Focus on Gene Therapy and Neuroinflammation in ALS Research: Stanley H. Appel, MD
The director of the Ann Kimball and John W. Johnson Center for Cellular Therapeutics at Houston Methodist Hospital spoke on about advances in ALS research and promising therapies on the horizon at the 2023 MDA conference. [WATCH TIME: 8 minutes]
WATCH TIME: 8 minutes
“Its wonderful to see all the potential therapies that are being now examined, tested, and studied that may have a meaningful outcome for patients with ALS. No group of patients is more worthy and more deserving of this approach. We're very excited about where we stand now, in terms of delivering the studies that will help meet the unmet need.”
Neuroinflammation plays a key role in the pathogenic mechanism in
The annual
At the conference, Appel, director of the Ann Kimball and John W. Johnson Center for Cellular Therapeutics at Houston Methodist Hospital, sat down with NeurologyLive® in an interview to discuss some of the symptomatic therapies that are available in ALS. Appel, director of the Muscular Dystrophy Association ALS Research and Clinical Center and emeritus MDA board member, also talked about the use of gene therapy as an option for patients, and neuroinflammation as the primary pathway of focus with regard to the disease.
REFERENCES
1. Vucic S, Henderson RD, Mathers S, et al. Safety and efficacy of dimethyl fumarate in ALS: randomised controlled study. Ann Clin Transl Neurol. 2021;8(10):1991-1999. doi:10.1002/acn3.51446
2. Thonhoff JR, Berry JD, Macklin EA, et al. Combined Regulatory T-Lymphocyte and IL-2 Treatment Is Safe, Tolerable, and Biologically Active for 1 Year in Persons With Amyotrophic Lateral Sclerosis. Neurol Neuroimmunol Neuroinflamm. 2022;9(6):e200019. Published 2022 Aug 29. doi:10.1212/NXI.0000000000200019
3. Appel S. Novel Treg-Enhancing Therapy Suppresses Neuroinflammation in ALS. Presented at: MDA Clinical & Scientific Conference; March 19-22, 2023; Dallas, TX. Advances in the Understanding in ALS session.
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