Sebastian F Winter, Eugene J Vaios, Jorg Dietrich
PURPOSE OF REVIEW: Neurotoxicity from antineoplastic treatment remains a challenge in oncology. Cancer treatment-induced central nervous system (CNS) injury can be therapy-limiting, severely disabling, and even fatal. While emerging cancer immunotherapies have revolutionized oncology during the past decade, their immunomodulatory properties can cause immune-related adverse effects (IRAE) across organ systems, including the nervous system. Central neurologic IRAEs from chimeric antigen receptor T cells (CAR-T) and immune checkpoint inhibitors (ICPI) are challenging complications of such therapies...
September 15, 2020: Current Opinion in Neurology
Simone P Norris, Marie-France N Likanje, Jinsy A Andrews
PURPOSE OF REVIEW: The current review will provide recent updates in the clinical management of amyotrophic lateral sclerosis (ALS). RECENT FINDINGS: Although there is no cure for ALS, there are new treatments, growing knowledge of genetics, development of clinical staging systems, and the recent coronavirus disease 2019 pandemic that have recently impacted the clinical management of ALS. Increased understanding of genetics has helped provide insights into pathophysiology, the staging systems and clinical measures help to provide tools for monitoring disease clinically, and the recent coronavirus disease 2019 pandemic has provided opportunities to develop telemedicine and remote monitoring of disease thereby increasing accessibility to care and reducing burden of travel to centers for people living with the disease and their caregivers...
August 27, 2020: Current Opinion in Neurology
Jantima Tanboon, Akinori Uruha, Werner Stenzel, Ichizo Nishino
PURPOSE OF REVIEW: Discoveries of myositis-specific antibodies, transcriptomic signatures, and clinicoseropathological correlation support classification of idiopathic inflammatory myopathies (IIM) into four major subgroups: dermatomyositis, immune-mediated necrotizing myopathy (IMNM), antisynthetase syndrome (ASS), and inclusion body myositis (IBM) whereas leaving polymyositis as a historical nonspecific diagnosis of exclusion. This review summarizes and comments on recent knowledge regarding the major subgroup of IIM...
August 25, 2020: Current Opinion in Neurology
Niall Pender, Marta Pinto-Grau, Orla Hardiman
PURPOSE OF REVIEW: The current review provides an up to date overview of the nature and progression of the cognitive and behavioural impairment in amyotrophic lateral sclerosis (ALS). Understanding these symptoms has implications for the management of the disease and the design of clinical trials, in addition to the support of patient and caregiver regarding mental capacity and end of life decision-making. RECENT FINDINGS: Cognitive and behavioural change in ALS are best characterized as the consequence of extensive network dysfunction...
August 21, 2020: Current Opinion in Neurology
Marinos C Dalakas
PURPOSE OF REVIEW: To provide an update on immunomodulating and immunosuppressive therapies in myasthenia gravis and highlight newly approved, or pending approval, therapies with new biologics. RECENT FINDINGS: Preoperative IVIg is not needed to prevent myasthenic crisis in stable myasthenia gravis patients scheduled for surgery under general anesthesia, based on controlled data. Rituximab, if initiated early in new-onset myasthenia gravis, can lead to faster and more sustained remission even without immunotherapies in 35% of patients at 2 years...
August 21, 2020: Current Opinion in Neurology
Anders Meyer
PURPOSE OF REVIEW: Although tumors with nerve sheath differentiation are vast, the main clinically significant problems faced by the pathologist are the separation of malignant peripheral nerve sheath tumors (MPNSTs) from histologic mimics, the diagnosis of neurofibromatous neoplasms with atypical features, and the separation of cutaneous neurofibromatous neoplasms from melanoma. This review briefly discusses a variety of common nerve sheath tumors and summarizes recent advances on these diagnostic fronts...
August 21, 2020: Current Opinion in Neurology
Claudia Sommer
No abstract text is available yet for this article.
August 10, 2020: Current Opinion in Neurology
Ruben P A van Eijk, Tessa Kliest, Leonard H van den Berg
PURPOSE OF REVIEW: To review the current developments in the design and conduct of clinical trials for amyotrophic lateral sclerosis (ALS), illustrated by a critical appraisal of RECENT FINDINGS: In total, 63 clinical trials were included in the analysis, of which 13 phase 1, 35 phase 2 and 15 phase 3. Virtually all phase 3 clinical trials can be classified as randomized, placebo controlled, whereas this is only true for 57% of the phase 2 clinical trials...
August 10, 2020: Current Opinion in Neurology
John Vissing
No abstract text is available yet for this article.
August 10, 2020: Current Opinion in Neurology
Nick Verber, Pamela J Shaw
PURPOSE OF REVIEW: This review draws together the most recent findings in ALS biomarker research from biochemical, imaging and neurophysiology techniques. RECENT FINDINGS: The potential of circulating RNA is highlighted, including new retrieval techniques. With ongoing genetic clinical trials, the need for pharmacodynamic biomarkers is essential. There is a strong case for neurofilament proteins being validated in ALS; their biomarker profile is discussed. Oxidative stress and neuroinflammation studies offer insight into disease mechanisms and offer good biomarker potential...
August 10, 2020: Current Opinion in Neurology
Claudia Nuñez-Peralta, Jorge Alonso-Pérez, Jordi Díaz-Manera
PURPOSE OF REVIEW: This review aims to discuss the recent results of studies published applying quantitative MRI sequences to large cohorts of patients with neuromuscular diseases. RECENT FINDINGS: Quantitative MRI sequences are now available to identify and quantify changes in muscle water and fat content. These two components have been associated with acute and chronic injuries, respectively. Studies show that the increase in muscle water is not only reversible if therapies are applied successfully but can also predict fat replacement in neurodegenerative diseases...
