Expert Opin Biol Ther [JOURNAL]
Sun
200 papers
Optimizing biosimilar development: current approaches to demonstrating pharmacokinetic and analytical similarity and a proposal for a single reference approach.
Kiely P, Murray D
Expert Opin Biol Ther · 2025 Apr · PMID 40035204 · Publisher ↗
OBJECTIVES: Many biosimilars have been approved in both the United States of America (U.S.A.) and European Union (EU). We aim to highlight how regulatory challenges and divergent requirements between both agencies exist....
OBJECTIVES: Many biosimilars have been approved in both the United States of America (U.S.A.) and European Union (EU). We aim to highlight how regulatory challenges and divergent requirements between both agencies exist. METHODS: A comprehensive review of biosimilars approved by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) was conducted. We aimed to highlight similarities and differences in approaches taken by the agencies regarding the use of non-local (i.e. non -US and non-EU) reference medicinal products in biosimilar development. A search of the six most frequent classes of biosimilars authorized (cutoff date: 12 June 2024) by these agencies were identified and their public assessment reports reviewed. RESULTS: The review highlighted that pharmacokinetic (PK) bioequivalence studies are often replicated, the current process is inefficient and not entirely necessary when critical quality attributes are considered. CONCLUSION: This article provides a comparative analysis of biosimilar approvals in EU and US regulatory agencies, focusing on non-local reference medicinal product (RMP) utilization and bioequivalence demonstration. The findings contribute to literature on biosimilar development and regulatory considerations, enhancing understanding of harmonization opportunities in biosimilar approval processes, potentially improving global access to high-quality, cost-effective biosimilars.
Psoriasis and cancer: the role of inflammation, immunosuppression, and cancer treatment.
Bruni M, Lobefaro F, Pellegrini C … +7 more , Mastrangelo M, Gualdi G, Esposito M, Antonetti P, De Sanctis P, Amerio P, Fargnoli MC
Expert Opin Biol Ther · 2025 Apr · PMID 40034077 · Publisher ↗
INTRODUCTION: The relationship between psoriasis, immunomodulatory therapies, and the risk of malignancies is complex and still debated. The scarcity of evidence in this field makes clinicians hesitate to prescribe biolo...
INTRODUCTION: The relationship between psoriasis, immunomodulatory therapies, and the risk of malignancies is complex and still debated. The scarcity of evidence in this field makes clinicians hesitate to prescribe biological therapies for 'difficult-to-treat' patients. AREAS COVERED: Based on a comprehensive MEDLINE/PUBMED search of articles published up to November 2024, this review synthesizes the current evidence on the association between psoriasis and cancer. This review specifically addresses four key aspects: the overall cancer risk in psoriatic patients, the potential role of cytokines involved in psoriasis pathogenesis in tumor development, the association between biological therapies and the incidence of new malignancies in this population, and the risk of cancer recurrence or progression in patients with a history of malignancy who are treated with biologics. EXPERT OPINION: Biological therapies do not significantly elevate malignancy risk compared to non-biological treatments or the general population. Evidence is also reassuring for patients with prior malignancy, showing no tumor progression or recurrence. These findings support the timely use of biological treatments in 'difficult-to-treat' patients. Regular cancer screenings and risk-factor minimization should always be recommended for psoriatic patients undergoing immunomodulatory therapies. Multidisciplinary management involving oncologists is suggested, particularly for patients with active and advanced oncological disease.
Corticosteroids, hyaluronic acid, platelet-rich plasma, and cell-based therapies for knee osteoarthritis - literature trends are shifting in the injectable treatments' evidence: a systematic review and expert opinion.
Bensa A, Bianco Prevot L, Moraca G … +3 more , Sangiorgio A, Boffa A, Filardo G
Expert Opin Biol Ther · 2025 Mar · PMID 40028854 · Publisher ↗
INTRODUCTION: The aim of this systematic review was to quantify the data available on corticosteroids (CS), hyaluronic acid, (HA), platelet-rich plasma (PRP), and cell-based therapies for knee osteoarthritis (OA) treatme...
