Giving Day—New Hope for ALS
For generations of patients, a diagnosis of ALS—amyotrophic lateral sclerosis, also known as Lou Gehrig's disease— has been a devastating one. ALS causes the death of motor neurons, specialized nerve cells that transmit signals from the brain and spinal cord to muscles. As the disease progresses, the death of these cells leads to muscles weakening and wasting away. For nearly a century, the prospect for ALS patients has been grim.
Columbia scientists like Hynek Wichterle are changing that. As Co-Director of the Clare Tow Center for Motor Neuron Biology and Disease at VP&S, Wichterle and his colleagues are making progress in our understanding of the origins of diseases like ALS. It has been one of the most stubborn challenges in medicine for decades -- and finally, the paradigm is shifting.
"Scientists in the Motor Neuron Center are working tirelessly to understand why motor neurons are susceptible to degeneration, and to develop new drugs and genetic interventions, to help patients fight this currently untreatable disease," says Wichterle, who holds joint appointments in the Departments of Pathology & Cell Biology and Neuroscience at VP&S. One of the obstacles in ALS research has been that the disease attacks adult motor neurons, while scientists have been able to grow only immature neurons from patient-derived stem cells in the laboratories. Recently, Dr Wichterle' s lab discovered an intricate molecular clock controlling neuronal maturation, revealing a roadmap to make mature neurons in a dish. Such improved models of ALS will finally give Columbia scientists the chance to get these cells to give up their secrets, to discover what causes their degeneration, and how to prevent it.
One drug they have developed in collaboration with Dr Brent Stockwell's laboratory has proven successful in the lab, protecting the motor neuron cells from toxic stressors that mimic insults observed in ALS patients. This has led to a successful Phase 1 clinical trial, for what could become the first meaningful intervention for patients diagnosed with sporadic ALS.
Wichterle recognizes that he is in the right place to make this kind of unprecedented progress. "Columbia is exciting, because of the close collaborations between basic scientists, clinicians, and medicinal chemists. The clinicians who see ALS patients here provide critical insights into the disease mechanisms and access to valuable clinical material. Our hope is that we can close the loop by bringing new therapeutic opportunities to their patients."
Dr. Wichterle's story is not singular. He is one of many Columbia scientists doing pioneering work, and the support of our friends has never been more important. We hope that you will consider making a gift to further the work of these extraordinary researchers and clinicians.
Dr. Wichterle is featured in the University’s Giving Day video highlighting important priorities at Columbia.
Make a gift to support Dr. Wichterle's work here.
You can learn more about Dr. Wichterle and his lab here.
Learn more about Columbia's Giving Day here.