Expert Opinion on Emerging Drugs最新文献_第4页

Cannabinoid treatment for the symptoms of autism spectrum disorder. 治疗自闭症谱系障碍症状的大麻素疗法。

IF 3.4 3区医学

Expert Opinion on Emerging Drugs Pub Date : 2024-03-01 Epub Date: 2024-01-23 DOI: 10.1080/14728214.2024.2306290

Adi Aran, Dalit Cayam Rand

{"title":"Cannabinoid treatment for the symptoms of autism spectrum disorder.","authors":"Adi Aran, Dalit Cayam Rand","doi":"10.1080/14728214.2024.2306290","DOIUrl":"10.1080/14728214.2024.2306290","url":null,"abstract":"Introduction: Autism spectrum disorder (ASD) is a neurodevelopmental disorder affecting approximately 3% of school-age children. The core symptoms are deficits in social communication and restricted and repetitive patterns of behavior. Associated problems in cognition, language, behavior, sleep and mood are prevalent. Currently, no established pharmacological treatment exists for core ASD symptoms. Risperidone and aripiprazole are used to manage associated irritability, but their effectiveness is limited and adverse events are common.Areas covered: This mini-review summarizes existing scientific literature and ongoing clinical trials concerning cannabinoid treatment for ASD. Uncontrolled case series have documented improvements in both core ASD symptoms and related behavioral challenges in children treated with cannabis extracts rich in cannabidiol (CBD). Placebo-controlled studies involving CBD-rich cannabis extracts and/or pure CBD in children with ASD have demonstrated mixed efficacy results. A similar outcome was observed in a placebo-controlled study of pure CBD addressing social avoidance in Fragile X syndrome. Importantly, these studies have shown relatively high safety and tolerability.Expert opinion: While current clinical data suggest the potential of CBD and CBD-rich cannabis extract in managing core and behavioral deficits in ASD, it is prudent to await the results of ongoing placebo-controlled trials before considering CBD treatment for ASD.","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"65-79"},"PeriodicalIF":3.4,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139471717","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"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

Review of phase 2/3 trials in polymyalgia rheumatica and giant cell arteritis. 多发性风湿痛和巨细胞动脉炎 2/3 期试验回顾。

IF 3.4 3区医学

Expert Opinion on Emerging Drugs Pub Date : 2024-03-01 Epub Date: 2024-01-13 DOI: 10.1080/14728214.2024.2303093

Pratheeshaa Nageswaran, Saad Ahmed, Hasan Tahir

{"title":"Review of phase 2/3 trials in polymyalgia rheumatica and giant cell arteritis.","authors":"Pratheeshaa Nageswaran, Saad Ahmed, Hasan Tahir","doi":"10.1080/14728214.2024.2303093","DOIUrl":"10.1080/14728214.2024.2303093","url":null,"abstract":"Introduction: GCA (giant cell arteritis) and PMR (polymyalgia rheumatica) are two overlapping inflammatory rheumatic conditions that are seen exclusively in older adults, sharing some common features. GCA is a clinical syndrome characterized by inflammation of the medium and large arteries, with both cranial and extracranial symptoms. PMR is a clinical syndrome characterized by stiffness in the neck, shoulder, and pelvic girdle muscles. Both are associated with constitutional symptoms.Areas covered: In this review, we assess the established and upcoming treatments for GCA and PMR. We review the current treatment landscape, completed trials, and upcoming trials in these conditions, to identify new and promising therapies.Expert opinion: Early use of glucocorticoids (GC) remains integral to the immediate management of PMR and GCA but being aware of patient co-morbidities that may influence treatment toxicity is paramount. As such GC sparing agents are required in the treatment of PMR. Currently there are limited treatment options available for PMR and GCA, and significant unmet needs remain. Newer mechanisms of action, and hence therapeutic options being studied include CD4 T cell co-stimulation blockade, IL-17 inhibition, IL-12/23 inhibition, GM-CSF inhibition, IL-1β inhibition, TNF-α antagonist and Jak inhibition, among others, which will be discussed in this review.","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"5-17"},"PeriodicalIF":3.4,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139106087","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"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

How will tovorafenib change our treatment of pediatric low-grade glioma? 托伐非尼将如何改变我们对小儿低级别胶质瘤的治疗？

IF 3.4 3区医学

Expert Opinion on Emerging Drugs Pub Date : 2024-03-01 Epub Date: 2024-02-02 DOI: 10.1080/14728214.2024.2312817

