Expert Opinion on Emerging Drugs最新文献

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Current and emerging drug treatment strategies to tackle sickle cell anemia. 应对镰状细胞性贫血的现有和新兴药物治疗策略。
IF 2.7 3区 医学
Expert Opinion on Emerging Drugs Pub Date : 2024-12-01 Epub Date: 2024-07-22 DOI: 10.1080/14728214.2024.2379260
Yogindra Persaud, Alexis Leonard, Parul Rai
{"title":"Current and emerging drug treatment strategies to tackle sickle cell anemia.","authors":"Yogindra Persaud, Alexis Leonard, Parul Rai","doi":"10.1080/14728214.2024.2379260","DOIUrl":"10.1080/14728214.2024.2379260","url":null,"abstract":"<p><strong>Introduction: </strong>Since its discovery in the early 1900s, sickle cell disease (SCD) has contributed significantly to the scientific understanding of hemoglobin and hemoglobinopathies. Despite this, now almost a century later, optimal medical management and even curative options remain limited. Encouragingly, in the last decade, there has been a push toward advancing the care for individuals with SCD and a diversifying interest in options to manage this disorder.</p><p><strong>Areas covered: </strong>Here, we review the current state of disease modifying therapies for SCD including fetal hemoglobin inducers, monoclonal antibodies, anti-inflammatory modulators, and enzyme activators. We also discuss current curative strategies with specific interest in transformative gene therapies.</p><p><strong>Expert opinion: </strong>SCD is a chronic, progressive disease that despite a century of clinical description, only now is seeing a growth and advance in therapeutic options to improve the lifespan and quality of life for individuals with SCD. We anticipate newly designed and even repurposed therapies that may work as a single agent or combination agents to tackle the progression of SCD. The vast majority of individuals living with SCD are unlikely to receive gene therapy, therefore improved disease management is critical even for those that may ultimately chose to pursue a potentially curative strategy.</p>","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"327-346"},"PeriodicalIF":2.7,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141579372","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 comprehensive review of phase 2/3 trials in osteoarthritis: an expert opinion. 骨关节炎 2/3 期试验全面回顾:专家意见。
IF 2.7 3区 医学
Expert Opinion on Emerging Drugs Pub Date : 2024-12-01 Epub Date: 2024-08-05 DOI: 10.1080/14728214.2024.2386174
Liyang Pan, Lydia Nagib, Sujata Ganguly, Arumugam Moorthy, Hasan Tahir
{"title":"A comprehensive review of phase 2/3 trials in osteoarthritis: an expert opinion.","authors":"Liyang Pan, Lydia Nagib, Sujata Ganguly, Arumugam Moorthy, Hasan Tahir","doi":"10.1080/14728214.2024.2386174","DOIUrl":"10.1080/14728214.2024.2386174","url":null,"abstract":"<p><strong>Introduction: </strong>Osteoarthritis (OA) is a chronic, degenerative, and debilitating disease associated with significant long-term morbidity and disability. The pathogenesis of OA is not completely understood but involves an interplay between environmental risk factors, joint mechanics, abnormal pain pathways and upregulation of inflammatory signaling pathways. Current therapeutic options for patients are limited to conservative management, minimal pharmacological options or surgical management, with significant caveats to all approaches.</p><p><strong>Areas covered: </strong>In this review, we have set out to investigate current phase II/III clinical trials by undertaking a PubMed search. Examined clinical trials have explored a myriad of potential therapeutics from conventional disease-modifying anti-rheumatic drugs and biologics usually used in the treatment of inflammatory arthritides, to more novel approaches targeting inflammatory pathways implicated in OA, cartilage degeneration or pain pathways.</p><p><strong>Expert opinion: </strong>Unfortunately, most completed phase II/III clinical trials have shown little impact on patient pain scores, with the exception of the traditional DMARD methotrexate and Sprifermin. Methotrexate has been shown to be beneficial when used in the correct patient cohort (MRI proven synovitis). Sprifermin has the longest follow-up data of 5 years and has been shown to reduce loss of MRI-measured cartilage thickness and pain scores.</p>","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"347-359"},"PeriodicalIF":2.7,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141859482","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
ONC201 (Dordaviprone): review of evidence to date in diffuse midline glioma, hope or hype? ONC201(多达维酮):弥漫性中线胶质瘤迄今为止的证据回顾,希望还是炒作?
