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Efficacy and safety of autologous BCMA-directed mRNA CAR T-cell therapy in generalized myasthenia gravis: Results from a phase 2b randomized placebo-controlled trial 自体bcma定向mRNA CAR - t细胞治疗广泛性重症肌无力的疗效和安全性：来自2b期随机安慰剂对照试验的结果
IF 6.9 2区 医学
Neurotherapeutics Pub Date : 2025-07-01 DOI: 10.1016/j.neurot.2025.e00672
Christopher M. Jewell Ph.D., Milos D. Miljkovic M.D., Hafsa Kamboh M.D., Andy Stewart, Metin Kurtoglu M.D., Emily English Ph.D.
{"title":"Efficacy and safety of autologous BCMA-directed mRNA CAR T-cell therapy in generalized myasthenia gravis: Results from a phase 2b randomized placebo-controlled trial","authors":"Christopher M. Jewell Ph.D., Milos D. Miljkovic M.D., Hafsa Kamboh M.D., Andy Stewart, Metin Kurtoglu M.D., Emily English Ph.D.","doi":"10.1016/j.neurot.2025.e00672","DOIUrl":"10.1016/j.neurot.2025.e00672","url":null,"abstract":"","PeriodicalId":19159,"journal":{"name":"Neurotherapeutics","volume":"22 4","pages":"Article e00672"},"PeriodicalIF":6.9,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144826779","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Targeting GCPII to promote remyelination in aging peripheral nerve injuries 靶向GCPII促进衰老周围神经损伤的髓鞘再生
IF 6.9 2区 医学
Neurotherapeutics Pub Date : 2025-07-01 DOI: 10.1016/j.neurot.2025.e00651
Yu Su M.D., Ph.D., Meixiang Huang M.D., Ph.D., John Maragakis M.D., Ph.D., Ajit Thomas, Kaitlyn Huizar, Yuxin Zheng, Ying Wu, Mohamed Farah, Barbara Slusher
{"title":"Targeting GCPII to promote remyelination in aging peripheral nerve injuries","authors":"Yu Su M.D., Ph.D., Meixiang Huang M.D., Ph.D., John Maragakis M.D., Ph.D., Ajit Thomas, Kaitlyn Huizar, Yuxin Zheng, Ying Wu, Mohamed Farah, Barbara Slusher","doi":"10.1016/j.neurot.2025.e00651","DOIUrl":"10.1016/j.neurot.2025.e00651","url":null,"abstract":"","PeriodicalId":19159,"journal":{"name":"Neurotherapeutics","volume":"22 4","pages":"Article e00651"},"PeriodicalIF":6.9,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144826889","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
The NIH HEAL Pain Therapeutics Development Program RFA-NS-24-019 美国国立卫生研究院治愈疼痛治疗发展计划RFA-NS-24-019
IF 6.9 2区 医学
Neurotherapeutics Pub Date : 2025-07-01 DOI: 10.1016/j.neurot.2025.e00652
Matthew Rice Ph.D., Jim Pomonis Ph.D., Mary Ann Pelleymounter Ph.D.
{"title":"The NIH HEAL Pain Therapeutics Development Program RFA-NS-24-019","authors":"Matthew Rice Ph.D., Jim Pomonis Ph.D., Mary Ann Pelleymounter Ph.D.","doi":"10.1016/j.neurot.2025.e00652","DOIUrl":"10.1016/j.neurot.2025.e00652","url":null,"abstract":"","PeriodicalId":19159,"journal":{"name":"Neurotherapeutics","volume":"22 4","pages":"Article e00652"},"PeriodicalIF":6.9,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144826890","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Azetukalner, a Novel, Potent KV7 Potassium Channel Opener in Development for the Treatment of Focal Onset Seizures, Primary Generalized Tonic-Clonic Seizures, and Major Depressive Disorder Azetukalner，一种新型、有效的KV7钾通道打开剂，用于治疗局灶性癫痫发作、原发性全身性强直-阵挛性癫痫发作和重度抑郁症
IF 6.9 2区 医学
Neurotherapeutics Pub Date : 2025-07-01 DOI: 10.1016/j.neurot.2025.e00648
Christopher Kenney M.D.
