A new experimental therapy using an approach known as antisense, in which a drug is designed to shut down the RNA that is responsible for the production of disease-causing proteins, is being prepared for a clinical trial in people with a familial form of ALS later this year. The research that resulted in the identification of this antisense drug was first funded by ALSA in 2003, and has been developed for the clinic through an academic/industry partnership. Drs. Don Cleveland, Richard Smith and Timothy Miller, in partnership with Isis Pharmaceuticals in Carlsbad, Calif., initiated experiments in a rat model of ALS to determine whether reducing the amount of SOD1 protein may be beneficial in treating the disease.
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