{"title":"Navigating the landscape of immune checkpoint inhibitors and novel immunotherapies in melanoma: long-term outcomes, progress, and challenges.","authors":"Sowmya Cheruvu, David McMahon, James Larkin","doi":"10.1080/14712598.2025.2456485","DOIUrl":"https://doi.org/10.1080/14712598.2025.2456485","url":null,"abstract":"<p><strong>Introduction: </strong>Melanoma has become the poster child for transformative outcomes in advanced malignancy from the use of immunotherapy over the last 10-15 years with median survival improving from ~ 1 to > 5 years. With the increasing repertoire of immune checkpoint inhibitors (ICI) and other novel immunotherapeutic approaches, integrating and sequencing treatments to create new paradigms has gained prominence, with focus on optimizing toxicity management and complex scenarios such as immunotherapy resistance, brain metastases, fertility, and duration of follow-up.</p><p><strong>Areas covered: </strong>In this review, we summarize the progress and emerging evidence in melanoma treatments to date and consider management and possible future directions to improve outcomes for above-mentioned specific patient cohorts.</p><p><strong>Expert opinion: </strong>Personalized care with integration of novel prognostic and predictive biomarkers is the way forward in tailoring not only patient selection and choice of therapy, but also duration of treatment and surveillance to allow for early recurrence detection and access to newer therapies such as tumor infiltrating lymphocytes (TIL) to maximize the curative fraction of melanoma patients. Further research is needed in optimizing ICI and other immunotherapy toxicity management, including reducing steroid exposure for better patient outcomes and preserving quality of life.</p>","PeriodicalId":12084,"journal":{"name":"Expert Opinion on Biological Therapy","volume":" ","pages":"1-12"},"PeriodicalIF":3.6,"publicationDate":"2025-02-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143079091","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":"How should we approach salvage therapy in advanced renal cell carcinoma after first-line immunotherapy combinations?","authors":"Paul Sackstein, Michael B Atkins","doi":"10.1080/14712598.2025.2459193","DOIUrl":"10.1080/14712598.2025.2459193","url":null,"abstract":"","PeriodicalId":12084,"journal":{"name":"Expert Opinion on Biological Therapy","volume":" ","pages":"1-4"},"PeriodicalIF":3.6,"publicationDate":"2025-02-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143046104","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":"Treating episodic migraine with precision: the evolving landscape of targeted therapies driven by insights in disease biology.","authors":"Robert Fleischmann, Sebastian Strauß, Uwe Reuter","doi":"10.1080/14712598.2025.2456464","DOIUrl":"10.1080/14712598.2025.2456464","url":null,"abstract":"<p><strong>Introduction: </strong>Migraine is a disabling neurological disorder with a complex neurobiology. It appears as a cyclic disorder of sensory processing, affecting multiple systems beyond nociception. Overlapping mechanisms, including dysfunctional processing of sensory input from brain structures are involved in the generation of attacks.</p><p><strong>Areas covered: </strong>This review provides a comprehensive synthesis on migraine neurobiology, which was additionally informed by search of research databases (PubMed, ClinicalTrials.gov). Findings from the most recent literature are integrated in a pathophysiological framework. By combining mechanistic insights and clinical trial data, this review highlights the trajectory of precision medicine in migraine treatment, offering a perspective on the near future of targeted and individualized therapeutic strategies.</p><p><strong>Expert opinion: </strong>Recent advances in migraine neurobiology offer potential solutions to longstanding challenges. While targeted CGRP therapies have shown promise by addressing specific mechanisms, the pathophysiology of migraine suggests that combination therapies targeting multiple pathways could be beneficial in migraine prevention. The growing diversity of treatment options presents challenges in therapy selection, underscoring the need for predictive biomarkers. These innovations can optimize treatment strategies and improve patient outcomes. As the field progresses, personalized, multimodal approaches are poised to become the standard of care, significantly advancing precision medicine in this area.</p>","PeriodicalId":12084,"journal":{"name":"Expert Opinion on Biological Therapy","volume":" ","pages":"1-15"},"PeriodicalIF":3.6,"publicationDate":"2025-02-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143002674","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}
