Diego Gómez-Puerto, Carlo M Cicala, Claudia Valverde, César Serrano
{"title":"Emerging treatments for sarcoma: from 2024 onward.","authors":"Diego Gómez-Puerto, Carlo M Cicala, Claudia Valverde, César Serrano","doi":"10.1080/14728214.2025.2460525","DOIUrl":"10.1080/14728214.2025.2460525","url":null,"abstract":"<p><strong>Introduction: </strong>Sarcomas are a rare and diverse group of mesenchymal-origin solid tumors, constituting only 1% of adult malignancies and classified into soft tissue and bone sarcomas. For localized diseases, surgery and radiotherapy remain the cornerstone treatments. However, systemic options for advanced stages are limited, with an overall survival of approximately 20 months. There is an urgent need to refine clinical trial designs and evaluate novel therapies to improve patient outcomes.</p><p><strong>Areas covered: </strong>This review summarizes recent advancements in sarcoma clinical trials, highlighting promising therapies that may reshape treatment paradigms. It also examines insights from comprehensive genomic profiling studies, which are paving the way for next-generation therapeutic approaches.</p><p><strong>Expert opinion: </strong>The heterogeneity and rarity of sarcomas present substantial challenges to the development and regulatory approval of effective therapies. Recent progress in the field has introduced innovative approaches, including novel chemotherapeutic agents, targeted therapies, and immunotherapeutics. However, despite these advancements, significant hurdles persist. While these strategies have demonstrated some success, the overall improvements in survival have often been modest, irrespective of the therapeutic modality. This underscores critical concerns regarding the true cost-benefit balance and the potential for adverse effects, particularly when evaluated over extended follow-up periods.</p>","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"1-18"},"PeriodicalIF":2.7,"publicationDate":"2025-02-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143058569","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}
Daniela Pastore, Chiara Lupia, Maria D'Amato, Andrea Bruni, Eugenio Garofalo, Federico Longhini, Luca Gallelli, Alessandro Vatrella, Girolamo Pelaia, Corrado Pelaia
{"title":"Emerging biological treatments for asthma.","authors":"Daniela Pastore, Chiara Lupia, Maria D'Amato, Andrea Bruni, Eugenio Garofalo, Federico Longhini, Luca Gallelli, Alessandro Vatrella, Girolamo Pelaia, Corrado Pelaia","doi":"10.1080/14728214.2025.2460529","DOIUrl":"10.1080/14728214.2025.2460529","url":null,"abstract":"<p><strong>Introduction: </strong>Severe asthma is a chronic airway disease characterized by many pathomechanisms known as endotypes. Biological therapies targeting severe asthma endotypes have significantly improved the treatment of this disease, thus remarkably bettering patient quality of life.</p><p><strong>Areas covered: </strong>This review aims to describe current biological therapies for severe asthma, highlighting emerging ones. Several studies have confirmed the beneficial effects of currently available monoclonal antibodies targeting immunoglobulin E (IgE), interleukin-5 (IL-5) or its receptor, and interleukin-4 (IL-4)/interleukin-13 (IL-13) receptors (IL-4R/IL-13R). However, patients with T2-low asthma are not eligible for the above biological therapies.</p><p><strong>Expert opinion: </strong>New treatments are now moving toward targeting the upstream pathways of asthma pathogenesis, coordinated by innate cytokines such as alarmins. These key proinflammatory mediators orchestrate the activation of complex cellular networks including both innate and adaptive immune responses. Alarmins include thymic stromal lymphopoietin (TSLP), interleukin-25 (IL-25), and interleukin-33 (IL-33), which are released from injured airway epithelial cells. TSLP and the other alarmins are suitable targets of biological therapies which are effective for add-on treatment of type 2 asthma. Moreover, anti-alarmin monoclonal antibodies can be also beneficial for patients with T2-low, poorly controlled severe asthma.</p>","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"1-11"},"PeriodicalIF":2.7,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143052103","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}
Mohammad Hassan Hodroj, Joseph Klim, Nicole Charbel, Ali Taher
{"title":"Mitapivat: a step forward across different hemolytic diseases.","authors":"Mohammad Hassan Hodroj, Joseph Klim, Nicole Charbel, Ali Taher","doi":"10.1080/14728214.2025.2458063","DOIUrl":"https://doi.org/10.1080/14728214.2025.2458063","url":null,"abstract":"","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"1-3"},"PeriodicalIF":2.7,"publicationDate":"2025-01-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143064867","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}
