Gene therapy reverses symptoms.

• Position: Parkinson's Disease

A gene therapy called NLX-P101 dramatically reduces movement impairment in Parkinson's disease patients, according to results of a Phase 2 study published in Lancet Neurology. The approach introduces a gene into the brain to normalize chemical signaling.

The study is the first successful randomized, double-blind clinical trial of a gene therapy for Parkinson's or any neurologic disorder, and it represents the culmination of 20 years of research by study coauthors Michael Kaplitt (vice chairman for research in the Department of Neurological Surgery at Weill Cornell Medical College and a neurosurgeon at New York-Presbyterian Hospital) and Matthew During (neurosurgeon at Connecticut's Yale-New Haven Hospital).

"Patients who received NLX-P101 showed a significant reduction in the motor symptoms of Parkinson's, including tremor, rigidity, and difficulty initiating movement," says Kaplitt, who pioneered the approach and helped design the clinical trial. "This not only confirms the results of our Phase 1 trial, but represents a major milestone in the development of gene therapy for a wide range of neurological diseases."

"This is great news for the 1,500,000 Americans living with Parkinson's disease," adds During. "Since this is also the first gene therapy study for a neurological disease to achieve success in a rigorous randomized, double-blind design compared with a sham group, this is also a crucial step forward toward...