Gene therapy for chronic pain a first in translational research

University scientists have begun a phase 1 clinical trial for the treatment of cancer-related pain, using a novel gene transfer vector injected into the skin to deliver a pain-relieving gene to the nervous system.

A gene transfer vector is an agent used to carry genes into cells. In this groundbreaking clinical trial, the investigators will use a vector created from herpes simplex virus (HSV) — the virus that causes cold sores — to deliver the gene for enkephalin, one of the body’s own natural pain relievers.

“In pre-clinical studies, we have found that HSV-mediated transfer of enkephalin can reduce chronic pain,” says Dr. David Fink, the Robert Brear Professor and chair of the department of neurology at the Medical School. Fink developed the vector with collaborators and will direct the study.

“After almost two decades of development and more than eight years of studies in animal models of pain, we have reached the point where we are ready to find out whether this approach will be effective in treating patients,” Fink says.

The investigators are recruiting 12 patients with intractable pain from cancer to examine whether the vector can be used safely to deliver its cargo to sensory nerves.

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