Scientists have developed a novel gene therapy called NLX-P101 that dramatically reduces movement impairment in Parkinson's patients.
Scientists have developed a novel gene therapy called NLX-P101 that dramatically reduces movement impairment in Parkinson's patients.
The approach introduces a gene into the brain to normalise chemical signalling.
The result of a Phase 2 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 co-authors Michael Kaplitt, a neurosurgeon at NewYork-Presbyterian Hospital/Weill Cornell Medical Center, and Matthew During, of the Ohio State University.
"Patients who received NLX-P101 showed a significant reduction in the motor symptoms of Parkinson's, including tremor, rigidity and difficulty initiating movement," said Kaplitt, who pioneered the approach and helped design the clinical trial.
"This not only confirms the results of our Phase 1 trial performed at NewYork-Presbyterian/Weill Cornell but also represents a major milestone in the development of gene therapy for a wide range of neurological diseases," he added.
"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 finally bringing gene therapy into clinical practice for patients with debilitating brain disorders." said During the co-inventor with Kaplitt, of the gene therapy procedure.
In the current study, 45 patients with moderate to advanced Parkinson's disease who were not adequately controlled with current therapies were enrolled in the double-blind trial, with half randomized to receive the gene therapy and the other half to a "sham surgery"-a mock procedure designed to make patients think they could have received the experimental approach.
"Improved motor control was seen at one month and continued virtually unchanged throughout the six-month study period," said Kaplitt, who also serves as associate professor of neurological surgery and director of the Laboratory of Molecular Neurosurgery at Weill Cornell Medical College.
The study has been published in the journal Lancet Neurology.
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