{"title":"Azetukalner, a Novel, Potent KV7 Potassium Channel Opener in Development for the Treatment of Focal Onset Seizures, Primary Generalized Tonic-Clonic Seizures, and Major Depressive Disorder","authors":"Christopher Kenney M.D.","doi":"10.1016/j.neurot.2025.e00648","DOIUrl":"10.1016/j.neurot.2025.e00648","url":null,"abstract":"","PeriodicalId":19159,"journal":{"name":"Neurotherapeutics","volume":"22 4","pages":"Article e00648"},"PeriodicalIF":6.9,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144826977","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
The phase 3 PROOF-HD trial demonstrates persistent benefits of pridopidine on function, cognition, and motor function through the 6 month open label extension in Huntington disease (HD) 3期PROOF-HD试验通过6个月的亨廷顿病（HD）开放标签延长，证明了普利多定对功能、认知和运动功能的持续益处。
IF 6.9 2区 医学
Neurotherapeutics Pub Date : 2025-07-01 DOI: 10.1016/j.neurot.2025.e00674
Michal Geva , Ralf Reilmann , Andrew Feigin , Anne Rosser , Sandra Kostyk , Kelly Chen , Yael Cohen , Y Paul Goldberg , Michael R. Hayden
{"title":"The phase 3 PROOF-HD trial demonstrates persistent benefits of pridopidine on function, cognition, and motor function through the 6 month open label extension in Huntington disease (HD)","authors":"Michal Geva , Ralf Reilmann , Andrew Feigin , Anne Rosser , Sandra Kostyk , Kelly Chen , Yael Cohen , Y Paul Goldberg , Michael R. Hayden","doi":"10.1016/j.neurot.2025.e00674","DOIUrl":"10.1016/j.neurot.2025.e00674","url":null,"abstract":"","PeriodicalId":19159,"journal":{"name":"Neurotherapeutics","volume":"22 4","pages":"Article e00674"},"PeriodicalIF":6.9,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144829471","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Go for the Gut: An innovative enteric pH-Driven Targeted Drug Delivery System for Patients with Maternally Inherited Mitochondrial Disease 走向肠道：一种创新的肠道ph驱动的靶向药物输送系统，用于母亲遗传线粒体疾病患者
IF 6.9 2区 医学
Neurotherapeutics Pub Date : 2025-07-01 DOI: 10.1016/j.neurot.2025.e00666
Martine Uittenbogaard Ph.D, Anne Chiaramello Ph.D
{"title":"Go for the Gut: An innovative enteric pH-Driven Targeted Drug Delivery System for Patients with Maternally Inherited Mitochondrial Disease","authors":"Martine Uittenbogaard Ph.D, Anne Chiaramello Ph.D","doi":"10.1016/j.neurot.2025.e00666","DOIUrl":"10.1016/j.neurot.2025.e00666","url":null,"abstract":"","PeriodicalId":19159,"journal":{"name":"Neurotherapeutics","volume":"22 4","pages":"Article e00666"},"PeriodicalIF":6.9,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144829728","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Discovery of clinical candidate 4ET1103, a peripherally-restricted MNK inhibitor for the treatment of pain 发现临床候选药物4ET1103，一种外周限制性MNK抑制剂，用于治疗疼痛
IF 6.9 2区 医学
Neurotherapeutics Pub Date : 2025-07-01 DOI: 10.1016/j.neurot.2025.e00656
James J. Sahn Ph.D. , Ted J. Price Ph.D. , Michele Curatolo Ph.D. , Joe Price , Craig Benson , Ayesha Ahmad , Juliet Mwirigi Ph.D. , Moeno Kume Ph.D.
{"title":"Discovery of clinical candidate 4ET1103, a peripherally-restricted MNK inhibitor for the treatment of pain","authors":"James J. Sahn Ph.D. , Ted J. Price Ph.D. , Michele Curatolo Ph.D. , Joe Price , Craig Benson , Ayesha Ahmad , Juliet Mwirigi Ph.D. , Moeno Kume Ph.D.","doi":"10.1016/j.neurot.2025.e00656","DOIUrl":"10.1016/j.neurot.2025.e00656","url":null,"abstract":"","PeriodicalId":19159,"journal":{"name":"Neurotherapeutics","volume":"22 4","pages":"Article e00656"},"PeriodicalIF":6.9,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144831145","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Repair mechanisms of the central nervous system: From axon sprouting to remyelination 中枢神经系统的修复机制：从轴突发芽到髓鞘再生。
IF 6.9 2区 医学
Neurotherapeutics Pub Date : 2025-07-01 DOI: 10.1016/j.neurot.2025.e00583
Lauren Gluck , Brittany Gerstein , Ulrike W. Kaunzner
{"title":"Repair mechanisms of the central nervous system: From axon sprouting to remyelination","authors":"Lauren Gluck ,&nbsp;Brittany Gerstein ,&nbsp;Ulrike W. Kaunzner","doi":"10.1016/j.neurot.2025.e00583","DOIUrl":"10.1016/j.neurot.2025.e00583","url":null,"abstract":"<div><div>The central nervous system (CNS), comprising the brain, spinal cord, and optic nerve, has limited regenerative capacity, posing significant challenges in treating neurological disorders. Recent advances in neuroscience and neurotherapeutics have introduced promising strategies to stimulate CNS repair, particularly in the context of neurodegenerative diseases such as multiple sclerosis. This review explores the complex interplay between inflammation, demyelination, and remyelination possibilities. Glial cells, including oligodendrocyte precursors, oligodendrocytes, astrocytes