Posted on July 31, 2001 at 10:00 a.m.
BIRMINGHAM, AL — UAB (University of Alabama at Birmingham) investigators are hoping an experimental drug for Huntington’s disease will prove effective in reducing symptoms of the disease, a hereditary brain disorder marked by progressive loss of motor function and thought processes.
A clinical trial of the drug olanzapine is under way at the Huntington’s Disease Clinic at Children’s Hospital in Birmingham. “This trial is the first to fully study the effects of the drug on its ability to help Huntington’s patients walk and control movement,” says Dr. Leon Dure, UAB associate professor of pediatrics and director of the clinic.
Olanzapine, approved by the Federal Drug Administration for the treatment of several psychiatric conditions, has been observed to help the motor difficulties common to most Huntington’s patients. “Anxiety and depression are frequently seen in Huntington’s patients,” Dure says. “Physicians treating those symptoms with olanzapine have seen improvement in motor function.”
Dure says the possibilities of finding successful treatments for Huntington’s are very promising. “This is a hereditary disorder, and we understand its genetic basis fairly well,” Dure says. “Real advances in treatment are looming on the horizon.”
The Huntington’s Disease Society of America recently named the Huntington’s Clinic at Children’s Hospital, the only such clinic in Alabama, as a Huntington’s Disease Center of Excellence.
More than a quarter of a million Americans have Huntington’s or are at risk of inheriting the disease from an affected parent. The disease affects as many people as hemophilia, cystic fibrosis or muscular dystrophy.
Early symptoms of Huntington’s Disease include depression, mood swings, forgetfulness, clumsiness, involuntary twitching and lack of coordination. As the disease progresses, concentration and short-term memory diminish and involuntary movements of the head, trunk and limbs increase. Walking, speaking and swallowing abilities deteriorate. Eventually the person is unable to care for him or herself. Death follows from complications such as choking, infection or heart failure.
The disease typically begins in mid-life, between the ages of 30 and 45, though onset may occur as early as the age of two. Children who develop the juvenile form of the disease rarely live to adulthood. It affects males and females equally and crosses all ethnic and racial boundaries. Each child of a person with Huntington’s has a 50-50 chance of inheriting the fatal gene. Everyone who carries the gene will develop the disease. In 1993, the Huntington’s Disease gene was isolated and a direct genetic test developed which can accurately determine whether a person carries the gene.