PROJECT SUMMARYThis research will be undertaken with the ultimate goal of FDA permission for a First-In-Human clinical trial of agene therapy to aid treatment-seeking cocaine users in becoming and remaining abstinent. The basis of thetherapy is a virus-mediated gene transfer of a normal plasma enzyme whose ability to metabolize cocaine intoharmless byproducts has been massively enhanced by 5 point mutations in the active site. Accumulated datafrom the investigator's laboratory indicates that such a treatment should be effective in reducing cocainereward value to a point where even a compulsive user will be much less motivated to keep taking the drug. Awide range of toxicology and pathology data acquired in the same series of studies on mice, rats, and rhesusmonkeys demonstrated NO adverse effects. These data were recently submitted to the FDA in a pre-INDpackage and discussed in a ?pre-pre-IND teleconference? with Agency personnel who raised no firm objectionsto the proposed human trial. However FDA requested new data and repetition of key studies with vectorproduced under ?good lab practice? (GLP) standards. The present proposal seeks funding to acquire theneeded (large) amounts of vector for definitive preclinical studies carried out with professional qualityassurance (QA). In addition, we are committed to paving the way for an initial human study with the validatedfinal reagent, to be carried out by Drs. Tom Newton and Tom Kosten at Baylor College of Medicine. Thus allfunds will go towards testing a promising new addiction therapy and carrying it across the threshold to clinicalapplication.
|Effective start/end date||8/1/16 → 7/31/18|
- National Institutes of Health: $1,430,948.00