A Clinical Preview of 2024 for Neuromuscular Disease: Richard Lewis, MD

WATCH TIME: 4 minutes

“Gene therapy is a promising avenue for various disorders, particularly genetic diseases, where its potential is evident. However, its excitement for immune and inflammatory disorders remains uncertain. The sheer abundance of genetic diseases makes it challenging to predict when specific conditions will benefit from gene therapy. Despite this uncertainty, there is considerable enthusiasm in the scientific community about the potential of gene therapy, and that's truly exciting."

In 2024, those in neuromuscular medicine are anticipating new research updates and pending approvals on potential therapies for certain disorders, including amyotrophic lateral sclerosis, myasthenia gravis, Duchenne muscular dystrophy, chronic inflammatory demyelinating polyneuropathy (CIDP), and others. Neuromuscular diseases, like CIDP, have experienced challenges in the clinical setting with the diagnosis of subtypes of the diseases and prescribing the most effective therapies for patients that improve outcomes.

CIDP is characterized as progressive weakness and reduction in senses of the arms and legs caused by damage to the fat-based protective covering on nerves.¹ Over the recent years, the neuromuscular community has made significant strides to understand the complexities of CIDP, with a greater focus on novel approaches for diagnosis, treatment, and management.

Investigators are continuing their pursuit in 2024 to understand more of the determinants, interactions, and the biological mechanisms of CIDP to discover innovative and improved management strategies. In a recent literature review published in Medicina, researchers established that the destruction of peripheral nerves and nerve roots typifies CIDP and is an autoimmune disease that influences the myelinating constructions of peripheral nervous system. Although several treatments for CIDP show promising results, researchers recommend that treatment decisions be personalized for each patient’s needs.²

Richard Lewis, MD, director of the electromyography laboratory and professor of neurology at Cedars-Sinai Medical Center, sat down in an interview with NeurologyLive® to share his clinical perspective on potential therapies in neuromuscular disease, specifically for patients with CIPD, for 2024. In the conversation, he talked about how neonatal Fc receptor inhibitors and complement inhibition might potentially show promise in CIDP treatment, and some of the challenges they could pose. While gene therapy holds promise for genetic diseases, he also discussed what uncertainties surround its potential effectiveness for immune and inflammatory disorders. Additionally, Lewis talked about how clinicians and patients can navigate the complexity of choices and make informed decisions with the plethora of treatment options available for CIDP.

REFERENCES
1. Patrick MM, Bielling R, Postma G, Trokthi B, Maitland CG. Chronic Inflammatory Demyelinating Polyneuropathy With Reversible Severe Cognitive Impairment and Gastrointestinal Dysfunction. Cureus. 2023;15(11):e49341. Published 2023 Nov 24. doi:10.7759/cureus.49341
2. Tsoumanis P, Kitsouli A, Stefanou C, et al. Chronic Inflammatory Demyelinating Polyneuropathy and Evaluation of the Visual Evoked Potentials: A Review of the Literature. Medicina (Kaunas). 2023;59(12):2160. Published 2023 Dec 13. doi:10.3390/medicina59122160