Yale researchers test drug for Wilson Disease in clinical trial

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A copper ring, known as Kayser Fleischer ring, appears in the Descemet's membrane of the cornea of a Wilson disease patient. (Image by Dr. Joel Geffin)

Yale School of Medicine associate professor Dr. Michael Schilsky is leading a clinical trial to test the dosing and safety of a drug for Wilson disease, a rare genetic disorder. The multicenter, multinational trial is the first in over a decade to test the compound known as WTX101.

“Yale is one of three sites within the United States, and at present, we’ve enrolled and treated the most patients in the world,” said Schilsky. The other sites include the University of Michigan; University of California-Los Angeles; Heidelberg University; Institute of Psychiatry and Neurology, Warsaw, Poland; and University of Vienna.

Patients with Wilson disease have a genetic mutation that prevents the body from properly metabolizing copper. When copper builds up in organs, such as the liver and the brain, it can cause liver disease, neurological damage, and even death. The phase 2 clinical trial will test whether WTX101 can lower and control copper levels in patients, preventing worsening of the disease and disability.

The version of WTX101 to be tested is a more stable form of the compound tetrathiomolybdate than researchers have investigated in the past, said Schilsky. If the trial is successful, the researchers could move on to phase 3 testing of the drug for efficacy as initial treatment and maintenance therapy.

Yale is designated a Center of Excellence for the care of patients with Wilson disease.

The research is supported by Wilson Therapeutics.

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Ziba Kashef: ziba.kashef@yale.edu, 203-436-9317