[2] Bis-choline tetrathiomolybdate has been evaluated in clinical trials in patients with various forms of cancer[3][4][5] and has received orphan designation in the US and EU as a potential therapy against Wilson's disease.

[11] As of November 2014, a Phase 2, multi-centre, open-label study was recruiting newly diagnosed Wilson's disease patients 18 and older to evaluate the efficacy and safety of bis-choline tetrathiomolybdate administration over a 24-week period.

[25][26] The data suggest that bis-choline tetrathiomolybdate can rapidly lower and control toxic free copper levels and improve clinical symptoms in Wilson's disease patients.

[22][23][24] Previous clinical studies with bis-choline tetrathiomolybdate in oncology patients have shown that it can lower and maintain copper levels using a once or twice daily oral dosing.

[4][5] This may be helpful since untreated Wilson's disease may lead to death within several years of the onset of symptoms,[27] and medication use should continue throughout the patient's lifespan.