Gene and Cell Therapies Emerge as Front-Runners in Neurology Innovation, with High Expectations in ALS, Parkinson’s, and Neuroimmune Disorders
Strong enthusiasm for novel modalities is tempered by neurologists’ demands for clinically meaningful endpoints and data superiority, according to Spherix Global Insights.
EXTON, PA, Nov. 03, 2025 (GLOBE NEWSWIRE) -- Spherix Global Insights’ latest Special Topix™: Neurology Pipeline Opportunities 2025 (US) service captures forward-looking insights from 79 U.S. neurologists across five key neurological conditions—Amyotrophic Lateral Sclerosis (ALS), Parkinson’s disease (PD), multiple sclerosis (MS), generalized myasthenia gravis (gMG), and chronic inflammatory demyelinating polyneuropathy (CIDP). Key findings reveal that despite a wide range of familiarity with investigational agents, neurologists share a clear message: they are ready to embrace innovation—provided new treatments deliver clinically meaningful outcomes on validated endpoints.
ALS is viewed as the most urgent area for therapeutic advancement, with 82% of neurologists stating that most of their ALS patients remain inadequately managed. Gene and cell-based therapies are seen as the most promising approaches to shift the treatment paradigm, with neurologists highlighting progression-free survival (PFS) as the most valuable benchmark for future therapies. Conceptual enthusiasm runs high, but adoption is expected to depend on clear efficacy signals, ideally demonstrating superiority over existing options.
In Parkinson’s disease, neurologists prioritize improvements in MDS-UPDRS scores and time to confirmed worsening. Early-stage interest is building around novel assets such as Bemdaneprocel (a dopaminergic stem cell therapy) developed by BlueRock Therapeutics and BIIB122/DNL151, a LRRK2 inhibitor co-developed by Biogen and Denali Therapeutics. While the mechanisms are compelling, neurologists remain measured, with uptake contingent on rigorous clinical data and proven long-term benefit.
CAR-T therapies—including Descartes-08 (Cartesian Therapeutics) and KYV-101 (Kyverna Therapeutics)—are gaining traction in the neuroimmune segment, particularly for refractory cases of MS, gMG, and CIDP. Despite the novel approach and strong early efficacy signals, concerns remain around cytokine release syndrome (CRS), long-term safety, cost, and logistical complexity. One neurologist summarized the cautious outlook: “We need to see head-to-head comparisons, a robust safety profile, and meaningful clinical superiority.”
As the neurology pipeline matures, neurologists are increasingly open to combination strategies and multimodal approaches to address historically difficult-to-treat conditions. Even therapies with low initial awareness can gain momentum quickly—if they meet the expectations of specialists who prioritize measurable, practice-relevant outcomes. Spherix’s findings highlight both the opportunity, and the evidence thresholds manufacturers must meet to drive adoption in this evolving, high-stakes therapeutic landscape.
About Special Topix™
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About Spherix Global Insights
Spherix is a leading independent market intelligence and advisory firm that delivers commercial value to the global life sciences industry, across the brand lifecycle.
The seasoned team of Spherix experts provides an unbiased and holistic view of the landscape within rapidly evolving specialty markets, including dermatology, gastroenterology, rheumatology, nephrology, neurology, ophthalmology, and hematology. Spherix clients stay ahead of the curve with the perspective of the extensive Spherix Physician Community.
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NOTICE: All company, brand or product names in this press release are trademarks of their respective holders. The findings and opinions expressed within are based on Spherix Global Insight’s analysis and do not imply a relationship with or endorsement of the companies or brands mentioned in this press release.
Bob Shewbrooks, Neurology Franchise Head Spherix Global Insights 4848794284 Bob.shewbrooks@spherixglobalinsights.com
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