A study published in the Lancet Neurology found that Neurologix's gene therapy NLX-P101 eased some Parkinson's disease symptoms in a mid-stage trial.
The gene therapy is injected deep into the brain, delivering genetic material directly into bilateral subthalamic nuclei (STN), a key brain region involved in motor function. When compared to the control arm that received a fake surgery, the treatment arm achieved previously defined moderate-to-large clinically-meaningful symptom improvements. The technique seemed to improve motor control and reduce jerking motions, according to Bloomberg. Importantly, the drug was safe and well-tolerate over a six month period.
Neurologix CEO Clark Johnson said the results "strongly support" the company's plans to move ahead with a Phase III trial of NLX-P101 in subjects with Parkinson's disease. The company plans to submit a late-stage trial protocol later this year.
"This is the first Phase I study conducted under a rigorous randomized, double-blind, sham-controlled surgical design to conclusively demonstrate that gene therapy can be effective for neurological diseases," noted senior author and co-principal investigator Andrew Feigin. "This confirms our Phase I results and indicates that NLX-P101 may provide a safe, effective and minimally invasive treatment option for patients with PD."
Once hailed as the next big thing in drug development, the gene therapy field faced a major setback when serious adverse events, including death, resulted from some treatments. Yet a report last year found that 189 companies are developing gene therapies, and 354 U.S. studies are underway to explore the therapy for a variety of ailments.
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