Therapeutic Advances in Neurological Disorders最新文献

{"title":"Neuromyelitis optica spectrum disorders.","authors":"Mohammad Ali Sahraian, Kazuo Fujihara","doi":"10.1177/17562864261430493","DOIUrl":"https://doi.org/10.1177/17562864261430493","url":null,"abstract":"","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"19 ","pages":"17562864261430493"},"PeriodicalIF":4.1,"publicationDate":"2026-03-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13039564/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147609748","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A new, glycoengineered form of anti-CD20 monoclonal antibody, in treatment of multiple sclerosis.","authors":"Krzysztof Selmaj, Ralf Gold, Igor Selmaj, Marcin P Mycko","doi":"10.1177/17562864261434868","DOIUrl":"https://doi.org/10.1177/17562864261434868","url":null,"abstract":"<p><p>B-cell depletion with anti-CD20 monoclonal antibody (mAb) represents a novel and highly effective treatment for patients with multiple sclerosis. Although all approved anti-CD-20 mAbs for multiple sclerosis (MS) treatment, ocrelizumab, ofatumumab, and ublituximab, target the same molecule on the B-cell surface, some differences in their molecular structure translate into important distinctions in their mechanisms of B-cell depletion. Differences between an antibody-dependent cell-mediated cytotoxicity (ADCC) versus a complement-dependent cytotoxicity (CDC) in anti-CD20 mAbs mechanism of action correspond to deeper B-cell depletion as well as better tolerability. Glycoengineering of the Fc portion of ublituximab with reduced fucosylation enhances affinity to Fc gamma receptor IIIa (FcγRIIIa) on natural killer (NK) cells. This molecular modification of ublituximab exhibits significantly higher ADCC activity in relation to CDC in B-cell depletion mechanisms. In addition, glycoengineering of ublituximab reduces the importance of the FcγRIIIa 158V/F polymorphism, which influences the effectiveness of B-cell depletion. In the ULTIMATE I and II studies, ublituximab showed a significant reduction in annual relapse rate versus teriflunomide in relapsing MS patients and strikingly reduced active and new/enlarging MRI lesions. Ublituximab also showed efficacy on the disease progression and increased disability improvement in the extended 5-year observation study. The advantage of ublituximab also correlates with good tolerability and reduced infusion-related reactions. Ublituximab infusion, 1 h, is significantly shorter in comparison to other intravenous (IV) preparations of anti-CD20 mAbs. Thus, ublituximab provides better convenience to MS patients as well as saves the time of healthcare providers.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"19 ","pages":"17562864261434868"},"PeriodicalIF":4.1,"publicationDate":"2026-03-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13039554/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147609719","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Outcomes of mechanical thrombectomy in acute stroke due to primary embolic middle cerebral artery M2 occlusions: a comparison of late versus early windows.","authors":"Huan Wang, Deren Wang, Elena Zapata-Arriaza, Marta Aguilar-Pérez, Asier de Albóniga-Chindurza, Henry Antonio Andrade Ruiz, Joan Montaner, Alejandro González García","doi":"10.1177/17562864261434562","DOIUrl":"10.1177/17562864261434562","url":null,"abstract":"<p><strong>Background: </strong>Current guidelines consider mechanical thrombectomy (MT) reasonable for M2 segment middle cerebral artery (MCA) occlusions within 6 h of symptom onset, yet the impact of delayed intervention in this population remains unclear.</p><p><strong>Objectives: </strong>This study compared effectiveness and safety outcomes of MT performed in late (>6 h) versus early (⩽6 h) treatment windows in patients with acute primary embolic M2 occlusions.</p><p><strong>Design: </strong>We retrospectively analyzed patients with primary embolic M2 occlusions treated with MT within 24 h of onset from the ARTISTA (A Registry for Thrombectomy In Stroke Therapy from Andalusia) registry (2017-2024) in Seville-Huelva, Spain.</p><p><strong>Methods: </strong>Outcomes compared between late and early treatment groups included good outcome (90-day modified Rankin Scale (mRS) 0-2 or return to prestroke mRS), futile recanalization (FR; 90-day mRS >2 despite successful recanalization), successful recanalization (modified Thrombolysis in Cerebral Infarction ⩾2b), and symptomatic intracranial hemorrhage (sICH). Multivariable logistic regression and inverse probability of treatment weighting (IPTW) analyses were performed to evaluate associations between treatment window and MT outcomes.