INTRODUCTION: The aim of this systematic review was to quantify the data available on corticosteroids (CS), hyaluronic acid, (HA), platelet-rich plasma (PRP), and cell-based therapies for knee osteoarthritis (OA) treatment. METHODS: A literature search was conducted on PubMed, Cochrane, and Web of Science according to the PRISMA guidelines. Inclusion criteria: clinical studies of any level of evidence, written in English, evaluating the intra-articular use of CS, HA, PRP, or cell-based therapies for knee OA treatment. RESULTS: The initial search identified 17,415 records. A total of 766 studies from 1959 were included. Of these, 401 were randomized controlled trials (RCTs), 110 comparative studies, and 255 case series, for a total of 75,834 patients. (11,245 treated with CS, 40,862 with HA, 16,174 with PRP, and 7,553 with cell-based therapies). The number of placebo-controlled RCTs remains limited and a negligible percentage of studies investigated possible disease-modifying effects of these treatments for knee OA. CONCLUSIONS: The evidence on injective knee OA treatments is increasing at different speeds with a more rapidly growing literature focusing on orthobiologics. Currently, HA has the largest evidence, followed by PRP that recently surpassed the number of studies evaluating CS. Cell-based therapies are also growing rapidly, although the number of studies is still lower. The rapid literature shift toward orthobiologics urges an update in societies' guidelines to align with the new body of evidence on knee OA treatments. PROTOCOL REGISTRATION: www.crd.york.ac.uk/prosperoi dentifier is CRD42024592972.
Drawing on collective action theory to foster sustainable biosimilar markets - insights from co-creation workshops with UK and Belgian stakeholders.
Barcina Lacosta T, Vulto AG, Turk F … +2 more , Huys I, Simoens S
Expert Opin Biol Ther · 2025 Apr · PMID 40022551 · Publisher ↗
BACKGROUND: Research has signaled the need for reformed biosimilar policy frameworks that adopt a behavioral approach, are informed by consensus-generating strategies and thus better align with the requirements of local...
BACKGROUND: Research has signaled the need for reformed biosimilar policy frameworks that adopt a behavioral approach, are informed by consensus-generating strategies and thus better align with the requirements of local healthcare communities. RESEARCH DESIGN AND METHODS: Through a series of co-creation workshops, the current study explores the feasibility of applying learnings from Collective Action Theory to formulate evidence-based multistakeholder-supported policy recommendations. RESULTS: Insights from the conducted workshops indicate that future policy frameworks would benefit from: 1) a working system of incentives and rewards aligned with stakeholder needs; 2) evaluating the cost-benefit balance for stakeholders prior to policy implementation; 3) involving multistakeholder panels in policy co-design; 4) adopting a long-term vision; 5) fostering coordination at the interface between levels of governance; 6) defining shared goals and efficient systems to monitor policy compliance; and 7) using policy outcome data to adapt current policy frameworks based on evolving needs. The incorporation of these elements to policies is expected to help prioritize long-term sustainable solutions, and balance short-term gains and long-term objectives in biosimilar markets. CONCLUSIONS: This study constitutes a first approach to developing multistakeholder-supported principles for sustainable biosimilar markets. This is a necessary step toward generating stakeholders' consensus on biosimilar policies.
Steps towards the clinical application of endometrial and menstrual fluid mesenchymal stem cells for the treatment of gynecological disorders.
Sadiasa A, Werkmeister JA, Gurung S … +1 more , Gargett CE
Expert Opin Biol Ther · 2025 Mar · PMID 39925343 · Publisher ↗
INTRODUCTION: The human endometrium is a highly regenerative tissue that contains mesenchymal stem/stromal cells (MSCs). These MSCs are sourced via office-based biopsies and menstrual fluid, providing a less invasive and...
INTRODUCTION: The human endometrium is a highly regenerative tissue that contains mesenchymal stem/stromal cells (MSCs). These MSCs are sourced via office-based biopsies and menstrual fluid, providing a less invasive and readily available option for cell-based therapies. This review provides an update on endometrial-derived MSCs as a treatment option for gynecological diseases. AREAS COVERED: This narrative review covers the characterization and therapeutic mechanisms of endometrium biopsy-derived MSCs (eMSCs) and menstrual fluid-derived mesenchymal stromal cells (MenSCs), highlighting similarities and differences. It also covers studies of their application in preclinical animal models and in clinical trials as potential cell-based therapies for gynecological diseases. EXPERT OPINION: eMSCs and MenSCs from a homologous tissue source have the potential to promote regenerative activity as a treatment for gynecological diseases. Both eMSCs and MenSCs demonstrate therapeutic benefits through their paracrine activity in tissue regeneration, immunomodulation, angiogenesis, and mitigating fibrosis. Further research is essential to establish standardized isolation and characterization protocols, particularly for heterogeneous MenSCs, and to fully understand their mechanisms of action. Implementing SUSD2 magnetic bead sorting for purifying eMSCs from endometrial tissues and menstrual fluid is crucial for their use in future cell-based therapies. Optimization of production, storage, and delivery methods will maximize their therapeutic effectiveness.
Literature review and expert opinion on diagnosis and current management of generalized pustular psoriasis.