Inci Yaman, Eric Bouffet

引用次数: 0

Targeting the PACAP-38 pathway is an emerging therapeutic strategy for migraine prevention. 以 PACAP-38 通路为靶点是一种新兴的偏头痛预防治疗策略。

IF 3.4 3区医学

Expert Opinion on Emerging Drugs Pub Date : 2024-03-01 Epub Date: 2024-02-12 DOI: 10.1080/14728214.2024.2317778

Lanfranco Pellesi, Messoud Ashina, Paolo Martelletti

{"title":"Targeting the PACAP-38 pathway is an emerging therapeutic strategy for migraine prevention.","authors":"Lanfranco Pellesi, Messoud Ashina, Paolo Martelletti","doi":"10.1080/14728214.2024.2317778","DOIUrl":"10.1080/14728214.2024.2317778","url":null,"abstract":"Introduction: The pituitary adenylate cyclase-activating polypeptide-38 (PACAP-38) has emerged as a key mediator of migraine pathogenesis. PACAP-38 and its receptors are predominantly distributed in arteries, sensory and parasympathetic neurons of the trigeminovascular system. Phase 2 trials have tested human monoclonal antibodies designed to bind and inhibit PACAP-38 and the pituitary adenylate cyclase-activating polypeptide type I (PAC1) receptor for migraine prevention.Areas covered: This review focuses on the significance of the PACAP-38 pathway as a target in migraine prevention. English peer-reviewed articles were searched in PubMed, Scopus and ClinicalTrials.gov electronic databases.Expert opinion: A PAC1 receptor monoclonal antibody was not effective for preventing migraine in a proof-of-concept trial, paving the way for alternative strategies to be considered. Lu AG09222 is a humanized monoclonal antibody targeting PACAP-38 that was effective in preventing physiological responses of PACAP38 and reducing monthly migraine days in individuals with migraine. Further studies are necessary to elucidate the clinical utility, long-term safety and cost-effectiveness of therapies targeting the PACAP pathway.","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"57-64"},"PeriodicalIF":3.4,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139711809","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"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

Novel and emerging drugs for the treatment of Crohn's disease: a review of phase II and III trials. 治疗克罗恩病的新型和新兴药物：II 期和 III 期试验回顾。

IF 3.4 3区医学

Expert Opinion on Emerging Drugs Pub Date : 2024-03-01 Epub Date: 2024-01-11 DOI: 10.1080/14728214.2024.2303116

Panu Wetwittayakhlang, Talat Bessissow, Peter L Lakatos

{"title":"Novel and emerging drugs for the treatment of Crohn's disease: a review of phase II and III trials.","authors":"Panu Wetwittayakhlang, Talat Bessissow, Peter L Lakatos","doi":"10.1080/14728214.2024.2303116","DOIUrl":"10.1080/14728214.2024.2303116","url":null,"abstract":"Introduction: Crohn's disease (CD) is a chronic inflammatory bowel disease characterized by unpredictable flare-ups and periods of remission. While several therapeutic options, such as anti-tumor necrosis factor (TNF), anti-integrin, and interleukin (IL) 12/23 inhibitors, as well as IL-23 and Janus kinase (JAK) inhibitors, have been approved for CD treatment, a substantial number of patients fail to respond adequately or experience a loss of response over time. In recent years, the scientific community has been actively investigating novel agents to address these challenges and improve the management of CD.Areas covered: This comprehensive narrative review provides an overview of recent developments in CD treatment, summarizing phase 2 and phase 3 clinical trial data. We delve into the clinical efficacy and safety profiles of emerging therapies, encompassing JAK inhibitors, IL-23 inhibitors, anti-adhesion molecules, S1P1 receptor modulators, and combined targeted treatments.Expert opinion: The armamentarium of CD therapeutic agents is constantly expanding. We analyze pivotal findings from phase 2 and phase 3 CD treatment trials. We also underscore the existing gaps in therapy and the paramount role of ongoing research and innovation in CD management.","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"19-34"},"PeriodicalIF":3.4,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139402527","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"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

How promising are the latest monoclonal antibodies targeting amyloid-β for the treatment of early Alzheimer's disease? 针对淀粉样蛋白-β的最新单克隆抗体治疗早期阿尔茨海默病的前景如何？

IF 3.4 3区医学

Expert Opinion on Emerging Drugs Pub Date : 2024-03-01 Epub Date: 2024-01-12 DOI: 10.1080/14728214.2024.2304059

