Rejuvenating Neurons to Halt ALS Progression
Innovative gene therapy techniques that rejuvenate motor neurons offer promising new avenues in the fight against ALS, potentially delaying disease onset and progression.
Recent breakthroughs suggest that restoring motor neurons to a youthful state could be a promising strategy in combating amyotrophic lateral sclerosis (ALS). Researchers from Columbia University Irving Medical Center, led by Hynek Wichterle and Emily Lowry, have demonstrated in animal models that gene therapy aimed at reprogramming neurons can delay ALS symptoms and improve neuronal resilience.
Initially, the concept faced skepticism, with doubts about whether adult neurons could be safely rejuvenated without loss of function. However, their latest study published in Nature Neuroscience shows that by reactivating specific developmental transcription factors—ISL1 and LHX3—adult motor neurons can be returned to a more immature, youthful gene expression profile. This process increased the neurons' resistance to ALS-related damage and slowed disease progression in mice.
The therapy involved using a viral vector to deliver genes targeting the motor neurons, a method developed within their lab. While replicating this approach in humans presents challenges, the team is investigating how these factors work to discover druggable targets that might mimic the effects without gene therapy.
ALS predominantly affects adults over 55, gradually impairing movement, speech, and breathing as motor neurons die. Interestingly, motor neurons in individuals with genetic mutations linked to ALS do not degenerate for decades, indicating that youthful neurons are inherently more resilient.
Their approach differs from traditional reprogramming methods, such as the Yamanaka factors, which risk making neurons too young to perform mature functions. Instead, Wichterle and Lowry's method temporarily reactivates developmental programs during neuronal maturation, providing a balanced rejuvenation.
Understanding the mechanisms behind this rejuvenation could open doors to treatments for other neurodegenerative diseases like Parkinson's and Alzheimer's. The hope is that future therapies will leverage these findings to restore neuronal resilience and slow or halt disease progression.
The study, titled "Embryonic motor neuron programming factors reactivate immature gene expression and suppress ALS pathologies in postnatal motor neurons," is available in Nature Neuroscience (2025). Source: https://medicalxpress.com/news/2025-08-neurons-young-als.html
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