Accelerating Medicines Partnership

AMP: Type 2 Diabetes

AMP - T2D header

Type 2 Diabetes (T2D) currently affects about 26 million people in the US and over 382 million people worldwide.  Up to 79 million adults in the U.S. have “pre-diabetes” and are at high risk of developing T2D.  Major complications with T2D include increased risk for cardiovascular disease, kidney failure, lower limb amputations, and blindness resulting from retinopathy.

The Challenge
Current therapies exist to control T2D, but none effectively reverse long-term disease progression or prevent complications. Thus, new drugs need to be developed to address these issues. However, many companies are reluctant to invest in developing new drugs due to the high cost and failure rate. Additionally, although much is known about T2D, scientists have an incomplete understanding of the many biological processes involved in disease development.  Together, these issues present a challenge for developing new and effective drugs for T2D.

The Overarching Strategy
Despite the scientific knowledge gap, researchers have collected an enormous amount of genetic data from hundreds of thousands of people with T2D.  Integrating data from such a large population will allow identification of rare genetic differences associated with T2D disease onset, severity, progression, complications, and protection, which could lead to discovery of novel relevant disease mechanisms and pathways. Importantly, these data could provide a model of what targeted drugs might achieve.  The outcome of such analyses could both inform and validate drug targets, which would increase the likelihood of identifying the most successful drug candidates and bringing them to market at a reduced cost and failure rate.

Proposed Research Plan
The AMP T2D public private partnership seeks to create a T2D Knowledge Portal  by building a database of DNA sequence, functional genomic and epigenomic, as well as clinical data from studies on T2D and its cardiac and renal complications across roughly 100,000 – 150,000 individuals.  The Knowledge Portal will house the dataset and analytical tools, which will be publicly accessible to academic and industry researchers for data mining and analysis.  The project will also seek to fill in data gaps in the Knowledge Portal by both collecting new data and executing specific data analyses. Researchers will investigate targets of particular interest by sequencing DNA from select individuals, incorporating the resulting new data into the common database. The end goal will be to have a powerful, publicly available T2D Knowledge Portal with a progressively growing database that incorporates the analytical tools to allow identification and validation of effective and successful T2D drug candidates.

The Members in the Partnership
The AMP T2D Partnership Steering Committee includes members from industry (Janssen Research and Development, LLC, Sanofi, Merck Sharp and Dohme Corp., Eli Lilly and Company, and Pfizer, Inc.), government (NIH), and academia (University of Pennsylvania). The AMP T2D Partnership is also supported by the Juvenile Diabetes Research Foundation and the American Diabetes Association. AMP T2D is managed by FNIH (contact information below).

Read the Detailed Research Plan here >>

Sanya Fanous Whitaker, PhD, PMP
Scientific Program Manager, Metabolic Disorders
Tel: 301-496-1518
Email: swhitaker [at] fnih [dot] org