NIH, non-profits and industry work to drive improved biomarker validation

Over the next 5 years, the National Institutes of Health, several nonprofit organizations and 10 biopharmaceutical companies will invest more than $230 million to distinguish new biomarkers in the areas of type 2 diabetes, Alzheimer’s disease and autoimmune disorders, eventually making all data publicly available, according to the announcement.

“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,” Francis S. Collins, MD, PhD, NIH Director, said in a press release. “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.”

This collaborative group, the Accelerating Medicines Partnership, or AMP, will operate through the Foundation for the NIH and the first projects will focus on type 2 diabetes, Alzheimer’s disease and autoimmune disorders such as rheumatoid arthritis and lupus.

Upon reaching important milestones and at the 3- and 5-year marks, these pilot projects will release data and analyses to the biomedical community, according to the press release. Research plans have been developed and the involved parties will share costs, expertise and resources.

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

Specifically, in type 2 diabetes, the group aims to build a knowledge portal in which DNA sequence, functional genomic and epigenomic information and clinical data on heart and kidney complications of type 2 diabetes will reside. The data will include existing studies as well as new information on 100,000 to 150,000 individuals, according to the release. The group will focus on critical DNA regions involved in the development or progression of type 2 diabetes and natural variations within populations that would improve development of treatments.

In looking at Alzheimer’s disease, AMP plans to identify biomarkers to predict clinical outcomes. The group explained in the release that it will incorporate an expanded set of biomarkers into four NIH-funded and industry-supported clinical trials designed to delay or prevent disease. The other focus will be large-scale, systems biology analyses of tissue samples from the brains of patients with Alzheimer’s disease; these samples will help validate biomarkers working in disease progression and improve understanding of molecular networks, all in an effort to identify new potential therapeutic targets.

RA and lupus will be analyzed via tissue and blood samples from patients in order to look at biological changes at the single cell level, comparing the diseases and looking at their processes, according to the release. The group hopes to identify differences between patients with rheumatoid arthritis who respond to treatments and those who do not while providing a systems-level understanding of mechanisms of disease in both rheumatoid arthritis and lupus.

For more information: NIH, industry and non-profits join forces to speed validation of disease targets