Every 90 minutes, someone is diagnosed with amyotrophic lateral sclerosis (ALS), a condition in which motor neurons of the spine degenerate, leading to a lack of control over the body’s most basic functions. Beginning as a subtle weakening of certain muscle groups, ALS can quickly escalate to paralysis, ripping away an individual’s ability to speak, eat, and even breathe. With no cure and limited treatment options, ALS is often seen as a race against time.
But what if that race could be won? What does it take to transform a diagnosis of ALS from a devastating inevitability to a treatable — or even curable — condition? Northeastern researchers are seeking answers by navigating the complex path from understanding the disease to developing a solution.
Here at Northeastern, over a decade of work has already gone into searching for a treatment for familial ALS (fALS) by the Agar Group, led by Dr. Jeffrey Agar. Familial ALS, a genetic form of the disease, makes up about 10–20% of all ALS cases seen today. Dr. Agar’s work addresses the movement issues caused by ALS, particularly by stabilizing proteins affected by genetic mutations. Using mouse models that replicate the mutations seen in human fALS, the group studies disease progression and tests potential therapies in a controlled environment. These models are invaluable in uncovering the mechanisms of motor neuron degeneration and developing targeted treatments.
Northeastern’s Center for Translational Neuroimaging (CTNI), led by Dr. Craig Ferris, complements this research by using mouse and rat models to study other neurodegenerative diseases, such as Parkinson’s and Alzheimer’s. These disorders, like ALS, involve the progressive degeneration of specific neurons, leading to debilitating motor and cognitive impairments. By leveraging advanced imaging technologies such as magnetic resonance imaging (MRI), the CTNI monitors real-time changes in brain structure and function in these models, offering insights into disease progression and potential treatment effects.
This same approach is applied to sporadic ALS (sALS), which accounts for the majority of ALS cases and has no known genetic cause. The use of animal models enables researchers to simulate the neurodegenerative processes seen in sALS, such as inflammation, oxidative stress, and protein misfolding, which can contribute to motor neuron loss. Imaging these changes provides a clearer understanding of how the disease develops and spreads throughout the body.
These preclinical research stages are vital precursors to finding a lasting cure for ALS. By developing effective testing methods in rodent models, researchers can pave the way for human trials, bringing potential treatments closer to real-world application. As these therapies progress, they hold the promise of becoming effective cures for ALS, offering hope to those affected by this devastating disease worldwide.