Combo Therapy Offers Hope for Krabbe's Disease

Mouse study showed life spans greatly extended in surprise finding

MONDAY, Jan. 15, 2007 (HealthDay News) -- By using a combination of bone marrow transplants and gene therapy, U.S. researchers were able to greatly extend the lives of mice with a fatal neurodegenerative disease that's similar to Krabbe disease in humans.

Krabbe disease, also called globoid-cell leukodystrophy, is an inherited disorder that eventually destroys the brain and nerves of the body.

The results of this study by researchers at the Washington University School of Medicine in St. Louis raise hopes about the possibility of finding a treatment for Krabbe disease and related disorders.

"We had everything we needed to test this combination treatment, so we said, 'Let's just give it a try,'" study senior author Mark Sands, an associate professor of medicine and of genetics, said in a prepared statement.

"Alone, these therapies resulted in a small survival benefit, but with the combination, we were seeing mice with dramatically extended life spans," Sands said.

Mice that received the combination treatment lived an average of twice as long (more than 100 days) than mice that received just one treatment or no treatment. The mice that were given bone marrow transplants and gene therapy also had improved motor skills compared to the other mice.

The study was published in the January issue of Molecular Therapy.

More information

The U.S. National Library of Medicine has more about Krabbe disease.

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