IF 2.7 3区 医学
Expert Opinion on Emerging Drugs Pub Date : 2024-12-01 Epub Date: 2024-11-21 DOI: 10.1080/14728214.2024.2426649
Jordan R Hansford, Neevika Manoharan, Gauthier Bouche, Vijay Ramaswamy, Nada Jabado, Eric Bouffet
{"title":"ONC201 (Dordaviprone): review of evidence to date in diffuse midline glioma, hope or hype?","authors":"Jordan R Hansford, Neevika Manoharan, Gauthier Bouche, Vijay Ramaswamy, Nada Jabado, Eric Bouffet","doi":"10.1080/14728214.2024.2426649","DOIUrl":"10.1080/14728214.2024.2426649","url":null,"abstract":"","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"321-325"},"PeriodicalIF":2.7,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142643379","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 immunotherapies in the Hodgkin lymphoma armamentarium. 霍奇金淋巴瘤武器库中的新兴免疫疗法。
IF 2.7 3区 医学
Expert Opinion on Emerging Drugs Pub Date : 2024-09-01 Epub Date: 2024-05-06 DOI: 10.1080/14728214.2024.2349083
Michael A Spinner, Ranjana H Advani
{"title":"Emerging immunotherapies in the Hodgkin lymphoma armamentarium.","authors":"Michael A Spinner, Ranjana H Advani","doi":"10.1080/14728214.2024.2349083","DOIUrl":"10.1080/14728214.2024.2349083","url":null,"abstract":"<p><strong>Introduction: </strong>Brentuximab vedotin and PD-1 inhibitors have improved outcomes for classic Hodgkin lymphoma (cHL), but better therapies are needed for patients who relapse after these agents. Based on an improved understanding of cHL biology, there is a robust pipeline of novel therapies in development. In this review, we highlight emerging immunotherapeutic agents and combinations for cHL.</p><p><strong>Areas covered: </strong>We review clinical trials of novel PD-1/PD-L1 inhibitors beyond FDA-approved agents, checkpoint inhibitors targeting CTLA-4, LAG-3, TIM-3, TIGIT, and CD47/SIRPα, PD-1 inhibitor combinations with immunomodulatory agents and epigenetic modifying therapies, antibody-drug conjugates, bispecific antibodies, and cellular therapies including anti-CD30 CAR-T and allogeneic NK cell therapy. We review the key safety and efficacy data from published phase 1-2 studies and highlight trials in progress, including the first phase 3 trial for PD-1 inhibitor-refractory cHL.</p><p><strong>Expert opinion: </strong>Many novel immunotherapies hold great promise in cHL. Rational combinations with existing agents and next-generation antibody and CAR-T constructs may improve response rates and durability. Identifying biomarkers of response to these immunotherapies and using more sensitive tools to assess response, such as circulating tumor DNA, may further inform treatment decisions and enable a precision medicine approach in the future.</p>","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"263-275"},"PeriodicalIF":2.7,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140862631","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 therapies for treatment of agitation, psychosis, or apathy in Alzheimer's disease. 治疗阿尔茨海默氏症患者躁动、精神错乱或冷漠的新兴疗法。
IF 2.7 3区 医学
Expert Opinion on Emerging Drugs Pub Date : 2024-09-01 Epub Date: 2024-06-11 DOI: 10.1080/14728214.2024.2363215
Hui Jue Wang, Arun Chinna-Meyyappan, Oriel J Feldman, Krista L Lanctôt
{"title":"Emerging therapies for treatment of agitation, psychosis, or apathy in Alzheimer's disease.","authors":"Hui Jue Wang, Arun Chinna-Meyyappan, Oriel J Feldman, Krista L Lanctôt","doi":"10.1080/14728214.2024.2363215","DOIUrl":"10.1080/14728214.2024.2363215","url":null,"abstract":"<p><strong>Introduction: </strong>Agitation, psychosis, and apathy are prevalent and highly distressing neuropsychiatric symptoms (NPS) of Alzheimer's disease (AD) that have been linked to numerous negative outcomes, including increased mortality, worsened cognitive decline, and caregiver burden. Current treatments for AD-associated agitation, namely atypical antipsychotics, provide some benefits but may increase the risk of serious adverse events and death. Meanwhile, no pharmacotherapies have been approved by regulatory agencies for the treatment of psychosis or apathy in AD. Over the past decade, many new and repurposed drugs have emerged as potential therapeutic options for managing these challenging NPS.