</p><p><strong>Results: </strong>Among 524 patients (median age 76, 48.3% women), 207 (39.5%) underwent MT in the late window. Late treatment showed numerically lower rates of good outcome (58.9% vs 64.4%) and higher rates of FR (37.8% vs 32.5%) and sICH (3.4% vs 3.2%), though none were statistically significant (all <i>p</i> ⩾0.211). Successful recanalization rates were nearly identical between the late and early windows (95.7% vs 95.6%; <i>p</i> = 0.970). IPTW-adjusted analyses also found no significant differences in good outcome (adjusted odds ratio (aOR), 0.82; 95% confidence interval (CI), 0.39-1.73; <i>p</i> = 0.600) or FR (aOR, 1.19; 95% CI, 0.56-2.50; <i>p</i> = 0.653) at 90 days.</p><p><strong>Conclusion: </strong>In this multicenter real-world cohort, MT performed beyond 6 h showed effectiveness and safety outcomes comparable to early-window intervention in patients with primary embolic MCA M2 occlusions.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"19 ","pages":"17562864261434562"},"PeriodicalIF":4.1,"publicationDate":"2026-03-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13033886/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147593950","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Economic value of anti-CD20 therapies in multiple sclerosis: a systematic review of cost-effectiveness studies.","authors":"Johannes Heinemann, Marco Zirkel, Oliver Schoeffski, Rick Dersch, Heinz Wiendl","doi":"10.1177/17562864261426816","DOIUrl":"10.1177/17562864261426816","url":null,"abstract":"<p><strong>Background: </strong>Disease-modifying therapies (DMTs) have transformed multiple sclerosis (MS) care but are associated with substantial costs. Among high-efficacy DMTs, anti-CD20 antibodies are widely used, yet their economic value, particularly compared with different classes of DMTs, has not been comprehensively synthesized.</p><p><strong>Objective: </strong>To systematically review evidence on the cost-effectiveness of anti-CD20 antibodies compared with other DMTs or best supportive care (BSC) in MS.</p><p><strong>Data sources and methods: </strong>We searched <i>PubMed</i>, <i>Embase</i>, <i>Web of Science</i>, and <i>International Network of Agencies for Health</i> Technology <i>Assessment</i> (July 2025) for cost-effectiveness analyses comparing rituximab, ocrelizumab, ofatumumab, or ublituximab with other DMTs or BSC. Two reviewers independently screened studies, extracted data, and assessed reporting quality using the Consolidated Health Economic Evaluation Reporting Standards 2022 checklist. All costs were converted to 2024 US dollars and adjusted for inflation.</p><p><strong>Results: </strong>Of 92 records screened, 20 studies met inclusion criteria. Analyses were conducted in diverse but mostly high-income regions, most commonly using Markov models with long time horizons. Anti-CD20 antibodies were dominant (more effective, less costly) or cost-effective in the majority of studies, particularly when compared with platform therapies. In contrast, comparisons with other high-efficacy DMTs yielded more heterogeneous results, with immune reconstitution therapies more cost-effective in several studies. Reporting quality was generally high, although patient involvement, assessment of heterogeneity, and a health economic analysis plan were rarely addressed.</p><p><strong>Conclusion: </strong>These findings support the use of anti-CD20 antibodies as an economically reasonable option in many healthcare settings, particularly when compared with platform therapies, while underscoring that cost-effectiveness relative to other high-efficacy treatments is context-dependent. The lack of data from low- and middle-income countries and limited transparency in Health Technology Assessment reports represent major limitations. Future research should prioritize context-specific evaluations and promote full disclosure of economic data to strengthen the evidence base for clinically and economically informed reimbursement decisions in MS.</p><p><strong>Trial registration: </strong>The study was prospectively registered with PROSPERO (ID: CRD420251109958).</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"19 ","pages":"17562864261426816"},"PeriodicalIF":4.1,"publicationDate":"2026-03-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13033870/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147593952","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Insights into adherence to cladribine tablets in the real world in multiple countries: results from patient support programs.","authors":"Jiwon Oh, Mavis Ayer, Raed Alroughani, Samar Farouk, Mounir Khoury, Sabrina de Souza, Michela Bossolasco, Kate Morgan, Laura Negrotto, Tracey Quinn, Amir Boshra, Joseph Youssef, Murad Al-Naqshbandi, Kelly MacDonald, Meritxell Sabidó, Jeffrey M Muir, Berenice Silva","doi":"10.1177/17562864261426842","DOIUrl":"10.1177/17562864261426842","url":null,"abstract":"<p><strong>Background: </strong>Patient support programs (PSP) help initiate and maintain disease-modifying treatment (DMT) for people with multiple sclerosis (PwMS). adveva^® (worldwide), Alcura Healthcare (Netherlands), and MS LifeLines^® (US) are nurse-pharmacy-led PSPs that collect clinical information, with consent, on PwMS receiving cladribine tablets (3.5 mg/kg over 2 years followed by a 2-year treatment-free period per product label).