Jordan Beveridge, Eileen Kaniecki, Aniketh Naidu, Bret David Silverglate, George Grossberg

{"title":"How promising are the latest monoclonal antibodies targeting amyloid-β for the treatment of early Alzheimer's disease?","authors":"Jordan Beveridge, Eileen Kaniecki, Aniketh Naidu, Bret David Silverglate, George Grossberg","doi":"10.1080/14728214.2024.2304059","DOIUrl":"10.1080/14728214.2024.2304059","url":null,"abstract":"Introduction: Monoclonal antibodies targeting amyloid-β are the first disease-modifying treatments for Alzheimer disease to have received FDA-approval. There are three different drugs approved or pending FDA-approval: aducanumab, lecanemab, and donanemab. These three drugs are each in different stages of regulatory approval by the FDA.Areas covered: We discuss the development of these drugs, the data regarding their clinical efficacy, their dosing regimens, and side effects. In addition, we examine pragmatic issues with their potential implementation as common treatments to slow the rate of decline in Alzheimer disease, and what unanswered questions remain regarding this new class of drugs.Expert opinion: We conclude that these new monoclonal antibodies that target amyloid-β represent a genuine advance in the treatment of Alzheimer disease. However, questions remain regarding their clinical significance. Additionally, it is presently unclear which patients would most benefit from these expensive drugs given the risk of side effects and the logistical difficulties concerning administration and the determination of eligibility.","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"35-43"},"PeriodicalIF":3.4,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139402526","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"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

Emerging drugs for the treatment of irritability associated with autism spectrum disorder. 治疗与自闭症谱系障碍有关的易激惹症的新兴药物。

IF 3.4 3区医学

Expert Opinion on Emerging Drugs Pub Date : 2024-03-01 Epub Date: 2024-02-04 DOI: 10.1080/14728214.2024.2313650

Ahmad Shamabadi, Hanie Karimi, Razman Arabzadeh Bahri, Mohsen Motavaselian, Shahin Akhondzadeh

{"title":"Emerging drugs for the treatment of irritability associated with autism spectrum disorder.","authors":"Ahmad Shamabadi, Hanie Karimi, Razman Arabzadeh Bahri, Mohsen Motavaselian, Shahin Akhondzadeh","doi":"10.1080/14728214.2024.2313650","DOIUrl":"10.1080/14728214.2024.2313650","url":null,"abstract":"Introduction: Autism spectrum disorder (ASD) is an early-onset disorder with a prevalence of 1% among children and reported disability-adjusted life years of 4.31 million. Irritability is a challenging behavior associated with ASD, for which medication development has lagged. More specifically, pharmacotherapy effectiveness may be limited against high adverse effects (considering side effect profiles and patient medication sensitivity); thus, the possible benefits of pharmacological interventions must be balanced against potential adverse events in each patient.Areas covered: After reviewing the neuropathophysiology of ASD-associated irritability, the benefits and tolerability of emerging medications in its treatment based on randomized controlled trials were detailed in light of mechanisms and targets of action.Expert opinion: Succeeding risperidone and aripiprazole, monotherapy with memantine may be beneficial. In addition, N-acetylcysteine, galantamine, sulforaphane, celecoxib, palmitoylethanolamide, pentoxifylline, simvastatin, minocycline, amantadine, pregnenolone, prednisolone, riluzole, propentofylline, pioglitazone, and topiramate, all adjunct to risperidone, and clonidine and methylphenidate outperformed placebo. These effects were through glutamatergic, γ-aminobutyric acidergic, inflammatory, oxidative, cholinergic, dopaminergic, and serotonergic systems. All medications were reported to be safe and tolerable. Considering sample size, follow-up, and effect size, further studies are necessary. Along with drug development, repositioning and combining existing drugs supported by the mechanism of action is recommended.","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"45-56"},"PeriodicalIF":3.4,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139650641","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"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

A review of phase II and III drugs for the treatment and management of endometriosis 治疗和管理子宫内膜异位症的二期和三期药物综述

IF 3.4 3区医学

Expert Opinion on Emerging Drugs Pub Date : 2023-12-15 DOI: 10.1080/14728214.2023.2296080

Umberto Perrone, Giulio Evangelisti, Antonio Simone Laganà, Stefano Bogliolo, Marcello Ceccaroni, Alberto Izzotti, Claudio Gustavino, Simone Ferrero, Fabio Barra

引用次数: 0

Systemic mastocytosis: 2023 update on diagnosis and management in adults. 系统性肥大细胞增多症：2023年成人诊断和治疗的最新进展。

