# Proof-of-Concept Human Laboratory Testing of Novel Drug Candidates Identified by INIA-NeuroImmune

> **NIH NIH U01** · SCRIPPS RESEARCH INSTITUTE, THE · 2020 · $522,391

## Abstract

Project Summary
The development of novel medications with robust effect sizes and good safety and tolerability is an INIA RFA
research priority. This translational project will respond to this research challenge by providing proof-of-concept
human laboratory testing of the top three neuroimmune drug candidates identified as having therapeutic
potential for alcohol use disorder by Dr. Mayfield/Farris/Ponomarev's “drugging the network” computational
genomic analyses and by Drs. Bell's, Blednov/Messing's, Crabbe/Ozburn's and Lasek's animal models. Drugs
identified for human study will either be FDA-approved for other indications or available for study under an IND
and will be selected by Dr. Mason based on scientific prioritization by the INIA-Neuroimmune Executive
Committee and safety considerations. Drugs will be tested in a highly standardized, reliable, and valid human
laboratory model informing the three stages of the addiction cycle: withdrawal/negative affect,
preoccupation/anticipation, and binge/intoxication, with human laboratory results serving as an analogue for
drinking outcomes of double-blind, placebo-controlled clinical trials. Subjects for each study will be 50 non-
treatment-seeking male and female paid volunteers who meet DSM-5 criteria for alcohol use disorder of ≥
moderate severity (AUD-MS). Studies are randomized, double-blind, and placebo-controlled. The treatment
duration is at least 1-week and guided by drug pharmacokinetics. The primary endpoint is craving scores in
response to in vivo alcohol cues presented in the laboratory, with confirmatory physiological outcomes, and
naturalistic measures of drinking, mood, and craving. Specific Aim 1: To evaluate INIA-Neuroimmune drug
candidates in paid non-treatment-seeking volunteers with current AUD-MS using the human lab model
informing targets for the 3 stages of the addiction cycle. Specific Aim 2: To identify potential biomarkers of
clinical outcomes in peripheral markers of stress and inflammation (e.g., high-sensitivity C-reactive protein,
selected chemokines, pro- and antiinflammatory cytokines, and cortisol) in collaboration with Drs.
Roberto/Roberts/Bajo. Safety Aim: To evaluate the safety and tolerability of INIA-Neuroimmune drug
candidates in subjects with AUD-MS, as assessed by significant changes from baseline in EKG, routine blood
and urine biochemistry, vital signs, physical exam, and subjective complaints relative to placebo. Hypothesis:
The overall hypothesis under test in that, relative to placebo, novel drug candidates identified by INIA-
Neuroimmune will significantly attenuate responsivity to alcohol cues in the human lab model and reduce
drinking, craving, and negative affect during treatment and 1-month of post-treatment follow-up. Interpretation
of Results: Positive findings will provide clinical validation of neuroimmune targets and mechanisms identified
by INIA-Neuroimmune and provide a rational basis for later phase testing of candidate drugs as potential...

## Key facts

- **NIH application ID:** 9853705
- **Project number:** 5U01AA025476-04
- **Recipient organization:** SCRIPPS RESEARCH INSTITUTE, THE
- **Principal Investigator:** BARBARA J MASON
- **Activity code:** U01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $522,391
- **Award type:** 5
- **Project period:** 2017-02-01 → 2022-01-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9853705

## Citation

> US National Institutes of Health, RePORTER application 9853705, Proof-of-Concept Human Laboratory Testing of Novel Drug Candidates Identified by INIA-NeuroImmune (5U01AA025476-04). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/9853705. Licensed CC0.

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