</p><p><strong>Areas covered: </strong>This review aims to provide a comprehensive summary of pharmacotherapies that have recently been investigated in phase 2 and 3 clinical trials for the treatment of agitation, psychosis, or apathy in AD.</p><p><strong>Expert opinion: </strong>Novel atypical antipsychotics, serotonergic antidepressants, cannabinoids, and dextromethorphan combination drugs have shown promising results for alleviating agitation. Pimavanserin appears to be the most effective emerging therapy for psychosis, while methylphenidate has demonstrated good efficacy for apathy. Further research on biomarkers of NPS severity and treatment response, as well as continued improvements in methodological approaches are needed to advance the field.</p>","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"289-303"},"PeriodicalIF":2.7,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141186324","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 short bowel syndrome. 治疗短肠综合征的新兴药物。
IF 2.7 3区 医学
Expert Opinion on Emerging Drugs Pub Date : 2024-09-01 Epub Date: 2024-05-23 DOI: 10.1080/14728214.2024.2357567
Tristan Frau, Myriam El Khatib, Brune De Dreuille, Lore Billiauws, Alexandre Nuzzo, Francisca Joly
{"title":"Emerging drugs for the treatment of short bowel syndrome.","authors":"Tristan Frau, Myriam El Khatib, Brune De Dreuille, Lore Billiauws, Alexandre Nuzzo, Francisca Joly","doi":"10.1080/14728214.2024.2357567","DOIUrl":"10.1080/14728214.2024.2357567","url":null,"abstract":"<p><strong>Introduction: </strong>SBS is a rare and disabling condition. The standard management is based on diet optimization with parenteral supplementation. In addition, glucagon-like peptide-2 (GLP-2)analogs, have shown promising results as disease-modifying therapies for SBS.</p><p><strong>Areas covered: </strong>Short bowel syndrome (SBS) is defined as a reduction in functional intestinal length to less than 200 cm, leading to intestinal failure (IF) leading to malnutrition and parenteral support dependency. This review discusses the current management of SBS-CIFpatients, the place of GLP-2 analog treatment in terms of efficacy, safety and availability, and the new perspectives opened by the use of enterohormones.</p><p><strong>Expert opinion: </strong>Clinical trials and real-world experience demonstrated that Teduglutide   reduces dependence on parenteral support and has a place in the management of patients with SBS-CIF.  The use of Teduglutide should be discussed in patients stabilized after resection and its introduction requires the advice of an expert center capable of assessing the benefit-risk ratio. The complex, individualized management of SBS-C IF requires theexpertise of a specialized IF center which a multidisciplinary approach. The arrival of new treatments will call for new therapeutic strategies, and the question of how to introduce and monitor them will represent a new therapeutic challenge.</p>","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"277-288"},"PeriodicalIF":2.7,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140957158","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 benign prostatic hyperplasia: a 2023 update. 治疗良性前列腺增生症的新药:2023 年更新。
IF 2.7 3区 医学
Expert Opinion on Emerging Drugs Pub Date : 2024-09-01 Epub Date: 2024-06-14 DOI: 10.1080/14728214.2024.2363213
Joshua Winograd, Nikit Venishetty, Alia Codelia-Anjum, Naeem Bhojani, Dean Elterman, Kevin C Zorn, Alexis Te, Bilal Chughtai
{"title":"Emerging drugs for the treatment of benign prostatic hyperplasia: a 2023 update.","authors":"Joshua Winograd, Nikit Venishetty, Alia Codelia-Anjum, Naeem Bhojani, Dean Elterman, Kevin C Zorn, Alexis Te, Bilal Chughtai","doi":"10.1080/14728214.2024.2363213","DOIUrl":"10.1080/14728214.2024.2363213","url":null,"abstract":"<p><strong>Introduction: </strong>Benign prostatic hyperplasia (BPH) is a condition that affects over 50% of men as they enter their fifth decade of life, often leading to lower urinary tract symptoms (LUTS). Primary treatment options include alpha blockers, 5-alpha reductase inhibitors, and phosphodiesterase-5 inhibitors. However, these medications can have some side effects, and there is a noticeable dearth of information addressing the long-term use of these medications. Thus, the exploration of all treatment modalities helps ensure patients receive personalized and effective care. Consequently, the primary objective of this review is to identify potential emerging medications for the treatment of BPH.