Doctors can’t do much for patients with a rare, aggressive form of Lou Gehrig’s disease, even though researchers are confident they have pinpointed its genetic cause. But now with help from a clever device that’s supposed to deliver RNA-silencing drugs where they couldn’t go before, Carlsbad, CA-based Isis Pharmaceuticals is hopeful it may have found new way to treat the deadly neurodegenerative disease.
This treatment, ISIS-SOD1RX, which Cambridge, MA-based Genzyme has an option to co-develop, is being prepped for its first clinical trial later this year for patients with an aggressive form of amoytrophic lateral sclerosis (ALS). The treatment uses the typical Isis approach, known as antisense, in which the drug is designed to shut down the RNA that enables the production of disease-causing proteins.
The treatment also could blaze a trail for a new mode of delivering antisense drugs for a range of neurological disorders, including ALS, Parkinson’s, and Huntington’s disease. The delivery technology is both scientifically interesting and poses big potential business implications for Isis (NASDAQ: [[ticker:ISIS]]).
Isis has never been able to pursue these major diseases because antisense molecules are too big to pass through the blood-brain barrier when they are delivered by conventional injection under the skin, which allows the drug to circulate throughout the body. To work around that problem, Isis developed a reformulated version that could be delivered through infusion with a spinal tap device. If this technique works in the upcoming trial, it could offer a potential new approach for a subset of patients with ALS, which is diagnosed in a total of 5,600 new patients each year in the U.S.
“We’re going into a patient population with no other therapies, and it’s an aggressive form of the disease with a life expectancy of less than one year,” says Frank Bennett, Isis’ senior vice president of research. “What excites me is that we have an opportunity to help those patients. We’re optimistic.”
This form of ALS is considered a good test case for the new delivery technology because