</p><p><strong>Objective: </strong>To evaluate initiation and completion of short courses of cladribine tablets treatment in Years 1 and 2 for PwMS and the frequency of subsequent treatment.</p><p><strong>Design: </strong>Retrospective, multinational, observational study using real-world data from PSPs.</p><p><strong>Methods: </strong>Clinical data from PSPs in Australia, Canada, Gulf, Latin America, Near East, Netherlands, UK, and US (December 2017-December 2023) included prior DMT use, cladribine tablets initiation and completion (Years 1 and 2), and subsequent treatment after Year 2 for those with ⩾48 months' follow-up.</p><p><strong>Results: </strong>Across all PSPs, 22,082 PwMS initiated cladribine tablets (Australia, <i>n</i> = 799; Canada, <i>n</i> = 3665; Gulf, <i>n</i> = 258; Latin America, <i>n</i> = 1854; Near East, <i>n</i> = 86; Netherlands, <i>n</i> = 681; UK, <i>n</i> = 2320; US, <i>n</i> = 12,418), of whom 70.9%-81.3% were female (mean age 34.2-49.1 years). Most were DMT-experienced (Australia, no contribution to analysis; Canada, 100%; Gulf, 52.7%; Latin America, 57.7%; Near East, 86.0%; Netherlands, 81.4%; UK, 30.0%; US, 79.5%) with oral DMTs most commonly used (36.5%-69.3%). In PwMS with ⩾18 months' follow-up, ⩾82.8% initiated Year 2 treatment at a mean of 12.4 (±3.8) to 13.9 (±3.5) months after initiating Year 1 treatment. Of those commencing Year 2 treatment, ⩾93.6% completed it. Additional treatment with cladribine tablets beyond 48 months was administered in a small proportion of PwMS: Canada, 5.6%; Latin America, 4.3%; Netherlands, 3.8%; and UK, 1.8%.</p><p><strong>Conclusion: </strong>Cladribine tablets adherence was high, with most PwMS completing short-course cladribine tablets treatment in Years 1 and 2. Of those with ⩾48 months' follow-up, only a small proportion received additional cladribine tablets treatment.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"19 ","pages":"17562864261426842"},"PeriodicalIF":4.1,"publicationDate":"2026-03-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13033868/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147593971","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Novel homozygous <i>SYNE1</i> missense variant in late onset autosomal recessive cerebellar ataxia 1: a case report.","authors":"Igor Selmaj, Nastassja Himmelreich, Krzysztof Selmaj","doi":"10.1177/17562864261433620","DOIUrl":"10.1177/17562864261433620","url":null,"abstract":"<p><p>SYNE1 ataxia is a rare representative of autosomal recessive hereditary cerebellar ataxias (ARCAs). It was originally described in the Beauce region of Quebec as a pure cerebellar form of ataxia. Later, more complex phenotypes with a wide range of extra-cerebellar neurological and non-neurological dysfunctions were discovered in other geographical locations. SYNE1 deficiency was predominantly linked to nonsense and frameshift variants; however, missense variants in the gene <i>SYNE1</i> have also been described as causative for this type of ataxia. We here describe a case of an adult female patient with a phenotype of progressive ataxic features over 15 years, combined with spastic paraparesis. Non-neurological symptoms were not present. The family history was negative, and the case was classified as sporadic. However, the parents of the patient were related to both grandmothers, being cousins. DNA was sequenced using the Illumina HiSeq/Nova Seq system, and the variant c.23413A>G; p.Arg7805Gly in the gene <i>SYNE1</i> was found in a homozygous state (NM_033071.4; NP_149062.2). This missense variant has not been described in the literature and is not listed in the relevant databases. This homozygous substitution resulted in an amino acid change, arginine for glycine, in a highly conserved region of the <i>SYNE1</i> gene. According to ACMG/ACGS guidelines, the criteria PM2+PM3_supporting+PP3 were applied, which classified the substitution c.23413A>G; p.Arg7805Gly as \"a variant of uncertain significance.\" The calculated effect of this substitution is mostly pathogenic based on several in silico prediction programs. The presence of spastic paraparesis grades this case as an ataxic complex syndrome. This is the first SYNE1 ataxia patient of Polish origin with a novel missense variant described with full clinical and MRI data.