The Pipeline project has been tracking Neurologix gene therapy for many years. On many levels, this is a very disturbing event – for the patients, the trial participants, the employees of the company and the researchers that started it. With so many PD trials failing, this was one trial that was actually getting positive results, and they were getting ready for a phase III.
To read the database record for Neurologix’s GAD Gene Therapy, go to www.pdpipeline.org
Click on Therapy Development Database.

It is listed on the Scorecard as Glutamic Acid Decarboxylase (GAD) Gene Therapy (NLX-P101)

Both phase I and II reported positive results and both studies were published in a Lancet journal.
Excerpts from database:
"The results of the phase I trial were published in the June 23, 2007 issue of the journal The Lancet ...
It was announced on Dec. 13, 2007 that the U.S. Food and Drug Administration has granted Fast Track Designation to Neurologix for its experimental gene transfer procedureť to treat advanced Parkinsons disease. Neurologix plans to launch
Phase II studies of its process by early 2008.

On March 26, 2008, during a year end 2007 financial report, CEO John Mordock stated, "We are now completing steps to initiate a Phase 2 clinical trial of our Parkinson's product in the first half of 2008, subject to clearance from the FDA. "

"...It was reported on March 27, 2008 that the FDA approved a Phase II trial, and expected to start recruiting 44 participants in the U.S. during the 2nd quarter of 2008. (BusinessWire 3/27/08)

In December 2009, Neurologix "announced the completion of all planned surgeries in an ongoing Phase 2 clinical trial. The controlled, double blinded, 44 patient Phase 2 trial is investigating the safety and efficacy of a novel non-dopamine approach to restore motor function in Parkinsons patients who are sub-optimally responsive to available drug therapy. The company expects to announce initial efficacy results from the trial in mid-2010. " (Businesswire 12/03/09) The investigators are assessing each of the trial participants over time for treatment effects, with the primary trial endpoint being a clinical assessment of motor function at 6 months using the Unified Parkinson’s Disease Rating Scale (UPDRS). All participants in the trial will also be monitored for safety for 12 months following their gene transfer procedure.

On June 22, 2010, Neurologix, Inc. "announced positive results in a Phase 2
trial of its investigational gene therapy for advanced Parkinson's disease,
NLX-P101. Study participants who received NLX-P101 experienced statistically
significant and clinically meaningful improvements in off-medication motor
scores compared to control subjects who received sham surgery. In the trial,
this benefit was seen at one month and continued virtually unchanged throughout the six month blinded study period...." (Neurologix press release)

Phase II results published in Lancet Neurology, March 2011. "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 co-authors..." (pressrelease)
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We should remain vigilant, as we learn more about the fate of the company , that the trial participants receive follow ups and medical care if needed, that they are given the chance to ask questions and have them answered. This is a good opportunity to show how trial participants should be treated with respect and provided with information. Mistakes have been made in the past. Hopefully we know how to do it better now.
We need to do whatever we can to convince another sponsor to buy the rights to this gene therapy, get the trials completed, and the treatment to the patients, if it proves effective. We don’t need another promising treatment to end up sitting on the shelf.