How is Wilson's Disease Being Treated?

Wilson's Disease is a very treatable condition. With proper therapy, disease progress can be halted and oftentimes symptoms can be improved. Treatment is aimed at removing excess accumulated copper and preventing its reaccumulation. Therapy must be lifelong. Patients may become progressively more sick from day to day, so immediate treatment can be critical. Treatment delays may cause irreversible damage.

The newest FDA-approved drug is zinc acetate (Galzin™). (To link to a page about Galzin, CLICK HERE.) Galzin is know as "Wilzin" in Europe.  Zinc acts by blocking the absorption of copper in the intestinal tract. This action both depletes accumulated copper and prevents its reaccumulation. Zinc's effectiveness has been shown by more than 30 years of considerable experience overseas. A major advantage of zinc therapy is its lack of side effects.

Other drugs approved for use in Wilson's Disease include penicillamine (Cuprimine, Depen) and trientine (Syprine). (To link to a page about Cuprimine and Syprine, Click Here.)  Both of these drugs act by chelation or binding of copper, causing its increased urinary excretion.

Tetrathiomolybdate is another chelating drug that is under investigation for initial treatment of Wilson's Disease. Thus far, it has not caused the neurological worsening often associated with penicillamine and even with trientine. 

Patients with severe hepatitis or liver failure may require liver transplant. Patients being investigated or treated for Wilson's Disease should be cared for by specialists in Wilson's Disease or by specialists in consultation with their primary physicians.

Stopping treatment completely will result in death, sometimes as quickly as within three months. Decreasing dosage of medications also can result in unnecessary disease progression.