August 10, 2020: Current Opinion in Neurology
Maike F Dohrn, Mario Saporta
PURPOSE OF REVIEW: Hereditary motor neuropathies (HMN) comprise a broad genotypic and phenotypic spectrum of rare, progressively disabling diseases manifesting with length-dependent muscle weakness and atrophy. To date, more than half of the cases cannot be genetically explained. To provide symptomatic and disease-modifying treatments in the future, a better understanding of disease mechanisms is required. RECENT FINDINGS: By whole exome and genome sequencing, the discovery of several novel genes (SCO2, TDRKH, SPTAN1, CADM3, and SORD) involved in the pathogenesis of HMN has now relevantly changed the pathophysiological knowledge...
August 10, 2020: Current Opinion in Neurology
Linde F Bouwman, Silvère M van der Maarel, Jessica C de Greef
PURPOSE OF REVIEW: Facioscapulohumeral muscular dystrophy (FSHD) is a neuromuscular disorder, which is caused by incomplete repression of the transcription factor double homeobox 4 (DUX4) in skeletal muscle. To date, there is no DUX4-targeting treatment to prevent or delay disease progression. In the present review, we summarize developments in therapeutic strategies with the focus on inhibiting DUX4 and DUX4 target gene expression. RECENT FINDINGS: Different studies show that DUX4 and its target genes can be repressed with genetic therapies using diverse strategies...
August 6, 2020: Current Opinion in Neurology
Christopher Grunseich, Kenneth H Fischbeck
PURPOSE OF REVIEW: The aim of this study was to illustrate the current understanding and avenues for developing treatment in spinal and bulbar muscular atrophy (SBMA), an inherited neuromuscular disorder caused by a CAG trinucleotide repeat expansion in the androgen receptor (AR) gene. RECENT FINDINGS: Important advances have been made in characterizing the molecular mechanism of the disease, including the disruption of protein homeostasis, intracellular trafficking and signalling pathways...
August 6, 2020: Current Opinion in Neurology
Kathryn R Wagner
PURPOSE OF REVIEW: Recent terminations of clinical trials of myostatin inhibitors in muscular dystrophy have raised questions about the predictiveness of mouse models for this therapeutic strategy. RECENT FINDINGS: A variety of myostatin inhibitors have been developed for preclinical and clinical studies. These inhibitors have ameliorated the phenotype of many but not all mouse models of muscular dystrophy. However, randomized double-blinded placebo controlled trials in both pediatric and adult muscular dystrophies have, as of yet, not demonstrated functional improvement...
August 6, 2020: Current Opinion in Neurology
Kristin Samuelsson, Rayomand Press
PURPOSE OF REVIEW: Chronic idiopathic axonal polyneuropathy (CIAP), a common neurological condition, is considered to be a benign neurological condition with a small risk of disability. However, many studies have shown a reduced quality of life and a nonnegligible affection of daily activities in patients with CIAP. Here we summarize recent data about CIAP. RECENT FINDINGS: We discuss some of the latest articles regarding risk factors, comorbidities, and possible pathogenic factors regarding CIAP...
August 6, 2020: Current Opinion in Neurology
David Adams, Michel Slama
PURPOSE OF REVIEW: Hereditary transthyretin amyloidosis (ATTRv) is a rare autosomal dominant, life-threatening disease. Until recently only early stages of ATTRv-PN (polyneuropathy) had access to disease-modifying therapy (DMT), whereas there was no specific treatment for ATTRv-CM (cardiomyopathy). This review updates our knowledge about results of three phase 3 clinical trials, expert's consensus for early diagnosis and emerging biomarkers. RECENT FINDINGS: Two phase 3 studies using RNAi and antisense oligonucleotides (ASO) were successful...
October 2020: Current Opinion in Neurology
Bjarne Udd
PURPOSE OF REVIEW: The last few years have confirmed previous assumptions of an enormous impact of the titin gene (TTN) on the occurrence of muscle disease, cardiomyopathy, or both together. The reason for this rather late understanding of its importance is because of the huge size which prevented sequencing of the whole gene by the previous Sanger technique in the individual cases. An update of the advances in diagnosing titinopathies is the main focus of this review. RECENT FINDINGS: High throughput methods are now widely available for TTN sequencing and a corresponding explosion of different types of identified titinopathies is observed and published in the literature, although final confirmation is lacking in many cases with recessive missense variants...
October 2020: Current Opinion in Neurology
Annu Aggarwal, Mohit Bhatt
PURPOSE OF REVIEW: The aim of this article is to review recent developments in the areas of the disease features and treatment of Wilson disease, and survey disorders that share its pathophysiology or clinical symptoms. RECENT FINDINGS: Knowledge of the clinical spectrum of Wilson disease has expanded with recognition of patients who present in atypical age groups - patients with very early onset (<5 years) and those in whom symptoms present in mid-to-late adulthood...
August 2020: Current Opinion in Neurology
Pavan A Vaswani, Abby L Olsen
PURPOSE OF REVIEW: Progressive supranuclear palsy (PSP) is a progressive adult-onset neurodegenerative disease. Abnormally, phosphorylated forms of the microtubule-associated protein tau containing four repeat domains (4R-tau) aggregate in neurons. Additionally, increasing evidence suggests that secretion and uptake of fragments of abnormal 4R-tau may play a role in disease progression. This extracellular tau is a natural target for immunotherapy. RECENT FINDINGS: Three monoclonal antibodies targeting extracellular tau are in clinical stages of development...
August 2020: Current Opinion in Neurology
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