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"19 ","pages":"17562864261433620"},"PeriodicalIF":4.1,"publicationDate":"2026-03-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13033058/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147581990","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Comparative effectiveness of anti-CD20 therapies and S1P receptor modulators in late-onset multiple sclerosis: real-world evidence from the MSBase registry.","authors":"Andrea Surcinelli, Tomas Kalincik, Izanne Roos, Emanuele D'Amico, Jeannette Lechner-Scott, Serkan Ozakbas, Pavel Hradilek, Dana Horáková, Marta Vachova, Ivan Panzera, Stefano Ruzza, Maria Grazia Piscaglia, Oliver Gerlach, Jose Eustasio Meca-Lallana, Gabriel Valero-López, Allan G Kermode, Marzena J Fabis-Pedrini, Julie Prevost, Radek Ampapa, Suzanne Hodgkinson, Francois Grand'Maison, Samia J Khoury, Nevin A John, Marek Peterka, Jana Houskova, Eva Recmanova, Zuzana Rous, Vahid Shaygannejad, Raed Alroughani, Jens Kuhle, Gregor Brecl Jakob, Pierre Grammond, Francesco Patti, Anneke Van der Walt, Helmut Butzkueven, Matteo Foschi","doi":"10.1177/17562864261430084","DOIUrl":"10.1177/17562864261430084","url":null,"abstract":"<p><strong>Background: </strong>Late-onset multiple sclerosis (LOMS), defined by symptom onset after age 50, is increasingly recognised as a distinct clinical entity. Evidence comparing disease-modifying therapies (DMTs) in this subgroup remains limited.</p><p><strong>Objectives: </strong>To compare clinical outcomes of anti-CD20 monoclonal antibodies and sphingosine-1-phosphate receptor modulators (S1PRMs) in LOMS.</p><p><strong>Design: </strong>Multicentre, observational cohort study based on real-world data from an international multiple sclerosis registry.</p><p><strong>Methods: </strong>We analysed data from the MSBase registry, including relapsing-remitting LOMS patients treated with anti-CD20 therapies (ocrelizumab, ofatumumab, rituximab) or S1PRMs (fingolimod, ozanimod, siponimod, ponesimod) for ⩾6 months. Primary outcomes were annualised relapse rate (ARR), Expanded Disability Status Scale (EDSS) change, confirmed disability worsening (CDW), progression independent of relapse activity (PIRA), and PIRA without MRI activity (PIRMA). Analyses used inverse probability of treatment weighting (IPTW). Causal forest and best linear projector (BLP) models explored effect modification.</p><p><strong>Results: </strong>After weighting, 347 patients (median age 53.7 years; 69% female; median follow-up 6.9 years) were included. No significant differences were found for ARR, EDSS change, CDW, PIRA, or PIRMA. Exploratory analyses suggested greater anti-CD20 benefit in patients with earlier onset (⩽55 years), shorter disease duration (⩽2 years from diagnosis), and lower disability (EDSS < 3).</p><p><strong>Conclusions: </strong>In this real-world LOMS cohort, no statistically significant differences were observed between anti-CD20 and S1PRM therapies. Exploratory analyses suggested anti-CD20 may be associated with better outcomes in selected subgroups; these findings are hypothesis-generating.</p><p><strong>Trial registration: </strong>Not applicable.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"19 ","pages":"17562864261430084"},"PeriodicalIF":4.1,"publicationDate":"2026-03-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13033882/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147594905","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Psoriasis under B-cell depleting therapies in multiple sclerosis: a retrospective multicenter analysis.","authors":"Patricia Kirschner, Franz F Konen, Franziska Axhausen, Ulas Ceylan, Erda Bucak, Romy Baumgart, Lars Masanneck, Stefan Gingele, Kerstin Steinbrink, Sven G Meuth, Ralf Gold, Stephanie Wolff, Simon Faissner, Thomas Skripuletz, Steffen Pfeuffer, Marc Pawlitzki","doi":"10.1177/17562864261427169","DOIUrl":"10.1177/17562864261427169","url":null,"abstract":"<p><strong>Background: </strong>B-cell depleting therapies (BCDT), including ocrelizumab, ofatumumab, and ublituximab, are highly effective disease-modifying therapies for multiple sclerosis (MS). Several case reports have raised concerns about new-onset or exacerbation of psoriasis under BCDT.</p><p><strong>Objectives: </strong>This article aims to analyze clinical characteristics, treatment courses, and outcomes of MS patients who developed or experienced worsening of psoriasis during BCDT.</p><p><strong>Design: </strong>This retrospective, multicenter analysis included patients from four German university hospitals (Düsseldorf, Hannover, Bochum, Giessen).</p><p><strong>Methods: </strong>We retrospectively screened 3228 MS patients under BCDT between 2020 and 2024 for development of psoriasis or an exacerbation of a known psoriasis. Clinical data, including Expanded Disability Status Scale, Psoriasis Area and Severity Index scores, treatment regimens, and comorbidities, were analyzed.</p><p><strong>Results: </strong>Among 3228 patients treated with BCDT, 7 developed new-onset psoriasis and 10 showed exacerbation of preexisting psoriasis. The median time to psoriasis onset or worsening was 13 months (3-83 months) under continuous treatment with BCDT. Topical therapies were effective in most cases, but a change of MS treatment or initiation of psoriasis-specific immunotherapies, including the interleukin-17A-antibody secukinumab, was required in four patients.</p><p><strong>Conclusion: </strong>Psoriasis onset or worsening during BCDT is rare. While most cases are manageable with standard psoriasis treatments, severe cases may necessitate therapy adjustments. The potential immunological interplay between MS and psoriasis treatment warrants further investigation.