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Translational Therapeutics Program

Overview

This program supports research that seeks to identify and validate targets for intervention development; assess mechanisms of action of putative interventions; identify and validate biomarkers of disease progression and treatment response; demonstrate and evaluate target engagement of proposed interventions; and evaluate their clinical efficacy. The overall goal is to facilitate the development of novel treatments for major mental disorders in adults (ages 18+) and clinical dimensions of psychopathology associated with these disorders.

Areas of Emphasis

  • Early-phase (Proof of Mechanism) studies to test and analyze novel pharmacological and neuromodulation interventions and their molecular and/or clinical targets
  • Early phase trials focused on evaluating metabolism and pharmacologic actions of interventions in humans, side effects associated with increasing doses, and early analysis of molecular and clinical targets
  • Proof of Concept clinical trials to demonstrate target engagement, safety, and early signs of efficacy of promising interventions
  • Evaluation of potential biomarkers, including receptor occupancy (PET) and engagement of relevant brain systems (fMRI, clinical electrophysiology, emotional reactivity, neurocognitive performance, etc.) to demonstrate target engagement and biological effects
  • Treatment studies integrating biomarkers with traditional endpoints to validate the utility of putative surrogate markers of treatment response
  • Studies to predict treatment response and facilitate individualized treatment strategies
  • “Repurposing” studies of drugs approved by FDA for other indications when research suggests that the compound(s) has the potential to affect a biological mechanism contributing to mental disorders
  • Studies to evaluate the mechanisms of treatment–emergent side effects of psychiatric therapeutics
  • Studies to predict treatment-emergent side effects of psychiatric therapeutics and facilitate individualized pharmacological therapeutics
  • Studies to identify intervention development to mitigate adverse effects of psychopharmacological and neuromodulation agents

Contact

Mi Hillefors, M.D., Ph.D.
Program Chief
6001 Executive Blvd., Room 7123, MSC 9632
301-443-2738, mi.hillefors@nih.gov