement by participants to make available the data and analysis generated by Accelerating Medicines Partnership to the broader biomedical community.

NIH director Francis Collins says the partnership is in some respects a recognition that current drug processes are not working properly, leaving too many patients and their families waiting for treatments that work. “The good news is that recent dramatic advances in basic research are opening new windows of opportunity for therapeutics,” says Collins in an NIH statement. “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.”

Of the $230 million budgeted over the next five years, $130 million will be devoted to work on Alzheimer’s disease, with $58 million going to type 2 diabetes, and $42 million for the autoimmune disorders. Of the total funding, $119 million is contributed by NIH, while industry is providing $111 million.

For Alzheimer’s disease, participants plan to identify an expanded set of biomarkers — genes, proteins, and other molecules — that can predict clinical outcomes, and incorporate those biomarkers into four clinical trials funded by NIH that are testing therapies to delay or prevent the disease. In addition, teams will use system biology tools to analyze tissue samples of patients with Alzheimer’s disease to validate current or uncover new treatment targets.

For type 2 diabetes, Accelerating Medicines Partnership members will create a portal storing genomic and epigenomic (chemicals generated by genomic modifications) data, DNA sequences, and clinical data from studies of type 2 diabetes, and related complications involving the heart and kidneys. That portal, amassing data from trials covering 100,000 to 150,000 patients, is expected to yield promising therapy targets. The project will also identify DNA regions important to the progression of type 2 diabetes, and find DNA variations in those regions that offer a greater likelihood of success for therapies.

For rheumatoid arthritis and lupus, the initiative plans to analyze tissue and blood specimens from people with these diseases, to identify changes in biology at the level of single cells, and provide better insights into the process behind the disease. The project expects to focus on cells and groups of cells related to auto-immunity, where the immune system is tricked into attacking healthy cells, and develop computational tools that integrate different data types to identify promising molecular pathways.

The companies taking part in Accelerating Medicines Partnership are AbbVie, Biogen Idec, Bristol-Myers Squibb, GlaxoSmithKline, Johnson & Johnson, Eli Lilly and Company, Merck, Pfizer, Sanofi, and Takeda Pharmaceutical. The not-for-profit groups taking part are Alzheimer’s Association, American Diabetes Association, Lupus Foundation of America, Foundation for the NIH, Geoffrey Beene Foundation, PhRMA, Rheumatology Research Foundation, and USAgainstAlzheimer’s. The U.S. Food and Drug Administration is also participating. Foundation for the NIH is serving as the project manager.

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