{"title":"Profiling deutetrabenazine extended-release tablets for tardive dyskinesia and chorea associated with Huntington's disease.","authors":"P Moondra, J Jimenez-Shahed","doi":"10.1080/14737175.2024.2376107","DOIUrl":"10.1080/14737175.2024.2376107","url":null,"abstract":"<p><strong>Introduction: </strong>Tardive dyskinesia (TD) and Huntington's disease (HD)-associated chorea are persistent and disabling hyperkinetic disorders that can be treated with vesicular monoamine transporter type 2 (VMAT2) inhibitors, including the recently approved once-daily (QD) formulation of deutetrabenazine (DTBZ ER). While its efficacy and safety profile have not been directly investigated, currently available data confirms bioequivalence and similar bioavailability to the twice-daily formulation (DTBZ BID).</p><p><strong>Areas covered: </strong>The authors briefly review the pivotal trials establishing efficacy of DTBZ for TD and HD-associated chorea, the pharmacokinetic data for bioequivalence between QD and BID dosing of DTBZ, as well as dose proportionality evidence, titration recommendations, and safety profile for DTBZ ER.</p><p><strong>Expert opinion: </strong>Long-term data show that DTBZ is efficacious and well tolerated for the treatment of TD and HD-associated chorea. DTBZ ER likely demonstrates therapeutic equivalence with no new safety signals. Due to the lack of comparative clinical trial data, no evidence-based recommendation about choice of VMAT2 inhibitor or switching between VMAT2 inhibitors can be made about best practice. Ultimately, QD dosing may offer the chance of improved medication adherence, an important consideration in patients with complex treatment regimens and/or patients with cognitive decline.</p>","PeriodicalId":12190,"journal":{"name":"Expert Review of Neurotherapeutics","volume":" ","pages":"849-863"},"PeriodicalIF":3.4,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141563082","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}
Matthew J Goldman, Alexandra M Baskin, Martyn A Sharpe, David S Baskin
{"title":"Advances in gene therapy for high-grade glioma: a review of the clinical evidence.","authors":"Matthew J Goldman, Alexandra M Baskin, Martyn A Sharpe, David S Baskin","doi":"10.1080/14737175.2024.2376847","DOIUrl":"10.1080/14737175.2024.2376847","url":null,"abstract":"<p><strong>Introduction: </strong>High-grade glioma (HGG) is one of the most deadly and difficult cancers to treat. Despite intense research efforts, there has not been a significant breakthrough in treatment outcomes since the early 2000's. Anti-glioma gene therapy has demonstrated promise in preclinical studies and is under investigation in numerous clinical trials.</p><p><strong>Areas covered: </strong>This manuscript reviews the current landscape of clinical trials exploring gene therapy treatment of HGG. Using information from clinicaltrials.gov, all trials initiated within the past 5 years (2018-2023) as well as other important trials were cataloged and reviewed. This review discusses trial details, innovative methodologies, and concurrent pharmacological interventions. The review also delves into the subtypes of gene therapy used, trends over time, and future directions.</p><p><strong>Expert opinion: </strong>Trials are in the early stages (phase I or II), and there are reports of clinical efficacy in published results. Synergistic effects utilizing immunotherapy within or alongside gene therapy are emerging as a promising avenue for future breakthroughs. Considerable heterogeneity exists across trials concerning administration route, vector selection, drug combinations, and intervention timing. Earlier intervention in newly diagnosed HGG and avoidance of corticosteroids may improve efficacy in future trials. The results from ongoing trials demonstrate promising potential for molding the future landscape of HGG care.</p>","PeriodicalId":12190,"journal":{"name":"Expert Review of Neurotherapeutics","volume":" ","pages":"879-895"},"PeriodicalIF":3.4,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141874557","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}
{"title":"The importance of synthetic pharmacotherapy for recessive cerebellar ataxias.","authors":"Marie Beaudin, Nicolas Dupre, Mario Manto","doi":"10.1080/14737175.2024.2376840","DOIUrl":"10.1080/14737175.2024.2376840","url":null,"abstract":"<p><strong>Introduction: </strong>The last decade has witnessed major breakthroughs in identifying novel genetic causes of hereditary ataxias, deepening our understanding of disease mechanisms, and developing therapies for these debilitating disorders.