Clinical Experience and Considerations With ALS Medication Tofersen: Sandeep Rana, MD

WATCH TIME: 3 minutes

"When Qalsody was approved, it was a very exciting moment for the ALS community. It represents a targeted therapy that can slow, or potentially halt, disease progression."

In the past decade or so, there has been an increasing amount of research dedicated to amyotrophic lateral sclerosis (ALS), a devastating and fatal neuromuscular disease that results in loss of muscle function and breathing ability. Riluzole, approved in 1995, was the first-ever drug to reach market that showed a modest ability to extend survival by a few months. More than 2 decades later, edaravone, a small-molecule antioxidant, followed as the next approved medication, offering functional benefits in patients with early stages of the disease.

In 2023, the field experienced a major breakthrough, as the FDA approved tofersen (Qalsody; Biogen) as the first therapy for SOD1 ALS, one of the most common familial forms of the disease. The drug, an antisense oligonucleotide, was approved under the accelerated approval pathway based on reductions in neurofilament light (NfL), a marker of neuroaxonal damage. Several in the field noted this as significant, considering it was the first time NfL had been used as a surrogate biomarker to predict clinical benefit, opening the door for other agents in the pipeline.

Sandeep Rana, MD, a neurophysiologist at Allegheny Health Network, has had recent experience with tofersen in treating patients with SOD1 ALS. In an interview with NeurologyLive®, Rana provided his initial clinical perspectives, explaining how this treatment is designed to slow or halt disease progression, with biomarker evidence already suggesting favorable effects in many patients. Rana, whose treated patients with ALS for decades, discussed the critical role of genetic testing in ensuring timely identification of eligible patients, particularly in community and non-urban settings where access to advanced therapies can be limited.