The Sean M. Healey & AMG Center for ALS at Mass General Brigham awarded the seventh annual Sean M. Healey International Prize for Innovation in ALS to the team of researchers who discovered that nucleotide-repeat expansions can cause ALS, revealing disease mechanisms and therapeutic targets.

The team consists of four researchers across four institutions:

  1. Nancy Bonini, PhD, University of Pennsylvania, USA
  2. Aaron Gitler, PhD, Stanford University, USA
  3. Rosa Rademakers, PhD, VIB-UAntwerp Center for Molecular Neurology, Belgium and Mayo Clinic, USA
  4. Bryan Traynor, MD, PhD, MMSc, FRCPI, National Institute on Aging, National Institutes of Health, USA

The prize was presented to the team during the 36th International Symposium on ALS/MND in San Diego, California by Merit Cudkowicz, MD, MSc, Director of the Healey & AMG Center and Executive Director of the Mass General Brigham Neuroscience Institute.

Drs. Rademakers, Traynor, Bonini, and Gitler are being recognized for discovering that nucleotide-repeat expansions can cause ALS. Drs. Rademakers and Traynor independently identified the most common genetic cause of ALS and frontotemporal dementia (FTD) as the hexanucleotide repeat expansion in the C9ORF72 gene. Similarly, Drs. Gitler and Bonini discovered that the repeat expansion in the ataxin-2 gene also causes ALS, and in so doing, revealed insight into disease mechanisms. The discoveries by Rademakers, Traynor, Bonini, and Gitler have profoundly impacted ALS research, offering therapeutic targets and unexpected insights into disease mechanisms.

“We are deeply grateful to the Sean M. Healey & AMG Center for ALS at Mass General Brigham for recognizing our team’s research,” the awardees say. “This prize underscores the importance of discovery-driven research and international collaboration. We hope that our findings will open up new possibilities for understanding the causes of ALS. Inspired by this recognition, we will redouble our efforts to uncover the mechanisms of this devastating disease and to develop new strategies for effective therapies.”

“The discoveries made by these remarkable scientists have transformed the field and brought hope to finding treatments for ALS,” states Dr. Cudkowicz. “We are excited to see future strategies and insights from this team, and we are grateful for their commitment to investigating causes and mechanisms in ALS."

The annual Sean M. Healey International Prize for Innovation in ALS is a global prize celebrating excellence in research for a team of investigators who catalyze exceptional discoveries leading to a transformative advance in therapy development in ALS. Learn more about this $50,000 USD prize and how to nominate a team.

For more information about the Sean M. Healey & AMG Center for ALS, please visit the website.

About the Sean M. Healey & AMG Center for ALS at Mass General Brigham

At the Sean M. Healey & AMG Center for ALS at Mass General Brigham, we are committed to bringing together a global network of scientists, physicians, nurses, foundations, federal agencies, and people living with ALS, their loved ones, and caregivers to accelerate the pace of ALS therapy discovery and development.

Launched in November 2018, the Healey & AMG Center, under the leadership of Merit Cudkowicz, MD and a Science Advisory Council of international experts, is reimagining how to develop and test the most promising therapies to treat the disease, identify cures and ultimately prevent it.

With many clinical trials and lab-based research studies in progress right now, we are ushering in a new phase of ALS treatment and care. Together, we will find the cures.