On June 8, Neurologix (NASDAQ: [[ticker:NRGX]]) released positive data from a trial of its experimental gene therapy treatment in patients with Parkinson’s Disease. It was the latest in a string of impressive announcements from the Fort Lee, NJ-based company, which has been on a roll since March, when the journal Lancet Neurology published six-month results from the study, a Phase 2 clinical trial involving 45 patients.
Has Wall Street applauded? Not exactly. Neurologix, a perennial penny stock, hasn’t traded over a buck since last December—the June 8 announcement bumped its stock from $0.67 to $0.70 a share. (It’s now at $0.79.) But with its Phase 2 completed, the company needs to raise about $50 million to complete the pivotal Phase 3 study that will be necessary to win FDA approval. It won’t be easy, admits Neurologix co-founder Michael Kaplitt, an associate professor of neurological surgery at Weill Cornell Medical College in New York. “The biggest concern” among investors, he says, “is fear of the unknown.”
Such are the frustrations of being a biotech company that’s trying to pioneer an entirely new approach to treating neurological diseases—and one that involves gene therapy, no less. The basic idea of gene therapy is to correct a dysfunctional disease-causing gene by replacing it with a functional gene that’s inserted into the body. It was once a hot concept in biotech, and a technique that many scientists believed could treat a range of diseases.
Then disaster struck. In 1999, 18-year-old Jesse Gelsinger died after receiving an experimental gene therapy for a rare disease, during a trial being conducted at the University of Pennsylvania. Funding for research into gene therapy around the world became scarce.
But Kaplitt and co-founder Matthew During were undeterred. They formed Neurologix in 1999 around a technique that they believed would ameliorate the risks inherent in earlier gene therapy attempts. One main approach to gene therapy involves packing copies of healthy genes into viruses known as “vectors,” which then transport the genes into cells. Scientists believe Gelsinger’s death was caused by an immune reaction to the vector—not to the gene itself. Neurologix’s brain treatment, which During and Kaplitt first described in a 1994 paper, uses a vector called adeno-associated virus 2, which doesn’t trip an immune reaction in humans.
Kaplitt and During also figured out how to deliver the gene only to the part of the brain that goes bad in Parkinson’s patients. Parkinson’s occurs when there’s an imbalance of two brain chemicals, GABA and glutamate, During explains. That, in turn, causes dopamine-producing cells in the brain to die—resulting in the symptoms characteristic of the disease, including
