Cutting-edge gene therapy may be one step closer to reaching Parkinson’s patients. Findings from a randomized, double-blind Phase II trial published in The Lancet Neurology show that six months after undergoing gene therapy surgery, 22 patients had a 23 percent improvement in motor function (measured by the Unified Parkinson’s Disease Rating Scale) compared to a 13 percent improvement observed in 23 patients comprising a control group that had sham surgery without the implantation of any genes.
The gene therapy that researchers from the Feinstein Institute for Medical Research in New York utilized in the trial was an injection of the glutamic acid decarboxylase (GAD) gene packaged in a benign virus delivered directly into the brain of patients who were previously unresponsive to other drug treatments.
Though not thoroughly impressed with the trial results, ACSH's Dr. Gilbert Ross still thinks that “the study was meticulous, and though it may prove to be very promising in future Parkinson’s disease research as well as in other similar conditions, these results hardly amount to a miraculous finding.” Dr. Ross emphasizes the need for better treatment therapies since Parkinson’s disease is characterized by progressive, debilitating symptoms such as masking of facial movements, abruptions in coordinated motion and a resting, "pill-rolling" tremor.
