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Drug Repurposing: Multiscale models of disease (Milestone 7.A)


Timeline Start - End

2016 - 2021

Research Implementation Area

Drug Repurposing and Combination Therapy Development

Expand existing and develop new systems biology and systems pharmacology research programs to build multiscale models of disease that will lead to the identification of networks/sub-networks as drug targets and readouts of therapeutic activity and advance the validation of existing and novel targets, rational drug repositioning and rational development of combination therapy.

Success Criteria

Launch at least 6 cross-disciplinary research programs that bring together experts in translational bioinformatics, computational biology, genetics, epidemiology, drug discovery and clinical research to develop a predictive model of the disease. Efforts should support:

  • development of computational tools and infrastructure to allow basic and clinical  researchers to query model in silico and validate it by using it for  patient stratification, predictions of efficacy, on- and off-target adverse effects.
  • identification of quantitative methods to access synergy between (1) multiple therapeutic agents and (2) pharmacologic/non-pharmacologic perturbations.
  • development of phenotypic screens (in cell based and/or animal models) to advance  rational drug repositioning and data-driven development of combination therapy based  on the ability of individual or  combinations of therapeutic agents to shift the network state away from disease.

Summary of Key Accomplishments

Through a targeted funding initiative, the NIA established the Drug Repurposing and Combination Therapy Development program. The program consists of over 20 funded projects and brings together cross-disciplinary teams of experts in data science machine, learning and AI and AD/ADRD clinical and basic research experts. The teams are using publicly available big data on AD/ADRD and other diseases (genetic/multi-omic/drug data/EHR/insurance claims data) to identify approved drugs currently used for other conditions, as well as candidate drugs from failed clinical trials, with potential to be efficacious in AD/ADRD as individual drugs, or as drug combinations. The program provides funding for testing the computational predictions in animal models and in proof-of-principle human studies.

The key accomplishments summary is current as of March 2022. 

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