Using Stem Cells to Improve Motor Function

March 10, 2024

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Understanding ALS Disease Biology to Pursue Repair and Regeneration

Using Stem Cells to Improve Motor Function: Brian Wainger, MD, PhD

When motor neurons are injured, whether due to traumatic injury or neurodegenerative diseases such as ALS, the muscle will no longer contract, and undergoes unrecoverable wasting from disuse. Preventing this degeneration of muscle is the key factor to successful restorative nerve surgery and may lead to improved motor function in those with ALS.

Brian Wainger, MD, PhD, is developing a novel therapy to help improve motor outcomes in nerve injury and ALS in collaboration with Justin Brown, MD in the Department of Neurosurgery. The strategy is to transplant engineered motor neurons that have been derived in the laboratory from human stem cells. Such motor neurons have already shown promise in animal experiments. Motor neurons are injected near the muscle, and the neurons then grow and make connections to thatmuscle. These connections have the potential to maintain muscle health and prevent muscle wasting.

The same approach may yield benefit in those affected by aALS. in two separate ways: first, motor neurons injected into the phrenic nerve can supply the diaphragm, the primary muscle for breathing. By using an implanted cuff electrode around the phrenic nerve, sufficiently strong respiratory function could be maintained without the need for a mechanical ventilator. Second, an increasingly large number of studies suggest that a vicious cycle of motor neuron dysfunction and muscle wasting are responsible for disease progression. When muscle atrophies, it can no longer provide sustenance to nearby motor neurons, which then accelerates motor neuron loss. By maintaining muscle innervation, the same strategy of injecting engineered motor neurons has the potential to reduce motor neuron loss and maintain motor function in ALS.

Brian Wainger, MD, PhD is the inaugural incumbent of the Alexander Healey Endowed Chair in ALS at Mass General, an attending physician nd Assistant Professor in Anesthesiology and Neurology at Harvard Medical School. His clinical expertise spans the intersection of neurology and pain medicine. His research focuses on understanding how the physiological properties of motor and sensory neurons may yield insight into relevant diseases such as ALS and painful neuropathy.