Advancing ALS Through Gene Therapies, Early Intervention, and Multidisciplinary Care: Brian Lin, PhD

WATCH TIME: 6 minutes

"At the MDA, we’ve seen gene therapy advance for a number of our diseases like Duchenne muscular dystrophy, for example, and it’s great to see that ALS is starting to enter the picture for the gene therapy landscape.”

In the United States, May is annually recognized as ALS Awareness Month, making it a time dedicated to increasing the public’s understanding of amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease.¹ Throughout the month, organizations such as the Muscular Dystrophy Association (MDA) host events and campaigns aimed at educating the public about the disease, supporting those impacted by the condition, and raising funds for continued research in the field. In MDA’s nationwide awareness campaign for the month, the organization is bringing together the voices of patient advocates and experts including Brooke Eby, Stanley Appel, MD, and more, to share their stories in ALS.

In its new release of the awareness campaign, the MDA noted that it is focused on the ALS Better Care Act to better support those diagnosed with essential multidisciplinary care. Furthermore, the organization reported that partnerships with others such as International Association of Fire Fighters, Dutch Bros, Rawlings, and more, will help to drive donations for the mission during the month. For over 75 years now, MDA has invested more than $178 million to date in ALS research such as for the development of gene therapies and supported families with a national network of 150 MDA Care Centers, including 47 MDA/ALS Care Centers.²

In honor of the awareness month, NeurologyLive^® spoke with Brian Lin, PhD, research portfolio director at the MDA, who outlined key developments in ALS research, with a focus on genetic and molecular therapies such as tofersen (Qalsody; Biogen), CRISPR-based tools, and gene therapies under investigation. During the conversation, Lin also emphasized MDA’s commitment to funding high-risk, early-stage research and nurturing the next generation of investigators. He noted in the interview that MDA's national clinic network delivers comprehensive multidisciplinary care, aiming to improve outcomes and quality of life for individuals with ALS.

REFERENCES
1. Ruffo P, Traynor BJ, Conforti FL. Advancements in genetic research and RNA therapy strategies for amyotrophic lateral sclerosis (ALS): current progress and future prospects. J Neurol. 2025;272(3):233. Published 2025 Feb 26. doi:10.1007/s00415-025-12975-8
2. Muscular Dystrophy Association Launches National ALS Awareness Campaign to Accelerate the Search for a Cure. News Release. Muscular Dystrophy Association. Published April 29, 2025. Accessed May 14, 2025. https://www.mda.org/press-releases/mda-launches-national-als-awareness-campaign-to-accelerate-the-search-for-a-cure