</p><p><strong>Areas covered: </strong>We conducted an extensive review of articles discussing pharmacotherapy for BPH spanning the last 15 years. Our information gathering process involved Scopus, PubMed-MEDLINE, Cochrane, Wiley Online Library Google Scholar, ClinicalTrials.gov, and the PharmaProjects database. This approach ensures that readers gain an in-depth knowledge of the existing therapeutic agents as well as promising avenues for managing BPH.</p><p><strong>Expert opinion: </strong>BPH treatment targets a patient's specific constellation of symptoms. Therefore, a broad knowledge base encompassing various treatment options is paramount in ensuring optimal treatment. Looking forward, the emphasis on personalization promises to reshape the landscape of BPH treatment and improve patient outcomes.</p>","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"205-217"},"PeriodicalIF":2.7,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141261337","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
An extract of phase II and III trials on recent developments in managing neuropathic pain syndromes: diabetic peripheral neuropathy, trigeminal neuralgia, and postherpetic neuralgia. 关于治疗神经性疼痛综合症(糖尿病周围神经病变、三叉神经痛和带状疱疹后神经痛)最新进展的 II 期和 III 期试验摘录。
IF 2.7 3区 医学
Expert Opinion on Emerging Drugs Pub Date : 2024-06-01 Epub Date: 2024-02-29 DOI: 10.1080/14728214.2024.2323193
William C Upshaw, Lenise G Soileau, Nicholas R Storey, Kassady A Perkinson, Patrick M Luther, Noah J Spillers, Christopher L Robinson, Benjamin C Miller, Shahab Ahmadzadeh, Omar Viswanath, Sahar Shekoohi, Alan D Kaye
{"title":"An extract of phase II and III trials on recent developments in managing neuropathic pain syndromes: diabetic peripheral neuropathy, trigeminal neuralgia, and postherpetic neuralgia.","authors":"William C Upshaw, Lenise G Soileau, Nicholas R Storey, Kassady A Perkinson, Patrick M Luther, Noah J Spillers, Christopher L Robinson, Benjamin C Miller, Shahab Ahmadzadeh, Omar Viswanath, Sahar Shekoohi, Alan D Kaye","doi":"10.1080/14728214.2024.2323193","DOIUrl":"10.1080/14728214.2024.2323193","url":null,"abstract":"<p><strong>Introduction: </strong>Neuropathic pain (NP) conditions involve lesions to the somatosensory nervous system leading to chronic and debilitating pain. Many patients suffering from NP utilize pharmacological treatments with various drugs that seek to reduce pathologic neuronal states. However, many of these drugs show poor efficacy as well as cause significant adverse effects. Because of this, there is a major need for the development of safer and more efficacious drugs to treat NP.</p><p><strong>Areas covered: </strong>In this review, we analyzed current treatments being developed for a variety of NP conditions. Specifically, we sought drugs in phase II/III clinical trials with indications for NP conditions. Various databases were searched including Google Scholar, PubMed, and clinicaltrials.gov.</p><p><strong>Expert opinion: </strong>All the mentioned targets for treatments of NP seem to be promising alternatives for existing treatments that often possess poor side effect profiles for patients. However, gene therapy potentially offers the unique ability to inject a plasmid containing growth factors leading to nerve growth and repair. Because of this, gene therapy appears to be the most intriguing new treatment for NP.</p>","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"103-112"},"PeriodicalIF":2.7,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139971526","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 hepatic fibrosis on nonalcoholic steatohepatitis. 治疗非酒精性脂肪性肝炎肝纤维化的新药。
IF 2.7 3区 医学
Expert Opinion on Emerging Drugs Pub Date : 2024-06-01 Epub Date: 2024-03-25 DOI: 10.1080/14728214.2024.2328036
Michihiro Iwaki, Masato Yoneda, Naohiro Wada, Tomohiro Otani, Takashi Kobayashi, Asako Nogami, Satoru Saito, Atsushi Nakajima
