to help manage symptoms, improve quality of life and pro- long survival, including Food and Drug Administration- approved medications such as riluzole and edaravone. Riluzole, originally approved as Rilutek tablets in 1995, was the first FDA-approved medication for treating ALS in all patients. It acts as a glutamate inhibitor to slow dis- ease progression and extend survival. In 2019, the FDA approved intravenous edaravone, marketed as Radicava, to slow the loss of physical function in ALS. As a neu- roprotective agent, it acts as a free-radical scavenger to reduce oxidative stress. A more convenient oral version, Radicava ORS, was later approved in 2022 to treat adults with ALS, offering the same efficacy as the intravenous formulation in slowing disease progression. A new study has suggested that edaravone, marketed as Radicava and Radicava ORS, could be beneficial to a wider range of patients with ALS than those included in the original trial, Study 19, which led to FDA approval.2 That report in Muscle & Nerve noted that Study 19 used a targeted enrollment enrichment strategy based on post hoc analyses from earlier trials, selecting patients with higher baseline function, more rapid disease progression, and better respiratory status. To evaluate the generaliz- ability of Study 19 results, however, subsequent post hoc analyses assessed the efficacy of edaravone in broader ALS populations. A machine learning-based analysis that retrospectively applied a validated model to data in an earlier trial sug- gested that up to 70% of those participants may have ben- efited from edaravone. A second analysis investigated eda- ravone efficacy in patients from Study 19 with forced vital capacity (FVC) < 80% predicted (%p) at the start of treat- ment and found that both high- and low-FVC subgroups demonstrated reduced ALS functional rating scale-revised decline at 48 weeks when treated continuously with edara- vone, according to the recent review. “These findings support the potential benefit of eda- ravone in a wider range of patients with ALS than those enrolled in Study 19, providing important insights into how clinical trial enrichment strategies may influence perceived efficacy, and underscoring the need for future prospective studies in more diverse ALS populations,” the authors wrote. No matter the pharmacological treatment used, the recent VA study on symptom burden and patients dem- onstrated that the VA’s approach to ALS care is highly comprehensive and coordinated. Veterans receiving care through VA interdisciplinary ALS clinics, especially Centers of Excellence and full team models, reported higher satisfaction and better support with symptom management. One reason, Howard suggested, is that VA’s proactive, team-based model helped ensure early access to services such as equipment, therapy and care coordina- tion, which can make a meaningful difference in the ALS care experience “These findings provide important baseline informa- tion to support ongoing quality improvement efforts across the VA system,” she advised. The results might also serve as a model for simi- lar efforts in other healthcare systems, the researchers recommended. The survey identified several areas for further improve- ment, including education on genetic testing and research and management of non-motor symptoms. Howard noted her team is working with the VA’s Genomic Medicine Service and the National Precision Oncology Program to implement direct ordering of genetic tests in VA neurol- ogy clinics this year. “To expand our future research capacity, we’re partner- ing with community organizations and the VA Office of Research and Development’s Brain Health Coordinating Center,” she explained. “We are also providing ongo- ing education to our clinical network on symptom man- agement treatments and current research opportuni- ties, including recent presentations from the VA Brain Bank, the ALL ALS consortium and the ALS Therapy Development Institute.” The study authors also are affiliated with the Birmingham (AL) VAMC; University of Alabama at Birmingham in Birmingham, AL; Stanford University in Palo Alto, CA; VA Palo Alto (CA) Health Care System; Yale University in New Haven, CT; VA Connecticut Healthcare System in West Haven, CT; and Richard L. Roudebush VAMC in Indianapolis. “I’d like to thank the veterans who shared their feed- back through this survey,” Howard said. “I am continually humbled by the selfless service of the veteran ALS com- munity, which clearly does not stop after diagnosis. With their help, we will keep working to improve care for those living with ALS now and for those who may be diagnosed in the future, until we have a cure.” Veterans can find more information on benefits and clinical care at www.va.gov/health/als.asp. 1 Howard I, Lyerly M, Reimer R, Patwa H, Darling L. Symptom Burden and Care Satisfaction in US Military Veterans With ALS: Results of a National Survey. Muscle Nerve. 2026 Apr;73(4):623-629. doi: 10.1002/ mus.70147. Epub 2026 Jan 20. PMID: 41556400. 2 Brooks BR, Ennist DL, Beaulieu D, Apple S. Generalizability of Edaravone Efficacy. Muscle Nerve. 2026 Feb;73 Suppl 1(Suppl 1):S16-S18. doi: 10.1002/mus.70042. Erratum in: Muscle Nerve. 2026 May;73(5):925. doi: 10.1002/mus.70197. PMID: 41653008; PMCID: PMC12882029. 2 0 2 6 C O M P E N D I U M O F F E D E R A L M E D I C I N E 4 3
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