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Experimental Therapeutics Program


The Experimental Therapeutics Program supports studies and clinical trials of novel interventions that assess mechanisms of action and seek to demonstrate and evaluate target engagement.  The goal is to facilitate the development of innovative treatments for major mental disorders or clinical dimensions of psychopathology associated with those disorders.  The Program supports multidisciplinary research on development and testing of novel pharmacological and non-pharmacological approaches to treating mental disorders, or clinical dimensions of psychopathology associated with those disorders.  Proposed research in this area is strongly encouraged to focus on the evaluation of relevant molecular or clinical target engagement; evaluation and validation of biomarkers (e.g., receptor occupancy and engagement of relevant brain systems to demonstrate biological effects) and assessment of their clinical utility in treatment trials.

Areas of Emphasis

  • Early-phase clinical studies to test and analyze novel pharmacological or non-pharmacological interventions and their molecular and/or clinical targets for treating major mental disorders or clinical dimensions of psychopathology (e.g., anhedonia, cognitive function, social engagement) associated with traditional mental disorders.
  • Small-scale Phase I or Phase IIa clinical trials (e.g., Proof of Concept) to demonstrate target engagement, safety, and early signs of efficacy of promising interventions.
  • Experimental Medicine studies, including initial Phase 0 (e.g., micro-dosing studies) and Phase Ia (e.g., First in Human) trials focused on rapidly evaluating metabolism and pharmacologic actions of interventions in humans, side effects associated with increasing doses, and early analysis of molecular and clinical targets to determine  whether to proceed with further testing in Phase Ib/Phase IIa trials.
  • Evaluation of potential biomarkers, including receptor occupancy (with PET) and engagement of relevant brain systems (with fMRI, clinical electrophysiology, emotional reactivity, neurocognitive performance, etc.), to demonstrate biological effects.
  • Clinical treatment studies integrating biomarkers with traditional endpoints to validate the clinical utility of putative surrogate markers of treatment response.
  • Studies to predict treatment response and facilitate individualized treatment strategies.
  • Testing of interventions that have been FDA-approved for other indications (re-purposing) if recent basic research discoveries suggest that the compound(s) has the potential to affect a biological mechanism contributing to mental disorders and has previously been untested in clinical studies.
  • Treatment studies that do not include measures to evaluate molecular and/or clinical targets of the proposed intervention will be deemed of lower priority to the Program.


Mi Hillefors, M.D., Ph.D.
Program Chief
6001 Executive Blvd., Room 7123, MSC 9632