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"19 ","pages":"17562864261427169"},"PeriodicalIF":4.1,"publicationDate":"2026-03-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13010001/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147514965","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The prospects of CAR T-cell therapies in changing the therapeutic algorithm of neurologic autoimmunities.","authors":"Marinos C Dalakas","doi":"10.1177/17562864261429570","DOIUrl":"10.1177/17562864261429570","url":null,"abstract":"<p><p>T cells, genetically modified to express chimeric antigen receptors (CAR T), successfully used in hemato-oncologic malignancies, are showing promising and sustained benefits in refractory autoimmune neurological diseases, including Myasthenia Gravis, stiff-person syndrome, neuromyelitis optica, myositis, autoimmune neuropathies, and multiple sclerosis. Several reported patients with a steadily progressive disease and evolving disability unresponsive to available therapies, including rituximab and new biologics, after 2-3 months of treatment with CARs targeting CD19-positive, antibody-secreting, long-lived plasma cells, and plasmablasts exhibit impressive, long-lasting, and drug-free clinical improvements with the potential for immune reset shifting immunity to a healthy state without the need for continuing more immunotherapy cycles. The review discusses what the unmet needs are with the present neuroimmunotherapeutics pointing out the disease stage and patient subsets for which CAR T-therapy is most suitable highlighting that CAR T should be applied in the early stages of disability development when patients reach early-active/refractory status rather than waiting for very late disease progression when neurological deficits might be irreversible. The future trajectory of CAR T cells is also described as a promising means destined to change the present therapeutic algorithm in all neuro-autoimmunites, even offering a promising path toward a cure, pointing out that, in contrast to currently approved biologics that selectively target one immunoregulatory factor, CD19 CAR T cells exert effects even beyond B cells, cross the blood-brain barrier and lymphoid tissues, and expand as \"living cells\" to memory cells ensuring sustained long-term benefits. Key therapeutic uncertainties and practicalities are however highlighted, including the exact duration of CAR T-cell therapy-induced drug-free remissions, logistical challenges, economic limitations, and the need for extensive collaborative efforts with experts in specialized clinical centers.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"19 ","pages":"17562864261429570"},"PeriodicalIF":4.1,"publicationDate":"2026-03-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13009801/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147514880","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Testing for neural antibodies in autoimmune encephalitis: who, what, where, when, why, and how.","authors":"Shaza Almweisheer, Kamala Sangam, Ola Ismail, Adrian Budhram","doi":"10.1177/17562864261429181","DOIUrl":"https://doi.org/10.1177/17562864261429181","url":null,"abstract":"<p><p>In recent decades, neural antibody testing has emerged as a cornerstone of autoimmune encephalitis (AE) diagnosis. Commercially available assays to detect these antibodies are now in widespread use, resulting in dramatically increased test accessibility and reduced turnaround times. However, there remains heterogeneity in testing approach and pitfalls related to test ordering as well as diagnostic test performance, which may vary across laboratories depending on their testing methodologies or algorithms. This creates the potential for both false-negative and false-positive results, which can lead to patient misdiagnosis if clinicians are not familiar with both the strengths and limitations of this testing in practice. In this narrative review, we discuss the approach to patient selection for antibody testing, importance of serum and cerebrospinal fluid testing using appropriately handled specimens, potential limitations of local versus reference laboratory testing, optimal timing of testing, benefit of testing to patient management, and diagnostic performance of different test methodologies and algorithms ( the \"who, what, where, when, why, and how\" of neural antibody testing in AE), with the aim of providing a practically useful framework for clinicians who order this testing.</p>","PeriodicalId":22980,"journal":{"name":"Therapeutic Advances in Neurological Disorders","volume":"19 ","pages":"17562864261429181"},"PeriodicalIF":4.1,"publicationDate":"2026-03-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12988283/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147469237","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}