Common Metabolic Diseases

Overview

The Accelerating Medicines Partnership® (AMP®) program is a public-private partnership between the National Institutes of Health (NIH), the U.S. Food and Drug Administration (FDA), and multiple public and private organizations. Managed through the Foundation for the NIH (FNIH), AMP aims to identify and validate the most promising biological targets for therapeutics. The AMP Common Metabolic Diseases (AMP CMD) project is aimed at identifying promising new targets for six common metabolic diseases: liver diseases such as nonalcoholic steatohepatitis, kidney diseases, obesity, cardiovascular diseases, type 2 diabetes/prediabetes, and type 1 diabetes.

Data generated in AMP will be considered precompetitive and made publicly accessible to the broad biomedical community for further research. This overview addresses the CMD research plan.

About Common Metabolic Diseases

Common Metabolic Diseases (CMD) encompass multiple related diseases associated with metabolic dysregulation in various tissues. Their increasing prevalence represents a major public health burden. A growing body of evidence suggests that these diseases may be driven by common underlying pathologies including genetic drivers and overlapping molecular pathways. The evidence is consistent with data suggesting that the presence of one or more of these diseases, increases the likelihood of developing additional metabolic diseases.

Need for New Therapies

Traditionally, common metabolic diseases have been targeted and treated as individual entities. Their potential shared molecular basis, however, represents an opportunity to treat multiple diseases simultaneously and to provide more effective treatment outcomes. Moreover, current therapies for many individual diseases, while effective, are unable to reverse disease progression or prevent complications. A better understanding of the biology of these diseases will generate new targets for improved therapies and will potentially identify common targets to effectively treat diseases across the common metabolic spectrum.

AMP Approach

Building on the rich genetic data that has been generated by the research community, the opportunity now exists to develop a deep understanding of the molecular basis of individual metabolic diseases and to clarify shared molecular pathways across the diseases. To meet this goal, AMP CMD brings together four biopharmaceutical companies with four teams of investigators at institutions funded by the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), including a Knowledge Portal team. The consortium will capitalize on existing genomic data, including from the AMP Type 2 Diabetes (AMP T2D) program, and will generate new largescale molecular data to achieve a deep understanding of genes and pathways that underlie common metabolic diseases. Using this information, the consortium will identify and integrate datasets to identify potential biotargets and new therapeutic targets.

CMD Knowledge Portal

The AMP CMD program builds on scientific advances and analytical tools developed as part of the earlier AMP T2D program which included development of the AMP T2D Knowledge Portal, a large database of genetic information from more than 1.5 million individuals with diabetes and related conditions worldwide. The AMP T2D Knowledge Portal’s structure and tools have since been widely expanded for curating genetic data on a variety of other traits. This adaptability is one of the features that has yielded the present opportunity, to identify underlying drivers of a broader array of common metabolic diseases.  

The new AMP CMD program will result in a substantial amount of new information added to the database, as well as the development of new tools to analyze links between these six diseases, incorporated into the AMP CMD Knowledge Portal. As with other AMP programs, data will be made available to the scientific research community with security and ethical protections in place for participants’ data.

Governance

The steering committee (SC) for CMD comprises of representatives from NIH, the FNIH, and participating companies. The SC meets on a regular basis and is responsible for monitoring ongoing progress towards milestones. The SC operates under the direction of the overall AMP Executive Committee, which comprises representatives from NIH, the FNIH and participating companies

Budget: 5 years ($57 Million Total Project Funding)

Total project funding ($M)

ACCELERATING MEDICINES PARTNERSHIP and AMP are registered service marks of the U.S. Department of Health and Human Services.