IF 3.4 3区医学

Expert Opinion on Emerging Drugs Pub Date : 2023-12-01 Epub Date: 2023-06-06 DOI: 10.1080/14728214.2023.2221028

Alessandro Costa, Emilia Scalzulli, Ida Carmosino, Marcello Capriata, Claudia Ielo, Chiara Masucci, Mauro Passucci, Maurizio Martelli, Massimo Breccia

{"title":"Systemic mastocytosis: 2023 update on diagnosis and management in adults.","authors":"Alessandro Costa, Emilia Scalzulli, Ida Carmosino, Marcello Capriata, Claudia Ielo, Chiara Masucci, Mauro Passucci, Maurizio Martelli, Massimo Breccia","doi":"10.1080/14728214.2023.2221028","DOIUrl":"10.1080/14728214.2023.2221028","url":null,"abstract":"Introduction: Systemic mastocytosis (SM) is a complex and heterogeneous disease, characterized by the clonal accumulation of mast cells in one or more organs. In 2022 both the World Health Organization (WHO) and the International Consensus Classification (ICC) modified the diagnostic and classification criteria of SM. Moreover, the identification of new clinical and molecular variables has improved prognostic tools and led to increasingly individualized therapeutic strategies.Areas covered: The aim of this review is to present the updates introduced by the International Consensus Classification in diagnostic criteria of SM. In addition, we report the latest data available from the most important clinical trials in patients both with non-advanced and advanced disease, including elenestinib and bezuclastinib.Expert opinion: Diagnosis and classification of SM has evolved over years. The most recent WHO and ICC classification improved SM diagnostic work-up, providing clinicians with a clear and simplified diagnostic scheme. New approved targeted therapies such as midostaurin and avapritinib modified the treatment paradigm in patients in advanced stage, and next-generation inhibitors actually investigated in clinical trials are expected in the next future.","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"153-165"},"PeriodicalIF":3.4,"publicationDate":"2023-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9577319","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"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: 1

Emerging drugs for dementia with Lewy Bodies: a review of Phase II & III trials. 治疗路易体痴呆症的新兴药物：II期和III期试验综述。

IF 3.4 3区医学

Expert Opinion on Emerging Drugs Pub Date : 2023-12-01 Epub Date: 2023-08-08 DOI: 10.1080/14728214.2023.2244425

Magdalena I Tolea, Reem Ezzeddine, Simone Camacho, James E Galvin

{"title":"Emerging drugs for dementia with Lewy Bodies: a review of Phase II & III trials.","authors":"Magdalena I Tolea, Reem Ezzeddine, Simone Camacho, James E Galvin","doi":"10.1080/14728214.2023.2244425","DOIUrl":"10.1080/14728214.2023.2244425","url":null,"abstract":"Introduction: Despite faster cognitive decline and greater negative impact on patients and family caregivers, drug development efforts in Dementia with Lewy Bodies (DLB) fall behind those for Alzheimer's Disease (AD). Current off-label drug DLB treatment options are limited to symptomatic agents developed to address cognitive deficits in AD, motor deficits in Parkinson's Disease, or behavioral symptoms in psychiatric disease. Aided by recent improvements in DLB diagnosis, a new focus on the development of disease-modifying agents (DMA) is emerging.Areas covered: Driven by evidence supporting different pathological mechanisms in DLB and PDD, this review assesses the evidence on symptomatic drug treatments and describes current efforts in DMA development in DLB. Specifically, our goals were to: (1) review evidence supporting the use of symptomatic drug treatments in DLB; (2) review the current DMA pipeline in DLB with a focus on Phase II and III clinical trials; and (3) identify potential issues with the development of DMA in DLB. Included in this review were completed and ongoing drug clinical trials in DLB registered on ClinicalTrials.gov (no time limits set for the search) or disseminated at the 2023 international conference on Clinical Trials in AD. Drug clinical trials registered in non-US clinical trial registries were not included.Expert opinion: Adoption of current symptomatic drug treatments used off-label in DLB relied on efficacy of benefits in other disorders rather than evidence from randomized controlled clinical trials. Symptoms remain difficult to manage. Several DMA drugs are currently being evaluated as either repurposing candidates or novel small molecules. Continued improvement in methodological aspects including development of DLB-specific outcome measures and biomarkers is needed to move the field of DMA drug development forward.","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"167-180"},"PeriodicalIF":3.4,"publicationDate":"2023-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9951464","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"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