Coming Soon: New Huntington's Treatment

By improving the brain's natural protective response to Huntington's disease, researchers from Stanford University Medical Center have been able to ease the uncontrollable tremors and extend the lives of animals afflicted with the disease. Previous research shows Huntington's patients' brains become clogged with clumps of abnormal proteins called aggregates. Lead author Lawrence Steinman, M.D., from SUMC, believed preventing the proteins from clumping into aggregates could control the disease. Dr. Steinman and colleagues injected mice that had a neurological disease similar to Huntington's with a compound called cystamine. After treatment, the mice had fewer tremors and abnormal movements. Also, lifespan was increased by 20 percent in these mice. However, the amount of aggregates remained unchanged.

From this unexpected finding, researchers compared the brains of mice treated with cystamine with untreated mice. Researchers found the cystamine-treated mice have elevated expressions of three genes, which are known to play a protective role in the brain. The same genes are also found in the brains of humans. Authors conclude that in patients with Huntington's disease, the brain unsuccessfully tries to protect itself against the disease. They also suggest cystamine may be a potential treatment for patients with Huntington's disease. Dr. Steinman says: "Before trying this with humans, we will search for even more effective and specific compounds. On the other hand, this is a horrendous, fatal disease. So, we will have to see at what pace it will be applied to humans."

SOURCE: Nature Medicine, 2002;8:143-149

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