Sending copper where it is needed most

Copper (Cu) is an essential component of human physiology, and it is indispensable for normal brain development. Cells use Cu in many processes, including respiration, formation of myelin sheath, immune responses, wound healing, and synthesis of neurotransmitters (1). A sophisticated network of Cu-transporting proteins retrieves Cu from dietary sources, transfers Cu across biological membranes, and distributes it within cells and tissues (2). The key component of this network, Cu-transporting adenosine triphosphatase 1 (ATP7A), is inactivated in Menkes disease (MNKD). This causes Cu deficit in the brain, neurodegeneration, and early death. Cu supplementation is ineffective in treating MNKD patients because Cu cannot reach many cellular destinations, especially the brain, without functional transporters. On page 620 of this issue, Guthrie et al. (3) show that a small Cu-binding molecule, elesclomol, can overcome this problem, improving Cu delivery to the brain and alleviating mortality of ATP7A-deficient mice.