The National Institutes of Health, 10 biopharmaceutical companies, and several nonprofit organizations have partnered 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 aims to pinpoint biological targets that are most likely to respond to new therapies. Partners in this initiative, which include major drug makers such as GlaxoSmithKline and Merck, along with nonprofits such as the Rheumatology Research Foundation, the Lupus Foundation of America, the American Diabetes Association, and the Alzheimer’s Association, are expected to invest more than $230 million over 5 years in the first projects, which will focus on rheumatoid arthritis and systemic lupus erythematosus (total funding of $41.6 million), type 2 diabetes ($58.4 million), and Alzheimer’s disease ($129.5 million).
Data and analyses generated by these projects will be made publicly available to the broad biomedical community through an Internet-based information portal, and the initial projects could set the stage for broadening the initiative into other diseases and conditions, the NIH says.
For rheumatoid arthritis and lupus, the project will focus on collecting and analyzing tissue and blood samples from patients to see how changes at the single-cell and systems levels can provide insight into the disease process and inform comparisons across diseases, as well as identify differences between patients in response to treatment.
The type 2 diabetes project will aim "to use and supplement the substantial amount of human genetic data available from people with type 2 diabetes – or at risk for developing type 2 diabetes – to identify and validate novel molecules and pathways as targets for therapeutic development." Researchers also will build a type 2 diabetes knowledge portal for academic and industry researchers that will contain DNA sequences, functional genomic and epigenomic information, and clinical data from studies on type 2 diabetes and its cardiac and renal complications totaling 100,000-150,000 individuals.
The Alzheimer’s project will seek to increase the amount of data available from clinical trials to increase support for therapeutic target validation in humans. This will be done by using selected biomarkers in four NIH- and industry-supported clinical trials that are designed to delay or prevent disease onset, as well as performing a large-scale analysis of brain tissue samples from Alzheimer’s disease patients to validate biological targets previously shown to play key roles in disease progression and increase understanding of the molecular pathways involved in the disease, in order to identify new potential therapeutic targets.