Maram Ea Abdalla Elsayed, Jasmina Cehajic Kapetanovic, Robert E MacLaren
{"title":"Gene therapy for choroideremia: progress, potential and pitfalls.","authors":"Maram Ea Abdalla Elsayed, Jasmina Cehajic Kapetanovic, Robert E MacLaren","doi":"10.1080/14712598.2025.2459850","DOIUrl":"https://doi.org/10.1080/14712598.2025.2459850","url":null,"abstract":"<p><p>Introduction Choroideremia is a rare disease with a significant disease burden. Gene-supplementation methods for choroideremia gene therapy have been the most successful form of gene therapy thus far. Areas covered The aim of the current review is to provide an overview of current progress of gene therapy trials to date, with a focus on potential and pitfalls of such trials. We propose a novel end point that may be clinically meaningful for obtaining regulatory approval in subsequent clinical trials. Additionally, we offer recommendations for further optimization of surgical techniques. Expert opinion Lessons learnt from this phase 3 clinical trial, encompassing optimal vector design, delivery techniques, patient selection criteria, and longterm safety profiles can be used in the development of treatments for polygenic retinal disorders, which may necessitate a more nuanced approach due to genetic complexity.</p>","PeriodicalId":12084,"journal":{"name":"Expert Opinion on Biological Therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-02-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143079088","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":"An evaluation of mepolizumab as an add-on maintenance treatment for severe eosinophilic asthma.","authors":"Zheng Zhu, Xiaoying Chen, Yiting Luo, Rui Feng, Zicong Zhou, Ruchong Chen","doi":"10.1080/14712598.2025.2457779","DOIUrl":"10.1080/14712598.2025.2457779","url":null,"abstract":"<p><strong>Introduction: </strong>Clinical experience with anti-interleukin (IL)-5 biologic therapies for severe asthma has been increasing, alongside deeper and broader research focusing on the role of IL-5 and the IL-5 targeted mepolizumab. This review aims to provide an update of the evidence on the role of IL-5 and mepolizumab, with discussions of the benefits of mepolizumab and its future potential, to promote the comprehension of the pathophysiology and therapeutic approaches to asthma.</p><p><strong>Areas covered: </strong>For this narrative review, we conducted a database search in PubMed and Embase using the keywords 'IL-5' and 'mepolizumab,' focusing on randomized controlled trials and real-world studies up to September 2024. An overview of the pathogenesis of severe asthma, new insights on the role of IL-5 and mepolizumab, and the evidence on the efficacy and safety of mepolizumab in the treatment of severe eosinophilic asthma is provided, and its benefits in clinical remission and future applications are also discussed.</p><p><strong>Expert opinion: </strong>Mepolizumab holds considerable promise in asthma treatment due to its mechanism of action and multiple potential benefits. In clinical practice, it may be worth considering the exploratory initiation of mepolizumab add-on treatment from the time of medium-dose inhaled glucocorticosteroid use.</p>","PeriodicalId":12084,"journal":{"name":"Expert Opinion on Biological Therapy","volume":" ","pages":"1-12"},"PeriodicalIF":3.6,"publicationDate":"2025-01-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143037727","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}
Giulia Costanzo, Giada Sambugaro, Silvio Sartorio, Andrea Zanichelli, Davide Firinu
{"title":"New drugs for the treatment of hereditary angioedema.","authors":"Giulia Costanzo, Giada Sambugaro, Silvio Sartorio, Andrea Zanichelli, Davide Firinu","doi":"10.1080/14712598.2024.2441845","DOIUrl":"10.1080/14712598.2024.2441845","url":null,"abstract":"<p><strong>Introduction: </strong>Revolutionary drugs have been developed and approved in the last 5 years for the treatment of hereditary angioedema (HAE). Increased knowledge of HAE pathophysiology has led to the development of innovative drugs for self-administered on-demand therapy and for short- and long-term prophylaxis (LTP). This has rendered possible a personalized approach for patients, allowing greater control of symptoms, better quality of life and reduction in the incidence of adverse effects linked to old treatments.