First Medication for Huntington's Chorea Approved

Huntington's disease is a fatal, inherited neurodegenerative disorder marked by disabling chorea. Chorea is a symptom of the disease, manifesting in spasmodic, uncontrollable movements that become disabling. The FDA has now approved the medication tetrabenazine for treatment of chorea offering hope to thousands. Tetrabenazine calms the jerky, uncontrollable movements of the body by its action on amines in the brain. Ninety percent of people with Huntington's disease suffer with chorea, so improvement in their symptoms offers dramatic improvement in their quality of life.

Tetrabenazine does not slow progression of the disease, which affects most people in middle age. Prestwick Pharmaceutical, Inc., a privately held company in Washington D.C., owns the rights to develop and sell tetrabenazine in the US. The company specializes in medications treating chronic CNS disorders. Tetrabenazine has been used in Europe for thirty years.

Xenazine®

Labels: Huntington's

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Unraveling the Mysteries of Neurodegenerative Diseases

In a previous post, Understanding Stem Cells and their Potential, we discussed the devastating neurodegenerative disease, Huntington's Disease. Woody Guthrie, a popular American folk singer, was diagnosed with the illness in the 1950's and died in 1967 after 17 years of hospitalization. My daughter's middle school class is using Healthline to research and write papers about a variety of diseases. My kid chose Huntington's Disease, riveted by the agonizing story of Carol Carr, elderly mother and caregiver, who shot and killed her two sons, diagnosed with the illness. Her adult sons' father, their grandmother, their aunt and an uncle had all inherited the disease and Ms. Carr had witnessed their slow deterioration, caring for them. The uncle had committed suicide when he realized his fate. Ms. Carr herself had health problems (heart disease, herniated discs, kidney disease); they were a blue collar family. The sons she killed had an unsuccessful suicide attempt, once they were in the early phases of the disease but before being admitted to a skilled nursing facility. Out of the mouth's of babes (my daughter); "The horrible thing about Huntington's is that you don't just get sick and then die, it goes on and on and on." It is unnerving to contemplate that a folk hero like Guthrie spent the last years of his life in state mental hospitals.

The State of Georgia "tempered justice with mercy." They did not want to convict Carr of a double murder, but they also argued that the courts could not just condone the situation, sympathetic though it might be, "because others might follow suit, who don't want to be bothered, or just want the insurance money." Ms. Carr pleaded guilty to a lesser charge of assisted suicide. As Lee Sexton, Ms. Carr's attorney opined, "...Georgia and other states that do not have a euthanasia statute are in dire need of one...I would be prosecuted for not euthanizing a dog in...[a similar state]." Humans should have the same right to die as they choose.

NIH News reports that researchers at the Mayo Clinic are looking at faulty DNA repair as a possible link in the onset in Huntington's. The interesting thing about the disease is that onset is delayed, usually until middle age, which has scientists looking to find ways to stop or slow down the process. Principal researcher Cynthia McMurray PhD., professor of pharmacology at Mayo Clinic states the gene, called huntingtin, produces a protein that has a destructive effect in the brain. Everyone knows it is a progressive disease, but no one knows why. Her research supports the idea that an "extra segment" on the huntingtin gene expands over time in nerve cells, which don't divide. The extra segment grows inside the cells over time because the DNA repair enzymes, which remove oxidative lesions (the byproducts of oxygen we breathe), can't keep up. Researchers are excited to work with some of these DNA repair enzymes and their role in oxidative lesions, and ultimately destructive proteins. They may find the keys to treatment of some of the devastating neurogenerative diseases that afflict an aging population: Alzheimer's and Parkinson's Diseases.

Thank you E Wayne, for use of the photo, Woody Guthrie.

Labels: assisted suicide, Huntington's, right to die, stem cells, woodie guthrie

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