{"title":"FcRn inhibitors in the context of myasthenia gravis.","authors":"Deepak Menon, Vinaya Bhandari","doi":"10.1080/14728214.2025.2458061","DOIUrl":"10.1080/14728214.2025.2458061","url":null,"abstract":"","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"1-4"},"PeriodicalIF":2.7,"publicationDate":"2025-01-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143052104","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}
{"title":"Muscarinic M1 and M4 agents as treatments for schizophrenia: what do they do and who do they do it for?","authors":"Philip D Harvey","doi":"10.1080/14728214.2025.2458053","DOIUrl":"10.1080/14728214.2025.2458053","url":null,"abstract":"","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"1-5"},"PeriodicalIF":2.7,"publicationDate":"2025-01-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143002924","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}
Kevin Mok, Olivia Chen, Johnny Yau, Landon L Chan, Stephen L Chan
{"title":"Systemic therapy for child-pugh B patients with hepatocellular carcinoma.","authors":"Kevin Mok, Olivia Chen, Johnny Yau, Landon L Chan, Stephen L Chan","doi":"10.1080/14728214.2025.2456774","DOIUrl":"https://doi.org/10.1080/14728214.2025.2456774","url":null,"abstract":"","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":""},"PeriodicalIF":2.7,"publicationDate":"2025-01-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143002928","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}
Kyle Valentino, Kayla M Teopiz, Sabrina Wong, Melanie C Zhang, Gia Han Le, Hayun Choi, Hana Ballum, Christine Dri, William Cheung, Roger S McIntyre
{"title":"Seltorexant for major depressive disorder.","authors":"Kyle Valentino, Kayla M Teopiz, Sabrina Wong, Melanie C Zhang, Gia Han Le, Hayun Choi, Hana Ballum, Christine Dri, William Cheung, Roger S McIntyre","doi":"10.1080/14728214.2025.2452514","DOIUrl":"10.1080/14728214.2025.2452514","url":null,"abstract":"<p><strong>Introduction: </strong>Preclinical and clinical pharmacologic evidence indicates that orexin systems are relevant to sleep-wake cycle regulation and dimensions of reward and cognition, providing the basis for hypothesizing that they may be effective as therapeutics in mental disorders. Due to the limited efficacy and tolerability profiles of existing treatments for Major Depressive Disorder (MDD), investigational compounds in novel treatment classes are needed; seltorexant, an orexin receptor antagonist, is a potential new treatment currently under investigation.</p><p><strong>Areas covered: </strong>Mechanisms implicated in MDD, including reward and sleep, are first overviewed. Then, the safety, tolerability, and efficacy profiles of seltorexant and the wider context of orexin receptor antagonism for depression are discussed in focus. Preclinical and clinical data are also discussed. PubMed, Medline, Cochrane Library, Embase, Scopus, and Web of Science were systematically searched from inception to 10 October 2024, in accordance with PRISMA guidelines.</p><p><strong>Expert opinion: </strong>Early clinical evidence suggests that seltorexant is effective in treating MDD, both in individuals diagnosed with insomnia and those not, although greater antidepressant effects are observed in individuals with severe sleep disturbance. Results from large phase III clinical trials are needed to confirm efficacy and safety.</p>","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"1-11"},"PeriodicalIF":2.7,"publicationDate":"2025-01-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142947157","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}
Davide Politano, Davide Tonduti, Roberta Battini, Elisa Fazzi, Simona Orcesi
{"title":"Exploring emerging JAK inhibitors in the treatment of Aicardi-Goutières syndrome.","authors":"Davide Politano, Davide Tonduti, Roberta Battini, Elisa Fazzi, Simona Orcesi","doi":"10.1080/14728214.2024.2445508","DOIUrl":"10.1080/14728214.2024.2445508","url":null,"abstract":"<p><strong>Introduction: </strong>Aicardi-Goutières syndrome (AGS) is a genetically heterogeneous monogenic autoinflammatory disorder classified as an 'interferonopathy'. Nine genes have been implicated in AGS, encoding proteins involved in nucleic acid clearance, repair, sensing, or histone pre-mRNA processing. Dysregulation in these pathways leads to excessive type I interferon production, the primary driver of the disease. AGS typically presents with early-life neurological regression, followed by stabilization with varying degrees of neurological impairment and common extra-neurological features, such as chilblains. Advances in understanding AGS pathogenesis have enabled the development of new therapies, with JAK inhibitors emerging as the most studied option for reducing interferon-mediated effects.</p><p><strong>Areas covered: </strong>This review discusses the clinical features, genetic basis, and molecular pathways of AGS while tracing the evolution of its therapeutic strategies. Particular emphasis is placed on JAK inhibitors, which target proteins activated by type I interferons, providing a novel direction in treatment.