An experimental gene therapy injected into the brains of patients with Parkinson’s disease improved tremor, stiffness, and other movement symptoms and was safe with few side effects over six months of follow-up, a study shows.
The small study represents the first successful clinical trial comparing a gene-based treatment to sham treatment in Parkinson’s or any neurologic disorder, says Michael Kaplitt, MD, PhD, who developed the gene therapy more than a decade ago. He hopes to market it if phase III trials confirm its effectiveness.
Kaplitt is vice president for research in the department of neurological surgery at Weill Cornell Medical College in New York City. He is a co-founder of the company Neurologix, which is developing the treatment and funded the study.