</p><p><strong>Areas covered: </strong>This article reviews the currently approved and most promising candidate pharmacotherapies in relation to the known disease mechanisms of the most prevalent autosomal recessive ataxias. Omaveloxolone is an Nrf2 activator that increases antioxidant defense and was recently approved for treatment of Friedreich ataxia. Its therapeutic effect is modest, and further research is needed to find synergistic treatments that would halt or reverse disease progression. Promising approaches include upregulation of frataxin expression by epigenetic mechanisms, direct protein replacement, and gene replacement therapy. For ataxia-telangiectasia, promising approaches include splice-switching antisense oligonucleotides and small molecules targeting oxidative stress, inflammation, and mitochondrial function. Rare recessive ataxias for which disease-modifying therapies exist are also reviewed, emphasizing recently approved therapies. Evidence supporting the use of riluzole and acetyl-leucine in recessive ataxias is discussed.</p><p><strong>Expert opinion: </strong>Advances in genetic therapies for other neurogenetic conditions have paved the way to implement feasible approaches with potential dramatic benefits. Particularly, as we develop effective treatments for these conditions, we may need to combine therapies, consider newborn testing for pre-symptomatic treatment, and optimize non-pharmacological approaches.</p>","PeriodicalId":12190,"journal":{"name":"Expert Review of Neurotherapeutics","volume":" ","pages":"897-912"},"PeriodicalIF":3.4,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141558439","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}
Benedikt Reuter, Annemarie Miano, Josepha Wassermann, Björn Elsner
{"title":"Why does exposure-based therapy fail in some individuals with obsessive-compulsive disorder?","authors":"Benedikt Reuter, Annemarie Miano, Josepha Wassermann, Björn Elsner","doi":"10.1080/14737175.2024.2365949","DOIUrl":"10.1080/14737175.2024.2365949","url":null,"abstract":"","PeriodicalId":12190,"journal":{"name":"Expert Review of Neurotherapeutics","volume":" ","pages":"723-726"},"PeriodicalIF":3.4,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141320795","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}
{"title":"An overview of the currently available and emerging long-acting formulations of risperidone for schizophrenia and bipolar disorder.","authors":"Justin Faden, Camila Ramirez, Vanessa Martinez, Leslie Citrome","doi":"10.1080/14737175.2024.2370349","DOIUrl":"10.1080/14737175.2024.2370349","url":null,"abstract":"<p><strong>Introduction: </strong>Long-acting injectable (LAI) antipsychotic medications can help improve treatment adherence in patients with schizophrenia and bipolar disorder. Despite this, they are underutilized. In 2003, intramuscular risperidone became the first available LAI atypical antipsychotic medication, and since then, a number of competing long-acting risperidone formulations have been brought to market, with additional options under active development. These include intramuscular, subcutaneous, long-acting oral, and implantable formulations.</p><p><strong>Areas covered: </strong>This review summarizes currently available and emerging long-acting risperidone formulations, including efficacy and safety data, and practical considerations aimed to help prescribers distinguish one formulation from another.</p><p><strong>Expert opinion: </strong>There is an expanding number of currently available LAI antipsychotic medications giving patients and providers an opportunity to personalize and individualize care. Rates of adherence to treatment in patients with schizophrenia and bipolar disorder are low, and individualizing care can help improve this. The risperidone LAI treatment landscape includes five options approved by the U.S. Food and Drug Administration, with others under clinical development. These options differ in regard to mode of administration, approved indications, available dose strengths, injection intervals, needle size, injection volume, storage, and other variables. Prescribers should be familiar with these differing options to help patients find the best fit for their individual needs.</p>","PeriodicalId":12190,"journal":{"name":"Expert Review of Neurotherapeutics","volume":" ","pages":"761-771"},"PeriodicalIF":3.4,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141751480","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}
{"title":"Distinguishing essential tremor from Parkinson's disease: clinical and experimental tools.","authors":"Luca Angelini, Giulia Paparella, Matteo Bologna","doi":"10.1080/14737175.2024.2372339","DOIUrl":"10.1080/14737175.2024.2372339","url":null,"abstract":"<p><strong>Introduction: </strong>Essential tremor (ET) and Parkinson's disease (PD) are the most common causes of tremor and the most prevalent movement disorders, with overlapping clinical features that can lead to diagnostic challenges, especially in the early stages.</p><p><strong>Areas covered: </strong>In the present paper, the authors review the clinical and experimental studies and emphasized the major aspects to differentiate between ET and PD, with particular attention to cardinal phenomenological features of these two conditions. Ancillary and experimental techniques, including neurophysiology, neuroimaging, fluid biomarker evaluation, and innovative methods, are also discussed for their role in differential diagnosis between ET and PD. Special attention is given to investigations and tools applicable in the early stages of the diseases, when the differential diagnosis between the two conditions is more challenging. Furthermore, the authors discuss knowledge gaps and unsolved issues in the field.