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ALS Alert Newsletter

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Dec 9
2020

Packard awards new research grant to fund study axon degeneration

Note: This grant was made possible through a generous gift from the Bruce Edwards Foundation.

The Packard Center has recently awarded a $50,000 research grant to Michael Coleman, Ph.D.  from the University of Cambridge for a project entitled: Programmed axon death: a preventable axon degeneration mechanism in some ALS patients.

The first structures lost in ALS are nerve endings and nerve-muscle contact sites called neuromuscular junctions (NMJs), causing paralysis. There is no treatment yet to prevent this. However, scientists have identified a mechanism of nerve and NMJ degeneration that is fully preventable in animal models. What's more, they have found evidence suggesting it contributes to ALS, at least in some subsets of patients. Different people have different propensities for activating this nerve degeneration mechanism and we have methods to determine who is at highest risk. We will test whether high-risk individuals are more common among ALS patients than in controls and use stem cell technology in Answer ALS samples to test for activation of the preventable nerve degeneration mechanism in patient-derived nerve cells. This will help deliver the right drugs to the right subgroup of patients and may have wider therapeutic implications for other forms.

“Understanding the role of axon degeneration in ALS is very important.  Through this work, Coleman and his colleagues will investigate whether some patients with ALS may be more at risk for a form of axonal degeneration that is amenable to treatment," noted Emily Baxi, PhD, Packard Center's Executive Director.  "Professor Coleman is a leader in the field of axonal degeneration and we are thrilled to support the application of his work to ALS.”

“We are delighted to join the Packard Center network to extend our ongoing work in mechanisms of axon loss in ALS," noted Coleman. "The collaborative and highly interactive style of the network is a great fit for our way of doing research and we look forward to both contributing to and learning from the experience."