and microglia play dual roles in injury response, with reactive gliosis promoting repair but also potentially inhibiting recovery through glial scar formation. There is also an emphasis on axonal regeneration, axonal sprouting and stem cell therapies. We highlight the role of neuroplasticity in recovery post-injury and the limited regenerative potential of axons in the CNS due to inhibitory factors such as myelin-associated inhibitors. Moreover, neurotrophic factors support neuronal survival and axonal growth, while stem cell-based approaches offer promise for replacing lost neurons and glial cells. However, challenges such as stem cell survival, integration, and risk of tumor formation remain. Furthermore, we examine the role of neurogenesis in CNS repair and the remodeling of the extracellular matrix, which can facilitate regeneration. Through these diverse mechanisms, ongoing research aims to overcome the intrinsic and extrinsic barriers to CNS repair and advance therapeutic strategies for neurological diseases.</div></div>","PeriodicalId":19159,"journal":{"name":"Neurotherapeutics","volume":"22 4","pages":"Article e00583"},"PeriodicalIF":6.9,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144033852","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Impact of sex on multiple sclerosis therapy 性别对多发性硬化治疗的影响。
IF 6.9 2区 医学
Neurotherapeutics Pub Date : 2025-07-01 DOI: 10.1016/j.neurot.2025.e00620
Patricia K. Coyle
{"title":"Impact of sex on multiple sclerosis therapy","authors":"Patricia K. Coyle","doi":"10.1016/j.neurot.2025.e00620","DOIUrl":"10.1016/j.neurot.2025.e00620","url":null,"abstract":"<div><div>Multiple sclerosis (MS) is a female predominant disease. The prototypic affected individual is a young woman of childbearing age. This article emphasizes the current thinking about this disease, using a sex-based approach. It begins with a review of male vs. female differences in the nervous system and immune system, as well as a general discussion of sex hormones. The demographic and clinical impact of sex on MS prognosis and symptoms is covered. Pregnancy, the best studied sex-based condition, is reviewed and includes updated counseling information on fertility and assisted reproductive technology. MS issues in less well studied areas involving puberty, the menstrual cycle and menopause are also presented. The impact of sex on the MS therapeutic response is considered. The final section covers MS considerations in males. This up to date review provides a comprehensive and succinct approach to sex considerations in MS.</div></div>","PeriodicalId":19159,"journal":{"name":"Neurotherapeutics","volume":"22 4","pages":"Article e00620"},"PeriodicalIF":6.9,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144369104","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Pediatric multiple sclerosis: Improving outcome through high-efficacy therapies 儿童多发性硬化症：通过高效疗法改善预后。
IF 6.9 2区 医学
Neurotherapeutics Pub Date : 2025-07-01 DOI: 10.1016/j.neurot.2025.e00631
Lama Saleh Aljomah, E. Ann Yeh
{"title":"Pediatric multiple sclerosis: Improving outcome through high-efficacy therapies","authors":"Lama Saleh Aljomah,&nbsp;E. Ann Yeh","doi":"10.1016/j.neurot.2025.e00631","DOIUrl":"10.1016/j.neurot.2025.e00631","url":null,"abstract":"<div><div>Pediatric-onset multiple sclerosis (POMS) refers to multiple sclerosis occurring in individuals under 18 years of age. It is characterized by poor cognitive outcomes and a more inflammatory course, more frequent clinical relapses, and a greater number of MRI lesions compared to adult-onset MS (AOMS). Prompt recognition of multiple sclerosis in this population is essential, as early intervention with disease-modifying therapies may change the trajectory of disease progression. In this paper, we will review diagnostic criteria for pediatric multiple sclerosis, differential diagnosis, and current and emerging therapeutic approaches. While a number of DMTs are approved for adult MS, few are approved for pediatric use. Many of these DMTs are used off-label, with real-world evidence demonstrating their effectiveness and safety. The review evaluates existing evidence for the use of these therapies in pediatric populations, with an emphasis on both existing clinical trials and real-world data that supports their use. In addition, we will briefly highlight ongoing clinical trials and emerging therapies for POMS.</div></div>","PeriodicalId":19159,"journal":{"name":"Neurotherapeutics","volume":"22 4","pages":"Article e00631"},"PeriodicalIF":6.9,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144529027","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
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