</p><p><strong>Expert opinion: </strong>Distinguishing ET and PD is crucial for prognostic purposes and appropriate treatment. Additionally, accurate diagnosis is critical for optimizing clinical and experimental research on pathophysiology and innovative therapies. In a few years, integrated technologies could enable accurate, reliable diagnosis from early disease stages or prodromal stages in at-risk populations, but further research combining different techniques is needed.</p>","PeriodicalId":12190,"journal":{"name":"Expert Review of Neurotherapeutics","volume":" ","pages":"799-814"},"PeriodicalIF":3.4,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141626512","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}
Andreas Reis, Marlon Westhoff, Hicham Quintarelli, Stefan G Hofmann
{"title":"Mindfulness as a therapeutic option for obsessive-compulsive disorder.","authors":"Andreas Reis, Marlon Westhoff, Hicham Quintarelli, Stefan G Hofmann","doi":"10.1080/14737175.2024.2365945","DOIUrl":"10.1080/14737175.2024.2365945","url":null,"abstract":"<p><strong>Introduction: </strong>Obsessive-compulsive disorder (OCD) is a prevalent mental health issue characterized by intrusive thoughts (obsessions) and repetitive behaviors (compulsions) that can cause significant life impairment. Despite cognitive-behavioral therapy (CBT) being the most effective treatment, some individuals experience insufficient symptom reduction or relapse.</p><p><strong>Areas covered: </strong>This special report explores the potential of mindfulness-based interventions as complementary treatments for OCD, examining the specific techniques used and their practical application. In the initial section, the authors examine ten randomized control trial studies included in the meta-analysis conducted by Chien et al. (2022), demonstrating the effectiveness of mindfulness interventions. The authors focus on elucidating the specific mindfulness techniques used in these studies. Then, the authors discuss the integration of these mindfulness strategies into CBT, focusing on enhancing emotional regulation, cognitive flexibility, and acceptance of intrusive thoughts.</p><p><strong>Expert opinion: </strong>While mindful based interventions (MBIs) show promise as adjunctive treatments for OCD, variability in OCD symptoms and treatment responses necessitate individualized therapeutic approaches. Further research is required to refine mindfulness-based techniques and optimize their effectiveness. Incorporating MBIs into standard CBT protocols may improve outcomes for patients with persistent OCD symptoms.</p>","PeriodicalId":12190,"journal":{"name":"Expert Review of Neurotherapeutics","volume":" ","pages":"735-741"},"PeriodicalIF":3.4,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141418497","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}
Joseph V Pergolizzi, Jo Ann LeQuang, Salah N El-Tallawy, Morgan Wagner, Rania S Ahmed, Giustino Varrassi
{"title":"An update on pharmacotherapy for trigeminal neuralgia.","authors":"Joseph V Pergolizzi, Jo Ann LeQuang, Salah N El-Tallawy, Morgan Wagner, Rania S Ahmed, Giustino Varrassi","doi":"10.1080/14737175.2024.2365946","DOIUrl":"10.1080/14737175.2024.2365946","url":null,"abstract":"<p><strong>Introduction: </strong>Trigeminal neuralgia is a rare condition that can be effectively treated by carbamazepine or oxcarbazepine but these older drugs are associated with dose-dependent and potentially treatment-limiting adverse effects. Third-generation anticonvulsants, new calcitonin gene-related peptide blockers for migraine, and older drugs such as ketamine and cannabinoids may be promising adjuvants or monotherapeutic options.</p><p><strong>Areas covered: </strong>The new drugs, their presumed mechanisms of action, safety and efficacy are discussed herein. There is a paucity of robust clinical evidence in support of these drugs for trigeminal neuralgia. New migraine agents are considered as well although migraines and trigeminal neuralgia are distinct, albeit similar, conditions. No new drugs have been released to market in recent years with the specific indication of trigeminal neuralgia.</p><p><strong>Expert opinion: </strong>In real-world clinical practice, about half of trigeminal neuralgia patients take more than one agent for prevention and combination therapy may be the optimal approach. Combination therapy might allow for lower doses of carbamazepine or oxcarbazepine, thus reducing the number and severity of potential adverse events but the potential for pharmacokinetic drug-drug interactions must be considered. Drug therapy for trigeminal neuralgia involves acute or abortive treatments, often administered in hospital versus long-term preventive therapy, usually involving oral agents.</p>","PeriodicalId":12190,"journal":{"name":"Expert Review of Neurotherapeutics","volume":" ","pages":"773-786"},"PeriodicalIF":3.4,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141316916","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}