</p><p><strong>Expert opinion: </strong>Inhibitors effectively reduce extra-neurological symptoms in AGS, though their impact on neurological outcomes remains unclear. The unknown natural history of AGS limits treatment evaluation. Despite growing insights, key aspects of pathogenesis and treatment optimization - including timing, administration, and long-term effects - remain unresolved, highlighting the need for further research.</p>","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"1-19"},"PeriodicalIF":2.7,"publicationDate":"2024-12-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142863686","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}
Heli A Patel, Linh Tran, Ralina Karagenova, Steven R Feldman
{"title":"Monoclonal antibodies in phase II and III trials for moderate to severe atopic dermatitis.","authors":"Heli A Patel, Linh Tran, Ralina Karagenova, Steven R Feldman","doi":"10.1080/14728214.2024.2416114","DOIUrl":"10.1080/14728214.2024.2416114","url":null,"abstract":"<p><strong>Introduction: </strong>Atopic dermatitis (AD) is a prevalent chronic inflammatory skin disease that significantly affects quality of life and mental health, especially in children. Traditional treatments include chemotherapeutics, topical corticosteroids, and immunomodulatory agents, but recent advances have introduced novel monoclonal antibody therapies. Through this comprehensive review paper, we aim to discuss these therapeutic options and their role in treating atopic dermatitis.</p><p><strong>Areas covered: </strong>A comprehensive search of the NIH Clinical Trials database was conducted from September 2023 to January 2024, focusing on phase 2 and 3 trials for AD treatments. Trials were filtered using keywords such as 'atopic dermatitis,' 'monoclonal antibody,' and 'phase 2/3.' Out of 25 trials analyzed, 11 were in phase 2 and 14 in phase 3. Only U.S.-based trials comparing novel therapies to placebo were included. In addition to the clinical trial database, we utilized the companies' websites and relevant abstracts to gather the latest results.</p><p><strong>Expert opinion: </strong>Currently investigated monoclonal antibodies have the ability to transform management by targeting specific mediators implicated in the inflammatory pathway of AD. The results of Phase II and III trials for monoclonal antibodies demonstrated strong therapeutic potential with significant reductions in EASI scores and represent a promising new targeted treatment option.</p>","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"361-368"},"PeriodicalIF":2.7,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142399909","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}
Leonardo Stella, Clemence Hollande, Yasmina Ben Merabet, Hugo Fakhouri, Vincent Leclerc, Francesca Romana Ponziani, Mohamed Bouattour
{"title":"Promising PD-1 antagonists for liver cancer: an evaluation of phase II and III results.","authors":"Leonardo Stella, Clemence Hollande, Yasmina Ben Merabet, Hugo Fakhouri, Vincent Leclerc, Francesca Romana Ponziani, Mohamed Bouattour","doi":"10.1080/14728214.2024.2430493","DOIUrl":"10.1080/14728214.2024.2430493","url":null,"abstract":"<p><strong>Introduction: </strong>Hepatocellular carcinoma (HCC), the most common primary liver cancer, is a major cause of cancer-related morbidity and mortality. Limited treatment options for advanced stages highlight the need for effective therapies.</p><p><strong>Areas covered: </strong>This review explores immune checkpoint inhibitors (ICIs), specifically PD-1, PD-L1, and CTLA-4 inhibitors, as emerging treatments for advanced HCC. It discusses data from phase II and III trials evaluating ICI combinations with tyrosine kinase inhibitors (TKIs), anti-angiogenic agents, and locoregional treatments like Transarterial Chemoembolization (TACE). Clinical outcomes, including progression-free survival and response rates, were analyzed alongside the incidence and management of immune-related adverse events (irAEs). A systematic review approach ensured comprehensive, high-quality study inclusion.</p><p><strong>Expert opinion: </strong>ICI-based therapies and their combinations are transforming advanced HCC treatment, offering improved outcomes and potential survival benefits. However, these therapies need optimization in sequencing and selection, particularly considering variations in liver function and disease stage. Effective management of adverse effects is critical to maximize clinical benefits. Further research is required to develop personalized strategies, tailoring treatments to patient-specific factors and enhancing safety and effectiveness in HCC management.</p>","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"369-382"},"PeriodicalIF":2.7,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142643385","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}