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Columbia Receives Up to $15 Million for SMA Research

Columbia University has been awarded up to $15 million from the Spinal Muscular Atrophy (SMA) Foundation. The grant will fund activities by Columbia’s new Center for Motor Neuron Biology and Disease to accelerate the discovery of medical advances for SMA, a devastating genetic disease that is the No. 1 killer of infants and toddlers.

SMA is characterized by the wasting of skeletal muscles due to progressive degeneration of nerve cells in the spinal cord. Loss of function leads to death from respiratory problems; half of its victims die before age 2. The SMA Foundation estimates about 25,000 to 55,000 people have SMA in the U.S., Europe, and Japan. Only palliative care is now available. Research progress in the last decade, however, has made SMA scientists and clinicians optimistic that treatments can be developed in the next few years.

The Center for Motor Neuron Biology and Disease, also referred to as the Motor Neuron Center (, was founded at Columbia in November 2005. It focuses on the biology of the motor neuron and two diseases caused by motor neuron degeneration – SMA and amyotrophic lateral sclerosis (ALS, or Lou Gehrig’s Disease). The center’s mission is to create a cohesive translational research center where lab findings are quickly translated into new treatments and clinical findings are brought into the lab to shape new research directions.

More than 40 leading researchers from numerous disciplines at CUMC and Morningside, including neurobiology, neurology, genetics, pathology, cell biology, physiology, anatomy, chemistry and pediatrics, have converged to form the center. Members include a Nobel Prize winner and three Howard Hughes Medical Institute investigators.

The gift, which will be distributed over five years, is the largest ever made by a private foundation for SMA research. The Center for Motor Neuron Biology and Disease will recruit new investigators to complement existing Columbia expertise.

“The SMA Foundation is a tremendous partner in our work,” said Chris Henderson, Ph.D., co-director of the Center for Motor Neuron Biology and Disease, along with Serge Przedborski, M.D., Ph.D., professor of neurology, pathology and cell biology, and Darryl De Vivo, M.D., the Sidney Carter Professor of Neurology. “We value their ongoing input and collaboration, which provides a new perspective on how to take basic research findings and move them toward the clinic. We hope that this approach will generate both new biological insights and relief for families living with SMA.”