New Hope for ALS Patients

The letters ALS written on three block surrounded by a stethoscope

In an extraordinary breakthrough researchers from Western University, led by Dr. Michael Strong, have uncovered a potential path toward curing amyotrophic lateral sclerosis (ALS), a devastating neurodegenerative disease. This monumental discovery, supported by decades of research and philanthropy from the Temerty Foundation, marks a significant milestone in the fight against ALS.

ALS, also known as Lou Gehrig’s disease, progressively impairs nerve cells responsible for muscle control, leading to severe muscle wastage, paralysis, and ultimately death. The average life expectancy post-diagnosis is a mere two to five years. For years, the medical community could offer little more than hope to ALS patients and their families. Now, however, Strong and his team have provided a tangible reason for optimism.

The team’s research, published in the journal Brain (if you like reading studies, click here), reveals that targeting an interaction between two specific proteins in ALS-affected nerve cells can halt or even reverse the disease’s progression. This discovery centers on the interaction between the protein TDP-43, which forms toxic clumps in nerve cells, and RGNEF, a protein with functions opposite to TDP-43. They identified a fragment of RGNEF, named NF242, that can neutralize the toxic effects of TDP-43. This interaction significantly reduces nerve cell damage and prevents cell death.

Dr. Strong emphasized the long journey to this discovery, saying, “It’s been 30 years of work to get here; 30 years of looking after families and patients and their loved ones when all we had was hope. This gives us reason to believe we’ve discovered a path to treatment.”

The breakthrough was achieved through various models, including fruit flies and mice, where the approach notably extended lifespan, improved motor functions, and protected nerve cells from degeneration. The next ambitious goal is to translate these findings into human clinical trials within the next five years, fueled by a $10 million investment from the Temerty Foundation.

The Temerty Foundation, established by James Temerty and Louise Arcand Temerty, has a long-standing commitment to ALS research at Western University. This latest investment brings the family’s total contribution to neurodegenerative disease research at Western to $18 million. James Temerty expressed his optimism, saying, “Finding an effective treatment for ALS would mean so much to people living with this terrible disease and to their loved ones. Western is pushing the frontiers of ALS knowledge, and we are excited for the opportunity to contribute to the next phase of this groundbreaking research.”

Dr. Strong’s dedication, combined with the visionary support of the Temerty Foundation, has accelerated progress in ALS research. Western University President Alan Shepard praised this collaboration, highlighting its transformative potential for ALS patients worldwide.

Dr. John Yoo, dean of Schulich Medicine & Dentistry, celebrated this pivotal moment, noting, “With Dr. Strong’s leadership, our continued investment in the best tools and technology, and the visionary support of the Temerty Foundation, we are thrilled to be heralding in a new era of hope for patients with ALS.”

This groundbreaking discovery not only offers new hope for ALS patients but also underscores the profound impact of sustained philanthropic investment in medical research. As the journey towards clinical trials begins, the promise of an effective ALS treatment is now within reach, potentially transforming the lives of thousands afflicted by this debilitating disease.