The Motor Neuron Center is focused on an urgent human need.
Patients with motor neuron diseases—spinal muscular atrophy (SMA) in infants and children and amyotrophic lateral sclerosis (ALS) in adults—develop progressive paralysis. These diseases are currently incurable and often lead to death.
Although ALS and SMA are triggered by different mechanisms, many of which remain unknown, they have in common a progressive degeneration and death of motor neurons, the nerve cells responsible for muscle contraction.
The Motor Neuron Center engages brilliant scientific minds in a translational approach to the study of the motor neuron. By taking new leads and tools from motor neuron biology, and testing and applying them to animal models of disease, as well as to clinical research in people, the Motor Neuron Center facilitates the speedy transfer of research and preclinical findings to patients in the hope of finding effective therapy.
The Motor Neuron Center creates synergistic, large-scale programs for target discovery and functional analysis in an intellectual, collegial and stimulating environment.
Columbia University offers an unparalleled location for this initiative because it currently houses, in close proximity, world-class research and clinical groups. These include scientists engaged in the study of motor neuron development, relevant aspects of stem cell biology and animal models of both ALS and SMA. These scientists work with internationally renowned clinicians experienced in clinical trials for both diseases, eager to feed into their programs the latest findings from basic research and able to provide advice about the feasibility of therapeutic approaches for humans.