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Huntington's Disease Discovery Could Lead to New Treatments

Area of the brain affected by Huntington's disease

Scientists have discovered how a protein associated with Huntington’s disease, an incurable and lethal disorder, impacts the severity and timing of its symptoms. This discovery could lead researchers to develop new treatments for the disease and similar conditions, such as Alzheimer’s and type 2 diabetes.

Huntington’s disease is currently incurable

About 250,000 people in the United States have Huntington’s disease, a neurodegenerative genetic disease that affects the brain and causes uncontrolled movements, loss of intellectual function, and emotional disturbances. The disease passes from parent to child via a mutation in the normal gene, and children of a parent with Huntington’s have a 50-50 chance of inheriting the disease-causing gene.

At Gladstone Institutes, researchers discovered that one form of a misfolded protein in neurons (cells in the brain that send and receive messages) is the best predictor of whether the neuron will die. In Huntington’s disease, the death of neurons is the key to the condition’s symptoms.

According to Steven Finkbeiner, MD, PhD, professor of neurology and physiology at the University of California, San Francisco, with which Gladstone Institutes is affiliated, and the study’s senior investigator, “we hope our newfound understanding of precisely which misfolded proteins contribute to disease symptoms will speed up drug development for sufferers.” In the case of Huntington’s disease, finding an effective treatment is critical, as currently there are none.

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Huntington’s disease is caused by mutations in the gene that creates a protein called htt (huntingtin). When the mutated gene produces htt, a portion of the protein expands, which distorts the protein’s natural function and structure, resulting in activities that can be toxic.

The findings of this study are exciting because “it will be easier to develop drugs that target the toxic form of htt that causes Huntington’s symptoms,” noted Finkbeiner. Currently, medications used to treat symptoms of Huntington’s include dopamine blockers, which may help reduce abnormal movements and behaviors; drugs such as amantadine and tetrabenazine, which may control movements; and coenzyme Q10, which may slow the course of the disease.

The discovery of the workings of a protein associated with Huntington’s disease could lead to new treatments for this currently incurable disease. Lennart Mucke, MD, director of neurological research at Gladstone, noted that “Dr. Finkbeiner’s powerful and precise approach could enable the development of pharmaceutical treatments for other diseases as well.”

Gladstone Institutes
PubMed Health

Picture credit: Wikimedia Commons