# Definitive Preclinical Studies of Hydrolase Gene Transfer to Treat Cocaine Abuse

> **NIH NIH UH3** · MAYO CLINIC ROCHESTER · 2021 · $853,821

## Abstract

PROJECT SUMMARY
This research will be undertaken with the ultimate goal of FDA permission for a First-In-Human clinical trial of a
gene therapy to aid treatment-seeking cocaine users in becoming and remaining abstinent. The basis of the
therapy is a virus-mediated gene transfer of a normal plasma enzyme whose ability to metabolize cocaine into
harmless byproducts has been massively enhanced by 5 point mutations in the active site. Accumulated data
from the investigator's laboratory indicates that such a treatment should be effective in reducing cocaine
reward value to a point where even a compulsive user will be much less motivated to keep taking the drug. A
wide range of toxicology and pathology data acquired in the same series of studies on mice, rats, and rhesus
monkeys demonstrated NO adverse effects. These data were recently submitted to the FDA in a pre-IND
package and discussed in a “pre-pre-IND teleconference” with Agency personnel who raised no firm objections
to the proposed human trial. However FDA requested new data and repetition of key studies with vector
produced under “good lab practice” (GLP) standards. The present proposal seeks funding to acquire the
needed (large) amounts of vector for definitive preclinical studies carried out with professional quality
assurance (QA). In addition, we are committed to paving the way for an initial human study with the validated
final reagent, to be carried out by Drs. Tom Newton and Tom Kosten at Baylor College of Medicine. Thus all
funds will go towards testing a promising new addiction therapy and carrying it across the threshold to clinical
application.

## Key facts

- **NIH application ID:** 10241314
- **Project number:** 5UH3DA042492-05
- **Recipient organization:** MAYO CLINIC ROCHESTER
- **Principal Investigator:** W. Michael Hooten
- **Activity code:** UH3 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $853,821
- **Award type:** 5
- **Project period:** 2016-08-15 → 2025-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10241314

## Citation

> US National Institutes of Health, RePORTER application 10241314, Definitive Preclinical Studies of Hydrolase Gene Transfer to Treat Cocaine Abuse (5UH3DA042492-05). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/10241314. Licensed CC0.

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