Miami Children's Research Team to Evaluate Treatments for Huntington's Disease

Published on: 09/22/2004
MIAMI - A Miami Children’s Hospital (MCH) research pharmacologist has been awarded a contract to work with the National Institutes of Health to help identify drugs to prevent the progression of Huntington’s disease (HD), a devastating degenerative brain disorder for which there is presently no effective treatment or cure.

Collin Hovinga, Pharm.D., a research neuropharmacologist with the MCH Brain Institute, is the lead investigator in SET-HD (Systematic Evaluation of Treatments for Huntington’s Disease), which seeks to identify the most promising treatments for Huntington’s disease. The effort is supported by the National Institute of Neurological Disorders and Stroke, a division of the National Institutes of Health, and is funded by the High-Q Foundation, a non-profit organization dedicated to finding and developing treatments for Huntington’s disease.

SET-HD is the first of many efforts of the worldwide initiative launched in 2002 known as the Huntington Project. The Huntington Project brings together the clinical research endeavors for Huntington’s disease that are underway throughout the world in a broad-based approach to find treatments that make a difference in the lives of those affected by HD.

To date, SET-HD has identified approximately 200 drugs or other compounds that have shown potential to modify the progression of the disease. In the current phase, Dr. Hovinga and his colleagues have singled out approximately 45 compounds with the greatest potential. The entire HD community is welcome to participate in Huntington Project and SET-HD by visiting the web site ( to view and nominate experimental treatments to be considered for clinical studies. Eventually they will help establish guidelines for clinical trials to evaluate the effectiveness of the compounds.

“Huntington’s disease is a devastating genetic disorder that destroys the lives of those who inherit it. Through our efforts, we give new hope to families who carry the Huntington disease gene,” said Dr. Hovinga.

The High-Q Foundation is a non-profit organization dedicated to bringing together clinical and basic scientists, individuals and families affected by Huntington’s disease, advocacy groups, and all others in the Huntington’s disease community to find and develop treatments that make a difference in the lives of those affected by Huntington’s disease.

Founded in 1950, Miami Children’s Hospital is the only licensed specialty hospital for children in South Florida. Ranked among the country’s best pediatric hospitals by U.S.News & World Report, Miami Children’s Hospital offers medical care and services for children from birth to age 21. The 268-bed medical facility treats more than 185,000 patients each year and has expertise in all aspects of pediatric medicine.

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