</p><p><strong>Areas covered: </strong>In this review we have highlighted which treatments are currently approved for HAE and some of the promising future therapies under development.</p><p><strong>Expert opinion: </strong>While the first generation of approved treatments improved disease control for most patients, innovative therapies may allow individualized action plans and reduce complexity of treatment. Switching therapies due to insufficient efficacy, patient preference or adverse events is becoming progressively feasible and common. New LTPs may lead to the achievement of attack-free remission, allowing us to hopefully reach complete disease control for all patients and further improving their quality of life. In particular, LTPs with longer administration intervals, and on-demand therapies administered via the oral route will have a key role and will set more prominent targets for the upcoming drugs.</p>","PeriodicalId":12084,"journal":{"name":"Expert Opinion on Biological Therapy","volume":" ","pages":"79-91"},"PeriodicalIF":3.6,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142812653","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}
Lucia Perez-Lamas, Jose Sandoval-Sus, Julio C Chavez
{"title":"Should CAR-T cell therapy be considered a standard of care for patients with refractory diffuse large B-cell lymphoma in second line treatment?","authors":"Lucia Perez-Lamas, Jose Sandoval-Sus, Julio C Chavez","doi":"10.1080/14712598.2025.2451888","DOIUrl":"10.1080/14712598.2025.2451888","url":null,"abstract":"<p><strong>Introduction: </strong>CAR-T therapy has transformed the treatment landscape for relapsed/refractory diffuse large B-cell lymphomas (DLBCL).</p><p><strong>Areas covered: </strong>This article reviews the existing evidence for using CAR-T therapy as a second-line treatment. Two major phase 3 trials, ZUMA-7 and TRANSFORM, have shown that axi-cel and liso-cel, respectively, offer superior outcomes compared to historical standard chemoimmunotherapy and consolidation with autologous hematopoietic stem cell transplantation (auto-HCT). Additionally, two promising phase 2 trials, PILOT and ALYCANTE, demonstrated the efficacy of CAR-T therapy in patients who are ineligible for auto-HCT. We also reviewed the potential biological factors behind these results.</p><p><strong>Expert opinion: </strong>Several factors support the use of CAR-T therapy in earlier treatment lines: better T-cell fitness in the infused product, reduced systemic inflammation in patients, and a more favorable tumor microenvironment. Although real-world data for second-line CAR-T therapy is still early, it is expected that CAR-T will be used more widely. Additional focus highlights the need for defining suitable patient populations and the efforts to enhance accessibility and cost-effectiveness of this groundbreaking treatment approach.</p>","PeriodicalId":12084,"journal":{"name":"Expert Opinion on Biological Therapy","volume":" ","pages":"139-148"},"PeriodicalIF":3.6,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142946890","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}
Alessandra Vultaggio, Laura Bergantini, Claudia Crimi, Andrea Matucci, Francesco Menzella, Jan Walter Volk Schroeder, Gianenrico Senna, Paolo Cameli
{"title":"Benralizumab: from tissue distribution to eosinophilic cytotoxicity up to potential immunoregulation.","authors":"Alessandra Vultaggio, Laura Bergantini, Claudia Crimi, Andrea Matucci, Francesco Menzella, Jan Walter Volk Schroeder, Gianenrico Senna, Paolo Cameli","doi":"10.1080/14712598.2024.2446600","DOIUrl":"10.1080/14712598.2024.2446600","url":null,"abstract":"<p><strong>Introduction: </strong>Benralizumab, a monoclonal IgG antibody, has emerged as a key therapeutic agent in severe asthma by specifically targeting eosinophils, pivotal cells that drive inflammation and tissue damage. Over the past two decades, the availability of such targeted therapies has allowed patients to achieve better disease control. Real-world evidence has consistently demonstrated the effectiveness of benralizumab in managing severe asthma.</p><p><strong>Areas covered: </strong>This paper discusses the kinetic and potential mechanism of action of benralizumab beyond the well-known antibody-dependent cell-mediated cytotoxicity involving natural killer cells.</p><p><strong>Expert opinion: </strong>The available data so far clearly show that reducing eosinophils, one of the main drivers of inflammation and tissue damage in SA, accounts for clinical benefits to these patients. Benralizumab is able to directly reduce tissue levels of eosinophils via multiple mechanisms, and additionally, it is potentially able to modulate the innate immune response. The complex and unique multiple modes of action of benralizumab and its pharmacokinetic features, seem to be the milestone on which the effectiveness of benralizumab is founded.