University of Maryland School of Medicine researchers are recruiting people for two multi-center studies that may result in new treatments for Huntington's disease (HD), an inherited, untreatable, lethal neurological disorder. The studies, funded by the National Institutes of Health, are designed and conducted by the Huntington Study Group, an international alliance of clinical investigators and scientists. The University of Maryland Medical Center is one of the institutions in the group.
In one study, called the Prospective Huntington At Risk Observational Study (PHAROS), people with a family history of Huntington's who are, therefore, at risk for the disease, will be examined periodically to determine the earliest signs of the disease, but will receive no experimental drugs or treatments.
"The Huntington's Study Group has been running the PHAROS project for several years with limited funding, but just recently, the NIH recognized the importance of this kind of research through its support," says William J. Weiner, M.D., principal investigator in the PHAROS trial at the University of Maryland, and professor and chairman of the Department of Neurology at the University of Maryland School of Medicine. "We hope PHAROS will yield the precise data we need to develop more effective medications to treat Huntington's disease."
"PHAROS is an unusual, ground-breaking study," says Karen E. Anderson, M.D., co-investigator and assistant professor of psychiatry and neurology at the University of Maryland School of Medicine. "In previous studies, investigators knew in advance which volunteers carried the Huntington 's gene. In this study, the person's genetic status will remain unknown. This will remove the chance of investigative bias and make it possible to focus on discovering the earliest possible signs or symptoms of the disease, so we can treat or prevent them in the future, as new medications are discovered."
PHAROS is recruiting 1,000 people at 43 centers in the United States and Canada between the ages of 26 and 55 who are at risk for HD but have not undergone genetic testing. PHAROS hopes to determine how many people at risk for Huntington's will actually develop signs of the illness over three years. The study also addresses the earliest signs of the illness: when do symptoms start and what factors influence the age at which a person carrying the HD gene develops the illness. The study will also attempt to determine the accuracy of the evaluations researchers use to detect the onset of HD.
Volunteers will be asked to submit a blood sample to be tested for the HD gene. The sample may be used for future research, but both the study participants and the researchers will never be told the results of these DNA tests. Every nine months, the volunteers will be examined and evaluated, using standard tools to rate the impact of the illness on movement, mental capacity, mood and behavior.
A second study, called Minocycline Dosing and Safety in Huntington's Disease (MINO), for patients diagnosed with early Huntington's disease, tests the dosing levels and safety of an antibiotic, minocycline, commonly used to treat acne. Minocycline has extended survival and delayed disease progression in rodent models of HD. Some of the MINO volunteers will be given minocycline in varying strengths while the rest will receive pills with no active ingredients.
Huntington's disease is a degenerative brain disorder that slowly impairs the person's ability to walk, think, talk and reason, until he or she becomes totally dependent upon others for care. Symptoms typically begin between ages 30 and 45, although 5 to 10 percent of HD carriers develop symptoms prior to age 20. It typically progresses for about 15 to 25 years after onset of symptoms. Death follows from complications such as choking, infection or heart failure. Approximately 30,000 people in the United States have clinical symptoms of HD, with an additional 150,000 at risk to develop the disease. HD affects as many people as hemophilia, cystic fibrosis or muscular dystrophy.
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