Scientists at the Oklahoma Medical Research Foundation have identified a compound that shows promise in treating Huntington’s disease. They are now performing pre-clinical tests to assess whether the chemical slows the progress of the deadly disease of the nervous system.
Led by Kenneth Hensley, Ph.D., OMRF researchers are performing pre-clinical tests using lanthionine ketimine (LK), a chemical product derived from metabolic processes. Early data indicate that LK could stop damage to nerve cells and reduce inflammation, thus delaying the motor function deterioration caused by Huntington’s disease.
“This is very early in the process,” Hensley said. “But this will give us a foundation of data to build on and create a second generation of compounds.”
A neurological disorder with no known cure, Huntington’s disease typically manifests itself by behavioral abnormalities—mood swings and depression—followed by a lack of coordination, involuntary body movements, paralysis and, ultimately, death. The genetic disease affects approximately 30,000 Americans. Among its victims was Woody Guthrie, the legendary Oklahoma-born folk singer.
With a grant that he has received from the Hereditary Disease Foundation, Hensley will test two forms of LK on mice that have been genetically engineered to develop symptoms of Huntington’s. The OMRF researcher will study whether the compound slows the loss of body control that begins early in the disease and also explore the compound’s ability to improve brain function and inhibit neuron destruction.
Although Hensley’s results are still too preliminary to predict whether LK might one day lead to a drug to treat humans, he is hopeful that the work will lead to more potent and effective drugs for Huntington’s disease.
“If we could just slow the rate of progression of Huntington’s, we could turn a 10- or 20-year illness into a 50-year illness,” said Hensley. “People could have decades of productive life. They could watch their children grow up.”
About OMRF
OMRF (omrf.org) is an independent, nonprofit biomedical research institute dedicated to understanding and developing more effective treatments for human disease. Its scientists focus on such critical research areas as Alzheimer’s disease, cancer, lupus and cardiovascular disease.