</p>","PeriodicalId":12084,"journal":{"name":"Expert Opinion on Biological Therapy","volume":" ","pages":"175-185"},"PeriodicalIF":3.6,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142871825","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":"Lebrikizumab: a new anti-IL-13 agent for treating moderate-to-severe atopic dermatitis.","authors":"Luca Stingeni, Silvia Ferrucci, Paolo Amerio, Caterina Foti, Cataldo Patruno, Giampiero Girolomoni","doi":"10.1080/14712598.2024.2435427","DOIUrl":"10.1080/14712598.2024.2435427","url":null,"abstract":"<p><strong>Introduction: </strong>Atopic dermatitis (AD) is a common chronic inflammatory skin disease. Moderate-to-severe AD severely affects patients' quality of life. New drugs selectively targeting molecular pathways involved in the pathogenesis of the disease led to a new era for the treatment of AD. However, the current available options are limited and do not completely fulfill patients' needs. Recently, lebrikizumab, a new humanized monoclonal antibody targeting IL-13, has been approved for treating moderate-to-severe AD.</p><p><strong>Areas covered: </strong>By analyzing scientific literature reporting lebrikizumab phase 3 pivotal clinical studies and summarizing recent advances in AD pathogenesis, in this article we focused on the mechanism of action of lebrikizumab in comparison to other biologics used for treating AD and discussed clinical data that led to the approval of this biologic agent.</p><p><strong>Expert opinion: </strong>Among biologics approved for moderate-to-severe AD, lebrikizumab is characterized by a unique mechanism of action and an attractive maintenance regimen, besides good efficacy and safety profiles. Moreover, clinical evidence suggests that patients naïve or pre-treated with other biologics and affected by AD localized in sensitive areas and by type 2 comorbidities might be successfully treated with lebrikizumab.</p>","PeriodicalId":12084,"journal":{"name":"Expert Opinion on Biological Therapy","volume":" ","pages":"15-20"},"PeriodicalIF":3.6,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142784837","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":"The cell replacement therapeutic potential of human pluripotent stem cells.","authors":"Adeleh Taei, Fatemeh-Sadat Sajadi, Sarvenaz Salahi, Zahra Enteshari, Nasrin Falah, Zahra Shiri, Saeed Abasalizadeh, Ensiyeh Hajizadeh-Saffar, Seyedeh-Nafiseh Hassani, Hossein Baharvand","doi":"10.1080/14712598.2024.2443079","DOIUrl":"10.1080/14712598.2024.2443079","url":null,"abstract":"<p><strong>Introduction: </strong>The remarkable ability of human pluripotent stem cells (hPSCs) to differentiate into specialized cells of the human body emphasizes their immense potential in treating various diseases. Advances in hPSC technology are paving the way for personalized and allogeneic cell-based therapies. The first-in-human studies showed improved treatment of diseases with no adverse effects, which encouraged the industrial production of this type of medicine. To ensure the quality, safety and efficacy of hPSC-based products throughout their life cycle, it is important to monitor and control their clinical translation through good practices (GxP) regulations. Understanding these rules in advance will help ensure that the industrial development of hPSC-derived products for widespread clinical implementation is feasible and progresses rapidly.</p><p><strong>Areas covered: </strong>In this review, we discuss the key translational obstacles of hPSCs, outline the current hPSC-based clinical trials, and present a workflow for putative clinical hPSC-based products. Finally, we highlight some future therapeutic opportunities for hPSC-derivatives.</p><p><strong>Expert opinion: </strong>hPSC-based products continue to show promise for the treatment of a variety of diseases. While clinical trials support the relative safety and efficacy of hPSC-based products, further investigation is required to explore the clinical challenges and achieve exclusive regulations for hPSC-based cell therapies.</p>","PeriodicalId":12084,"journal":{"name":"Expert Opinion on Biological Therapy","volume":" ","pages":"47-67"},"PeriodicalIF":3.6,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142827772","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}