A new study co-authored by an Oklahoma Medical Research Foundation scientist suggests that a once-controversial drug might extend the lives of patients with amyotrophic lateral sclerosis (ALS), the deadly neurological disease also known as Lou Gehrig’s disease.
Kenneth Hensley, Ph.D., a scientist in OMRF’s Free Radical Biology and Aging Research Program, co-authored the study with researchers at the Cornell University Medical College in New York City. The study, published in this month’s Journal of Neuroscience, indicates that the drug thalidomide might slow the progression of the disease known for striking the legendary first baseman of the New York Yankees.
In the past, thalidomide has been associated with birth defects and was withdrawn from the market for decades after women gave birth to severely deformed babies. However, the new study suggests that thalidomide and its derivative, lenalidomide, shut down production of messenger proteins thought to be crucial to progression of ALS, a fatal neurological condition with no known cure.
The new study showed that in animal studies, treatment with thalidomide increased their lifespan and enhanced their motor performance.
“This is a significant breakthrough,” said Hensley. “It offers hope to patients who currently face an extremely bleak treatment landscape.”
More than 30,000 Americans—most between the ages of 40 and 70—suffer from ALS, which attacks motor neurons in the brain and spinal cord. The disease results in the gradual loss of motor function and is always fatal.
Currently, there is only one drug approved by the Food and Drug Administration for ALS. That drug, Riluzole, adds an average of only three months to patients’ lives. Physicians are now conducting clinical trials of thalidomide in the United States and Europe to determine if treatment is safe and well tolerated in conjunction with Riluzole.
“Previous concerns about thalidomide stemmed from birth defects,” said Hensley. “But pregnancy is not an issue for women suffering from ALS.” He also noted that thalidomide is now used to treat some forms of arthritis.
“A quarter-century ago, who would have imagined that a drug best known for causing birth defects might one day help treat a deadly neurodegenerative disease or joint pain?” said Hensley. “These new results underscore why researchers continually need to examine and re-examine problems both old and new.”
The research was funded by grants from the Muscular Dystrophy Association, The Amyotrophic Lateral Sclerosis Association and the National Institutes of Health.
About OMRF:
Celebrating its 60th birthday in 2006, OMRF (www.omrf.org) is a nonprofit biomedical research institute dedicated to understanding and curing human disease. Its scientists focus on such critical research areas as Alzheimer’s disease, cancer, lupus and cardiovascular disease. It is home to Oklahoma’s only member of the National Academy of Sciences.
