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Drug Repurposing: Data-driven drug repositioning and combination therapy (Milestone 7.C)

In Progress

Timeline Start - End

2018 - 2024

Research Implementation Area

Drug Repurposing and Combination Therapy Development

Continue to develop resources, capabilities and partnerships to advance data-driven drug repositioning and combination therapy.

Success Criteria

  • Support the development of an AD connectivity Map (CMap), whereby genes, drugs, and disease states are connected by common gene and other omics expression signatures in disease-relevant cell types (iPSC neurons, microglia, astrocytes, mixed cell cultures, organoids).

  • Establish an academic/industry partnership where industry partners can submit failed phase II/III compounds for molecular profiling that would enable computational drug repositioning analysis for AD.

  • Improve the regulatory environment for repurposing of drugs and combination therapies to obtain longer periods of exclusivity similar to those for orphan indications.

Summary of Key Accomplishments

NIA supports big data approaches to drug repurposing and combination therapy development through a targeted funding initiative aimed at developing “connectivity maps” matching the molecular signatures of AD/ADRD with the molecular signatures of FDA-approved drugs in cultured brain cells. This program also invites projects in which academic institutions partner with collaborators from the biotech/pharmaceutical industry aimed at repurposing failed drug candidates for AD treatment and prevention. Lastly, in November 2021, NIA convened a workshop that brought together NIA-funded investigators with representatives from the pharmaceutical industry and FDA to discuss progress and challenges in drug repurposing for AD/ADRD treatment, including regulatory approval pathways and regulatory science issues.

The key accomplishments summary is current as of July 2022. 

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