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 amyotrophic lateral sclerosis (ALS) and also regulatory T cells (Tregs) as they impact the rate of disease progression. A fumaric acid that enhances Treg levels and suppresses proinflammatory in T cells is dimethyl fumarate.1 Prior research suggest infusions of expanded autologous Tregs combined with IL-2 injections may improve Treg suppressive functions in persons with ALS and slow the progression rate of the disease.2 Thus, treatment advances in gene therapy and neuroinflammation may show a promising benefit in this patient population.

The annual 2023 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, held March 19-22, in Dallas, Texas, showcased several topics that highlighted recent scientific and clinical advances for ALS. Stanley H. Appel, MD, presented in a session at the conference on the advances in ALS, specifically, he spoke about the favorable results of a pilot clinical trial that combined the suppression of proinflammatory macrophages with the enhancement of regulatory T lymphocyte.3

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.

Click here for more coverage of MDA 2023.

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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