Isis, Genzyme Use Antisense to Go Where Many Drugs Fail—The Brain

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it involves overproduction of a specific protein called Superoxide dismutase1, which can be toxic to the central nervous system, Bennett says. A traditional small-molecule drug wouldn’t really work if it were made to bind with this enzyme, he says. Isis believes antisense will work better because it will stop the overproduction of the protein in the first place.

Until now, delivery has been the key challenge. Isis scientists have been working on a new formulation that can be used in tandem with new spinal infusion delivery technologies from companies like Medtronic (NYSE: MDT), Johnson & Johnson’s Codman division, and Inset Technologies. The new tools are designed to allow slow, steady infusions of drugs for pain and spasticity, but can be used to deliver other treatments as well, Bennett says. It’s possible that patients can stay on such a system for years, either getting continuous infusions, or possibly intermittent on-off cycles.

If the Isis treatment reaches its goals of safely delivering the antisense drug into the brain, then it could establish a precedent for a couple other delivery-challenged neurology drugs in the pipeline for Huntington’s and Parkinson’s, Bennett says. Those programs, which haven’t yet entered clinical trials, are funded by a couple of well-known patient advocacy groups, the Cure Huntington’s Disease Initiative, and the Michael J. Fox Foundation for Parkinson’s Research. Naturally, the financial prospects of developing drugs for those more common diseases could add up to bigger rewards for Isis as a company.

Despite Isis’ optimism, Bennett sounded like he didn’t want to get too carried away, and emphasized that all the programs are still at early-stages of development: “We’ll keep our fingers crossed,” he says.

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