A biotech company developing gene therapies for disorders affecting the brain and central nervous system believes its gene technology could be used to treat Parkinson’s disease.

Neurologix (Fort Lee, New Jersey) has completed its Phase I trial of gene therapy for Parkinson’s disease with what it calls “statistically significant results.”

The study, performed at New York-Presbyterian Hospital/Weill Cornell Medical Center , began in August 2003 using the company’s NLX-P101 technology on 12 patients with severe Parkinson’s disease for whom other therapies were no longer effective. Neurologix presented data from the trial at the 36th annual meeting of the Society of Neuroscience (Washington) in Atlanta earlier this month.

In the gene transfer procedure, using the adeno-associated virus (AAV) vector, doctors injected a therapeutic gene — glutamic acid decarboxylase (GAD) — directly into the subthalamic nucleus which is overactive in Parkinson’s disease. The AAV is a virus that has been used in a variety of clinical gene therapy trials and is the vehicle Neurologix said would be used in all of its first generation products.

The significance of the trial, Matthew During, MD, told Medical Device Daily, is that it demonstrated the safety of the procedure in all 12 patients. During and Michael Kaplitt, MD — both co-founders of Neurologix — conducted the study. The patients were recruited and evaluated by Andrew Feigin, MD, and David Eidelberg, MD, at North Shore University Hospital (Manhasset, New York).

“We are extremely encouraged. Every patient [in the trial] was discharged from the hospital within 48 hours without a single fever and that was extremely encouraging,” During said. “It’s a simple procedure and we believe it will be a significant advantage over existing treatments.”

The National Parkinson Foundation (Miami, Florida) estimates that 4 million to 6 million people worldwide suffer from Parkinson’s disease, and that 60,000 new cases are diagnosed in the U.S. each year, the foundation said, adding to the 1.5 million Americans who have the disease. The condition — characterized by tremor, rigidity and slowness — usually develops after age 60 but about 15% of those diagnosed are under 50.

Although the patients in the trial showed significant improvement, During said, the Phase I trial did not have blinded controls. The next phase, he said, will be a more rigorous trial in which blind controls are used. Neurologix expects the next phase to get underway sometime in 2007, During said.

At one year, all 12 patients as a group demonstrated a clinical improvement of 25% in the Unified Parkinson’s Disease Rating Scale and nine of them showed an average improvement of 37% with five of those patients showing improvement between 40% and 65%.

“This gene therapy trial is particularly unique and the clinical data unusually promising because the treatment was confined to just one side of the brain,” During said.

In its next trial, Neurologix plans to infuse its treatment into both sides of the brain.

“The company is very excited about the results of this trial,” said John Mordock, president/CEO of Neurologix. “We look forward to further validating these results in the next trial and continuing our efforts to develop a significant new treatment for Parkinson’s disease worldwide.”

Neurologix is focused on developing gene therapies for disorders affecting the brain and central nervous system.