{"title":"Emerging drugs for the treatment of hepatic fibrosis on nonalcoholic steatohepatitis.","authors":"Michihiro Iwaki, Masato Yoneda, Naohiro Wada, Tomohiro Otani, Takashi Kobayashi, Asako Nogami, Satoru Saito, Atsushi Nakajima","doi":"10.1080/14728214.2024.2328036","DOIUrl":"10.1080/14728214.2024.2328036","url":null,"abstract":"<p><strong>Introduction: </strong>Approved drug therapies for nonalcoholic steatohepatitis (NASH) are lacking, for which various agents are currently being tested in clinical trials. Effective drugs for liver fibrosis, the factor most associated with prognosis in NASH, are important.</p><p><strong>Areas covered: </strong>This study reviewed the treatment of NASH with a focus on the effects of existing drugs and new drugs on liver fibrosis.</p><p><strong>Expert opinion: </strong>Considering the complex pathophysiology of fibrosis in NASH, drug therapy may target multiple pathways. The method of assessing fibrosis is important when considering treatment for liver fibrosis in NASH. The Food and Drug Administration considers an important fibrosis endpoint to be histological improvement in at least one fibrosis stage while preventing worsening of fatty hepatitis. To obtain approval as a drug for NASH, efficacy needs to be demonstrated on endpoints such as liver-related events and myocardial infarction. Among the current therapeutic agents for NASH, thiazolidinedione, sodium-glucose co-transporter 2, and selective peroxisome proliferator-activated receptors α modulator have been reported to be effective against fibrosis, although further evidence is required. The effects of pan-peroxisome proliferator-activated receptors, obeticholic acid, and fibroblast growth factor-21 analogs on liver fibrosis in the development stage therapeutics for NASH are of particular interest.</p>","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"127-137"},"PeriodicalIF":2.7,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140101402","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
Evaluating emerging drugs in phase II & III for the treatment of amyotrophic lateral sclerosis. 评估治疗肌萎缩性脊髓侧索硬化症的二期和三期新药。
IF 2.7 3区 医学
Expert Opinion on Emerging Drugs Pub Date : 2024-06-01 Epub Date: 2024-03-22 DOI: 10.1080/14728214.2024.2333420
Xiaoyan Li, Richard Bedlack
{"title":"Evaluating emerging drugs in phase II & III for the treatment of amyotrophic lateral sclerosis.","authors":"Xiaoyan Li, Richard Bedlack","doi":"10.1080/14728214.2024.2333420","DOIUrl":"10.1080/14728214.2024.2333420","url":null,"abstract":"<p><strong>Introduction: </strong>Amyotrophic Lateral Sclerosis is a rapidly progressive motor neuron disorder causing severe disability and premature death. Owing to the advances in uncovering ALS pathophysiology, efficient clinical trial design and research advocacy program, several disease-modifying drugs have been approved for treating ALS. Despite this progress, ALS remains a rapidly disabling and life shortening condition. There is a critical need for more effective therapies.</p><p><strong>Areas covered: </strong>Here, we reviewed the emerging ALS therapeutics undergoing phase II & III clinical trials. To identify the investigational drugs, we searched ALS and phase II/III trials that are active and recruiting or not yet recruiting on clinicaltrials.gov and Pharmaprojects database.</p><p><strong>Expert opinion: </strong>The current pipeline is larger and more diverse than ever, with drugs targeting potential genetic and retroviral causes of ALS and drugs targeting a wide array of downstream pathways, including RNA metabolism, protein aggregation, integrated stress response and neuroinflammation.We remain most excited about those that target direct causes of ALS, e.g. antisense oligonucleotides targeting causative genes. Drugs that eliminate abnormal protein aggregates are also up-and-coming. Eventually, because of the heterogeneity of ALS pathophysiology, biomarkers that determine which biological events are most important for an individual ALS patient are needed.</p>","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"93-102"},"PeriodicalIF":2.7,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140184153","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
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