Studies With Mice Show Promise for New Huntington’s Disease Treatment

Studies in mice may lead to a new treatment for Huntington’s disease, Harvard researchers report in today’s issue of the journal Nature. The disease is known to be caused by a defect in the gene that is the blueprint for a protein called huntingtin, but scientists are not sure how the defect produces the disease. Mice with the defective gene develop Huntington’s symptoms. Dr. Robert M. Friedlander and his Harvard colleagues have found that blocking the action of another enzyme, called caspase-1, sharply slows disease progression and death in mice. Caspase-1 is involved in apoptosis, or programmed cell death. Friedlander speculates that drugs blocking the effects of caspase-1 could delay disease progression in humans as well.

Compiled by Times medical writer Thomas H. Maugh II