Source: Thailand Medical News Jan 08, 2020 4 years, 10 months, 6 days, 33 minutes ago
In an attempt to try out a new radical approach to treat
cocaine addition, researchers at the Mayo Clinic are seeking approval for first-in-human studies of a single-dose
gene therapy. To support the safety and efficacy of this approach they have demonstrated the successful delivery of a
gene coding for an enzyme that metabolizes
cocaine into harmless byproducts in mice. The study is published in Human
Gene Therapy.
Dr Stephen Brimijoin and colleagues from Mayo Clinic, Rochester, MN, coauthored the article entitled "Systemic Safety of a Recombinant AAV8 Vector for Human
Cocaine Hydrolase
Gene Therapy: A Good Laboratory Practice Preclinical Study in Mice." In advance of filing for an Investigational New Drug Application with the U.S. Food and Drug Administration, which would allow for human testing, the researchers needed to show the systemic safety of their recombinant adeno-associated viral (AAV) 8 vector, which targets its therapeutic
gene payload to the liver. They showed a total lack of viral vector-related adverse effects in both
cocaine-experienced and
cocaine-naïve mice at different doses. In fact, mice who received the
gene therapy followed by daily
cocaine injections had much less tissue pathology than those mice who received daily
cocaine injections but did not have the
gene therapy.
Chief author, Dr Terence R. Flotte, MD, Celia and Isaac Haidak Professor of Medical Education and Dean, Provost, and Executive Deputy Chancellor, University of Massachusetts Medical School, Worcester told
Thailand Medical News, "Substance use disorders present an immense public health problem in the US and other industrialized countries. Putting the power of an innovative
gene therapy to work on this problem presents an exciting new approach."
The research team is expected to start human trials of the
gene therapy in
cocaine addicts by the first quarter of 2020.
Reference
: Vicky Ping Chen et al, Systemic Safety of a Recombinant AAV8 Vector for Human
Cocaine Hydrolase Gene Therapy: A Good Laboratory Practice Preclinical Study in Mice,
Human Gene Therapy (2019).
DOI: 10.1089/hum.2019.233
