Six doses, one patient, and a marker of nerve damage cut in half

Genetic testing found one ALS patient carried a mutation in CHCHD10, a rare cause of the disease. So the Mayo Clinic's N-of-1 Therapeutics Program and the n-Lorem Foundation did something almost unheard of: they screened more than 320 antisense molecules and built a therapy for that one person. After six spinal-fluid doses, the treatment was well tolerated — and neurofilament light, a blood marker of nerve-cell injury, fell by as much as half. Breathing, thinking and movement held steady or improved slightly. "I didn't think I would see this kind of research in my lifetime," said neurologist Björn Oskarsson, senior author of the paper. It's a framework, they say, for doing precision medicine rigorously and equitably. Published in Med.