Giulio Emilio Brancati, Anna Magnesa, Donatella Acierno, Marco Carli, Ugo De Rosa, Alessandro Froli, Samuele Gemignani, Lisa Ventura, Francesco Weiss, Giulio Perugi
{"title":"Current nonstimulant medications for adults with attention-deficit/hyperactivity disorder.","authors":"Giulio Emilio Brancati, Anna Magnesa, Donatella Acierno, Marco Carli, Ugo De Rosa, Alessandro Froli, Samuele Gemignani, Lisa Ventura, Francesco Weiss, Giulio Perugi","doi":"10.1080/14737175.2024.2370346","DOIUrl":"10.1080/14737175.2024.2370346","url":null,"abstract":"<p><strong>Introduction: </strong>Stimulants, including methylphenidate and amphetamines, are the first-line pharmacological treatment of ADHD in adults. However, in patients who do not respond or poorly tolerate stimulants, non-stimulant medications are usually recommended.</p><p><strong>Areas covered: </strong>The authors provide a narrative review of the literature on non-stimulant treatments for adult ADHD, including controlled and observational clinical studies conducted on adult samples. Atomoxetine has been extensively studied and showed significant efficacy in treating adult ADHD. Issues related to dosing, treatment duration, safety, and use in the case of psychiatric comorbidity are summarized. Among other compounds indicated for ADHD in adults, antidepressants sharing at least a noradrenergic or dopaminergic component, including tricyclic compounds, bupropion, and viloxazine, have shown demonstratable efficacy. Evidence is also available for antihypertensives, particularly guanfacine, as well as memantine, metadoxine, and mood stabilizers, while negative findings have emerged for galantamine, antipsychotics, and cannabinoids.</p><p><strong>Expert opinion: </strong>While according to clinical guidelines, atomoxetine may serve as the only second-line option in adults with ADHD, several other nonstimulant compounds may be effectively used in order to personalize treatment based on comorbid conditions and ADHD features. Nevertheless, further research is needed to identify and test more personalized treatment strategies for adults with ADHD.</p>","PeriodicalId":12190,"journal":{"name":"Expert Review of Neurotherapeutics","volume":" ","pages":"743-759"},"PeriodicalIF":3.4,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141445915","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}
Michail Vikelis, Dimitrios Rikos, Andreas A Argyriou, Pinelopi Papachristou, Dimitrios Rallis, Theodoros Karapanayiotides, Andreas Galanopoulos, Konstantinos Spingos, Nikolaos Dimisianos, Emmanouil Giakoumakis, Periklis Zavridis, Konstantinos Notas, George S Vlachos, Panagiotis Soldatos, Konstantinos Bilias, Georgia Xiromerisiou, Jobst Rudolf, Emmanouil V Dermitzakis, Alan M Rapoport
{"title":"Preferences and perceptions of 617 migraine patients on acute and preventive migraine treatment attributes and clinical trial endpoints.","authors":"Michail Vikelis, Dimitrios Rikos, Andreas A Argyriou, Pinelopi Papachristou, Dimitrios Rallis, Theodoros Karapanayiotides, Andreas Galanopoulos, Konstantinos Spingos, Nikolaos Dimisianos, Emmanouil Giakoumakis, Periklis Zavridis, Konstantinos Notas, George S Vlachos, Panagiotis Soldatos, Konstantinos Bilias, Georgia Xiromerisiou, Jobst Rudolf, Emmanouil V Dermitzakis, Alan M Rapoport","doi":"10.1080/14737175.2024.2365312","DOIUrl":"10.1080/14737175.2024.2365312","url":null,"abstract":"<p><strong>Background: </strong>To identify the preferences and perceptions of migraine patients for acute and preventive treatment options and to investigate which treatment outcomes are the most important.</p><p><strong>Design and methods: </strong>The authors performed a choice-format survey in a cohort of migraine patients from Greece and Cyprus. A self-administered questionnaire developed in collaboration with the Greek Society of Migraine Patients was used.</p><p><strong>Results: </strong>Questionnaires were collected from 617 migraine patients. Efficacy was preferred over safety as the single most important parameter, both in acute and preventive treatment. When analyzing single outcomes, patients prioritized a complete pain remission at 1-hour post-dose for acute therapies. Regarding migraine prevention, a 75% reduction in frequency, intensity of pain, accompanying symptoms and acute medication intake were considered as most important. Conversely, outcomes routinely used in clinical trials, namely complete or partial pain remission at 2-hours post-dose for acute treatment and 50% or 30% reduction in migraine frequency for prevention, were not deemed particularly relevant. Tablet formulation was mostly preferred, both in acute and preventive treatment. Conclusion: Listening to patients' needs may add a piece of the puzzle that is generally missing in clinical practice and often explains the lack of adherence in both acute and preventative anti-migraine therapies.</p>","PeriodicalId":12190,"journal":{"name":"Expert Review of Neurotherapeutics","volume":" ","pages":"815-826"},"PeriodicalIF":3.4,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141316917","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}