Gene therapy is a growing field of medicine with enormous treatment potential for many currently incurable diseases. Scientists have so far created gene therapies to tackle a range of diseases, including the leading cause of blindness, sickle cell anemia, and a range of rare genetic diseases.
Now, scientists from the University of California San Diego have managed to tackle another medical issue using gene therapy: spinal cord injuries.
Around half of the individuals who experienced spinal injuries also deal with neuropathic pain. This is where the damaged nerves communicate to the brain in an abnormal way. This failure in the nervous system causes chronic burning and shooting pains, a difficult and uncomfortable condition to live with.
The only way to reduce neuropathic pain was with drugs and pharmaceutical therapy. However, the drugs need sophisticated medical administration and are linked to many adverse side effects. This new gene therapy, however, carries no adverse side effects and delivers treatment specifically to the damaged nerves.
The therapy works by introducing the gene gamma-aminobutyric acid (GABA) to these cells. GABA binds to the pain-causing spinal cells, blocking communication with the brain, and stopping pain signals from transferring. Tests with lab mice showed great promise using this therapy, and the reduced pain persisted for at least 2.5 months after treatment.
“One of the prerequisites of a clinically acceptable antinociceptive (pain-blocking) therapy is minimal or no side effects like muscle weakness, general sedation, or development of tolerance for the treatment,” said senior author Martin Marsala. “A single treatment invention that provides a long-lasting therapeutic effect is also highly desirable. These findings suggest a path forward on both.”
Source study: Molecular Therapy – Precision spinal gene delivery-induced functional switch in nociceptive neurons reverses neuropathic pain