CuATSM

CuATSM (Copper ATSM) is a small synthetic molecule designed to deliver copper to damaged mitochondria, the energy-producing organelles in cells. It is membrane-permeant and can cross the blood-brain barrier. The drug was initially developed in Japan as an imaging agent for hypoxic tissues but has since been investigated for its therapeutic potential in neurodegenerative diseases, particularly amyotrophic lateral sclerosis (ALS) and motor neuron disease (MND). CuATSM acts by delivering copper ions to mitochondria, potentially correcting mitochondrial dysfunction and reducing oxidative stress—both implicated in ALS pathogenesis. Preclinical studies have shown that CuATSM can ameliorate disease phenotypes in mutant superoxide dismutase mouse models of ALS. In human cell studies, it appears to act as a metabolic switch, reprogramming astrocyte metabolism from excessive mitochondrial activity toward increased glycolysis and lactate production, which may support motor neuron survival. Clinical trials have demonstrated safety and tolerability; however, efficacy results remain inconclusive with some post-mortem analyses showing no significant benefit on neuronal preservation or disease markers[1][2][3][4][5][6][8].

Other names

Copper ATSM