Joanne Fertig, Ph.D. and Kathy Jung, Ph.D.
During the past two decades progress has been made in developing medications to treat Alcohol Use Disorders (AUD). Currently there are only three Food and Drug (FDA)-approved medications for the treatment of AUD: disulfiram, oral and long-term injectable naltrexone, and acamprosate. However, given the heterogenous nature of AUD, many individuals show only limited or no response to these medications. Once alcohol has resulted in organ damage, which, for purposes of this RFA include: pathology in liver, lung, muscle, bone, pancreas, fetus, the cardiovascular system, or disruption of immune function, and development of certain cancers, the situation is even more dire since there are no FDA approved medications to treat these alcohol-related consequences. Because of this shortage of approved medications to prevent and treat AUD and AROD, developing new, safe and efficacious medications are a high priority for NIAAA.
The purpose of this RFA is to support research that advances compounds towards FDA approval. NIAAA is seeking cooperative agreement applications (U01) for studies that move promising compounds through the drug development pipeline for the treatment of Alcohol Use Disorder (AUD) or Alcohol-Related Organ Damage (AROD).
The aim of this initiative is to move candidate compounds through any of the following drug development phases: pre-clinical Investigational New Drug (IND) requirements; Phase 1 safety, tolerability, and dosing studies; Phase 2 human laboratory and proof-of-concept trials. Phase 3 trials will not be accepted for this RFA. Within these phases of drug development, each proposed project should have a defined entry and exit point. This funding opportunity will support pre-clinical animal efficacy studies if, and only if, the candidate compound is patentable and has a plausible path to commercialization. Studies that are not intended to support an FDA submission are not appropriate for this RFA and should be submitted to the appropriate parent Funding Opportunity Announcement (FOA).
Applicants who propose candidate compounds for this RFA must minimally satisfy the following conditions:
- The candidate compound must be adequately characterized in terms of structure/identity, selectivity, other modality-specific characteristics.
- The candidate compound should be patentable and have a plausible path to commercialization.
- All proposed studies must be conducted consistent with FDA regulatory requirements.
- The applicant must have a clear path to the intellectual property related to the candidate compound.
This RFA will accept applications for studies with new chemical entities and repurposed drugs products. For example, repurposing can include chemical modifications of the parent molecule, changes in the route of administration and/or combination of the parent drug with another compound. Repurposed drugs products must meet both intellectual property and FDA regulatory requirements to be eligible for this RFA.
Justification for Re-issue/Outcome
This RFA is a reissue of the FOA, Medications Development for the Treatment of Alcohol Use Disorder (RFA-19-005). The previous RFA solicited studies exclusively for the treatment of AUD. Because of the lack of FDA approved medications for AROD, this RFA will also accept applications for candidate compounds to prevent and treat AROD. Five applications were funded to study novel compounds to treat AUD. Those applications are ongoing, and no results are currently available. NIAAA anticipates that issuing this RFA for medications for both AUD and AROD, will highlight our commitment to these research priorities and stimulate investigators from both the private and public sectors.
NIAAA seeks cooperative agreement applications (U01). This mechanism requires the appointment of an NIH Project Scientist, who will have substantial involvement above and beyond the normal program stewardship of the award. The NIH Project Scientist is a partner within the research team representing the government's interest in the substantive work of the research team with specific responsibilities.
Applications will be accepted from for-profit and not-for-profit entities, including academic institutions, pharmaceutical and biotechnology companies, private and public foundations, and small businesses.