University of Michigan Medical Center April 23 1997
Contact: Kristen Lidke, Media coordinator; (313) 764-2220 Fax: (313) 647-2104 E-mail: [email protected]
U-M forges new treatment for a 'heavy metal' disorder
Dementia. Drooling. Slurred speech. Temper outbursts. Violent tremors. These are just a few of the symptoms of a potentially fatal condition called Wilson's disease, a recessive genetic disorder that interferes with the body's ability to excrete copper. If left untreated, the metal collects in the liver and brain, causing these organs to become severely damaged. Symptoms are often misdiagnosed as hepatitis, substance abuse or Parkinson's disease, thus delaying treatment and increasing the possibility of permanent organ damage.
Researchers at the University of Michigan Medical Center have developed a drug, recently approved by the federal government, that can halt the progression of - and often completely reverse - the symptoms of Wilson's disease. The disorder strikes some 5,000 Americans, usually during their teen-age or young-adult years.
Called zinc acetate and pioneered by George Brewer, M.D., professor of human genetics and of internal medicine at the U-M Medical Center, the drug is now available under the brand name Galzin, which is manufactured by Gate Pharmaceuticals, a division of Teva Pharmaceutical Industries. It works by blocking the absorption of copper from the intestine. It must be taken for the rest of the patient's life to keep copper levels in check and thus prevent disabling symptoms from recurring. The drug produces few or no side effects.
Brewer discovered the benefits of zinc acetate in lowering copper levels by accident about 15 years ago, while working with sickle-cell anemia patients. When he noticed that the drug caused copper deficiency in some of the patients, he tried it among Wilson's disease sufferers and, over time, their symptoms largely disappeared. Since then he has drawn Wilson's disease patients from around the world to the U-M Medical Center's General Clinical Research Center - the only place in the nation that researches this disease and treats large numbers of such patients.
Because Wilson's is considered an "orphan disease" - one that affects fewer than 200,000 people - the recent approval of zinc acetate is especially significant, considering that most drug companies are unwilling to sponsor orphan drugs for market approval due to their lack of profit potential. According to the National Organization of Rare Disorders, of the 5,000 orphan diseases in the United States, only about 140 have approved drug treatments.
"It's exciting to have zinc acetate become an official therapy for Wilson's disease," says Brewer, who credits Gate Pharmaceuticals for its support, as well as the resources of the U-M General Clinical Research Center, from its nurses to its special dietary unit, which allows meticulous control of the amount of copper in each patient's meal.
For more information, media should call Kristen Lidke or Margo Schneidman, U-M Medical Center Department of Public Relations, (313) 764-2220. Patients can call U-M TeleCare, (800) 742-2300, category 6230.
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