Thomas O’Halloran (Chemistry and Molecular Biosciences), founding director of the Chemistry of Life Processes Institute, is responsible for the discovery of bis-choline tetra-thiomolybdate, a copper-protein binding agent that safely scavenges excess copper from the body.
This new compound has been licensed and renamed WTX101 by Wilson Therapeutics, a biopharmaceutical company based in Stockholm. The company has successfully completed clinical studies demonstrating the safety and efficacy of the drug for treatment of Wilson Disease. The disease results from a genetic disorder that causes impaired copper transport and excretion resulting in accumulation of free copper in the bloodstream, and ultimately, in damaging accumulations of copper in the liver, brain and other organs. Wilson Disease is fatal if left untreated.
Wilson Disease affects approximately one in every 30,000 people worldwide. WTX101 has received orphan drug designation for the treatment of Wilson Disease in the US and EU. In addition, WTX101 has shown potential as a treatment for several other medical conditions including Amyotrophic Lateral Sclerosis (ALS). WTX101 has received US orphan drug designation in July 2017 for the treatment of ALS.