BCM team investigates early signs, treatments of Huntington's
Identifying the first indicators of Huntington's Disease could improve treatment options for the disorder, according to researchers at Baylor College of Medicine in Houston.
The BCM team is following at-risk patients to track these symptoms, as well as conducting clinical trials of drugs that could eventually offer help to patients.
"It's helpful to establish what the earliest signs of the disease are because that might be important as we design neuroprotective treatments," said Dr. Joseph Jankovic, director of the Parkinson's Disease Center and Movement Disorders Clinic at BCM. "Eventually, we hope to have drugs that will slow down the disease, and these studies will identify some of the earliest signs to look for."
Huntington's Disease is a neurodegenerative disorder, which causes 30,000 Americans to gradually loose their mental abilities as well as physical control of their bodies.
In one study, Jankovic and his colleagues are following patients who, based on a DNA test, know they will eventually develop the disease, or patients at risk who show minimal symptoms. The researchers hope to identify the first signs of the disease in order to help diagnose and treat future patients. In a similar study, the investigators are looking for early warning signs in people who have a 50 percent chance of developing the disease because one of their parents was diagnosed with it.
Currently there are no drugs that prevent Huntington's Disease, but there are therapies that lessen the symptoms. One sign of the disease is the presence of irregular and involuntary movements called chorea. There are drugs that prevent chorea by blocking dopamine receptors, but these have side effects that include another form of involuntary movement called tardive dyskinesia.
Researchers at BCM are conducting trials on a drug called tetrabenazine, which treats chorea by decreasing the amount of dopamine in the brain but doesn't cause tardive dyskinesia. The trials are part of the process to get the drug approved by the U.S. Food and Drug Administration. "Reducing chorea is clearly a desirable goal in the treatment of Huntington's Disease," said Jankovic. "Tetrabenazine is the drug that we think is the drug of choice for chorea."
Through these and other clinical and research studies, "hopefully we'll have a much better understanding of what the huntingtin protein is doing and how to prevent damage by the protein using therapeutic interventions," said Jankovic.