Therapeutics: ATPase Cu++ transporting β polypeptide (ATP7B)
Patient sample and rat studies suggest the bacterial peptide methanobactin could help treat Wilson's disease, in which ATP7B deficiency results in pathological accumulation of copper in the liver. In hepatocyte mitochondria derived from ATP7B-deficient rats and hepatocyte-like cells differentiated from Wilson patient-derived induced pluripotent stem (iPS) cells, methanobactin decreased copper levels compared with no treatment. In an ATP7B-deficient rat model of Wilson's disease, methanobactin decreased copper levels in hepatocyte mitochondria, liver damage and liver failure, and increased survival compared with the standards of care D-penicillamine and Syprine trientine hydrochloride. Next steps could include formulating methanobactin for oral delivery.
Valeant Pharmaceuticals International Inc. markets Syprine, a chelating compound for removal of excess copper from the body, for Wilson's disease...