January 15, 2024 (Arlington, VA) – The Annals of Clinical and Translational Neurology has published new findings showing that Qalsody (tofersen), a genetically targeted treatment for SOD1-ALS, has the potential not only to stabilize the disease but also to restore lost function. These results, gathered in a real-world setting, represent a monumental shift in the expected outcomes of ALS treatment and offer hope that we are closer to a future where effective treatments are available to all people living with ALS.
Significance of Real-World Results
“For decades, neurologists and scientists have thought that disease-modifying therapies for ALS would either slow down or stop further progression of the disease,” said Kuldip Dave, Ph.D., senior vice president of research at the ALS Association. “Qalsody is proving that improvement in function is not only possible, it’s happening. The fact that these data were collected during standard of care treatment in real-world settings further validate that functional recovery is real and significant.”
The study followed seven people with SOD1-ALS treated with Qalsody at Washington University in St. Louis from November 2021 to February 2024. All participants showed either stabilization or slight improvement in function, as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R). Neurofilament light (NfL) levels, a biomarker of disease progression, declined significantly, and participants also demonstrated gains in muscle strength and functional independence.
Global Evidence Supporting Antisense Therapy
This research builds on growing evidence from Europe, where long-term studies in Italy and Germany have also shown stabilization and improvement among people with SOD1-ALS treated with Qalsody. These findings reinforce the promise of antisense therapy not just for SOD1-ALS but for the broader ALS community.
In 2004, the ALS Association was the first to fund research into ALS-specific antisense oligonucleotide (ASO) technology, investing over $1.3 million in groundbreaking studies that laid the foundation for the development of Qalsody.
This early commitment supported preclinical studies, safety trials, and the first-in-human phase 1 trial, which established SOD1 as a viable therapeutic target. “When we first invested in the antisense technology that led to Qalsody 20 years ago, it was a gamble. Something like this had never been tried in the neurodegenerative disease space before,” Dr. Dave said. “Now, Qalsody is changing the way we think about the future of ALS treatment because we know that improvement in function—recovery—is possible. This is not just hope for people living with SOD1-ALS, but for the entire ALS community.”
In 2023, Qalsody became the first genetically targeted therapy approved for ALS, thanks in part to the ALS Association’s efforts, including a united push from the ALS community for FDA accelerated approval.
Hope for All People Living with ALS
The ALS Association remains committed to supporting further advancements in research, advocating for access to treatments, and ensuring that all people living with ALS can benefit from innovative therapies like Qalsody.
View the study on PubMed
About the ALS Association
The ALS Association is the largest ALS organization in the world. The ALS Association funds global research collaborations, assists people with ALS and their families through its nationwide network of care and certified clinical care centers, and advocates for better public policies for people with ALS. The ALS Association is working to make ALS a livable disease while urgently searching for new treatments and a cure. For more information about the ALS Association, visit our website at www.als.org.
About ALS
Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord. Over the course of the disease, people lose the ability to move, to speak, and eventually, to breathe. The disease is always fatal, usually within five years of diagnosis. Few treatment options exist, resulting in a high unmet need for new therapies to address functional deficits and disease progression