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Scientists want human trials for gene therapy that could help battle addiction

In recent years, new gene editing tools have been used for everything from genetic modification of plants to increase crop yields to, far more controversially, genetic tampering with human embryos. Could a form of gene therapy also be useful in helping treat cocaine addiction, a form of addiction that proves highly resistant to alternative approaches, such as conventional medical treatment and psychotherapy? That’s what researchers from the world-famous Mayo Clinic are hoping to prove.

They are seeking approval for the first-in-human studies of an innovative new single-dose gene therapy. Their approach involves the delivery of a gene coding for an enzyme, called AAV8-hCocH, which metabolizes cocaine in the body into harmless byproducts. In order to progress to this next step in their work, they first have to gain permission from the U.S. Food and Drug Administration (FDA) in the form of an Investigational New Drug Application.

The researchers have already demonstrated the safety of their approach in mice. In a prior experiment, they showed a complete lack of adverse effects in mice which had both been previously exposed to cocaine and those which had not.

“Mice given one injection of AAV8-hCocH and regular daily injections of cocaine had far less tissue pathology than cocaine-injected mice with no vector treatment,” the researchers wrote in the abstract for their paper describing the work. “Biodistribution analysis showed the vector located almost exclusively in the liver. These results indicate that a liver-directed AAV8-hCocH gene transfer at reasonable dosage is safe, well-tolerated, and effective. Thus, gene transfer therapy emerges as a radically new approach to treat compulsive cocaine abuse.”

This is not the first time similar work has been carried out. In February 2017, scientists at the University of British Columbia genetically engineered a mouse so as to be incapable of becoming addicted to cocaine. However, one of the researchers on the project told Digital Trends that transferring this work across to humans for possible treatment for addiction was not straightforward. Instead, that work was more focused on exploring the link between drug use and genetics and biochemistry.

There’s still a whole lot more research that needs to be done in this area. Even if the FDA grants the Mayo Clinic researchers permission for their human trials, we’ll most likely be waiting a few years at least before this treatment could be rolled out to the general public. It’s an exciting leap forward, nonetheless.

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