Marzano AV, Fargnoli MC, Gisondi P … +9 more , Balato A, Bianchi L, Calzavara-Pinton P, Chiricozzi A, Costanzo A, Megna M, Micali G, Piaserico S, Prignano F
Expert Opin Biol Ther · 2025 Mar · PMID 39925164 · Publisher ↗
INTRODUCTION: Generalized pustular psoriasis (GPP) is a rare, chronic, systemic, autoinflammatory disease characterized by the eruption of sterile pustules, often accompanied by more general symptoms, such as fever, fati...
INTRODUCTION: Generalized pustular psoriasis (GPP) is a rare, chronic, systemic, autoinflammatory disease characterized by the eruption of sterile pustules, often accompanied by more general symptoms, such as fever, fatigue, and a burning sensation in the skin. GPP can be potentially life-threatening, if untreated, as it can lead to complications, such as sepsis and heart failure. AREAS COVERED: In this literature review and expert opinion article, we provide an overview of the epidemiology, pathogenesis, clinical presentation, diagnosis, and treatment of GPP. Eleven dermatologists representing seven different Italian regions considered relevant evidence in the literature to discuss the current diagnosis and treatment of GPP. The expert panel of dermatologists identified several weaknesses in the current clinical management of GPP. EXPERT OPINION: There is an inconsistent definition and classification of the disease across the literature, which can lead to misdiagnosis and delay in disease treatment. Furthermore, there are no international and standardized clinical guidelines on disease management, especially in Europe. There is a profound need for the development of novel therapeutic agents with sustained efficacy to decrease the impact of the comorbidities and mortality associated with GPP, prevent the onset of complications, and support the unmet needs of these patients.
An evaluation of linvoseltamab for treatment of relapsed/refractory multiple myeloma.
Avigan ZM, Rattu MA, Richter J
Expert Opin Biol Ther · 2025 Mar · PMID 39923122 · Publisher ↗
INTRODUCTION: Bispecific antibody therapies, primarily targeting B-cell maturation antigen (BCMA), have shown remarkable outcomes in the treatment of heavily pretreated patients with multiple myeloma (MM). However, there...
INTRODUCTION: Bispecific antibody therapies, primarily targeting B-cell maturation antigen (BCMA), have shown remarkable outcomes in the treatment of heavily pretreated patients with multiple myeloma (MM). However, there are no current head-to-head trials informing practice for selection or sequencing of optimal T cell redirection strategies in later lines of therapy. Linvoseltamab is a novel BCMA-targeted bispecific antibody that was recently evaluated in the phase 1/2 LINKER-MM1 trial and is currently pending formal regulatory approval. AREAS COVERED: In this drug evaluation, we review efficacy and toxicity profiles of linvoseltamab in the context of currently approved bispecific antibody therapies for MM. Specifically, we highlight differences in dosing strategy, cytokine release syndrome (CRS) kinetics, and indirect efficacy comparisons between trials of linvoseltamab and other bispecific antibodies. EXPERT OPINION: Linvoseltamab has a similar efficacy profile with favorable CRS incidence and time-to-onset compared to other approved bispecific antibodies. Pending final regulatory approval and labeling, further real-world analyses are needed to evaluate its final role in the evolving landscape of T cell redirection therapy for MM.
Successes and failures: the latest advances in the clinical development of amyloid-β-targeting monoclonal antibodies for treating Alzheimer's disease.
Panza F, Dibello V, Sardone R … +10 more , Zupo R, Castellana F, Leccisotti I, Moretti MC, Altamura M, Bellomo A, Daniele A, Solfrizzi V, Resta E, Lozupone M
Expert Opin Biol Ther · 2025 Mar · PMID 39908579 · Publisher ↗
INTRODUCTION: The amyloid cascade hypothesis postulated that the accumulation of amyloid-β (Aβ) was the first step of the Alzheimer's disease (AD) pathological process. Effective reduction of Aβ plaque load by numerous d...
INTRODUCTION: The amyloid cascade hypothesis postulated that the accumulation of amyloid-β (Aβ) was the first step of the Alzheimer's disease (AD) pathological process. Effective reduction of Aβ plaque load by numerous drug candidates, among which anti-Aβ monoclonal antibodies, has produced discussible clinical successes and several failures. It was questioned whether Aβ may be the principal AD pathogenic factor and a valid therapeutic target and if targeting Aβ different species could make the difference. AREAS COVERED: This review article summarized successes and failures of anti-Aβ monoclonal antibody therapy for AD, delineating the latest advances for their clinical development also according to their target engagement and downstream biomarkers. EXPERT OPINION: The preliminary success of the recent Phase III randomized clinical trials (RCTs) of lecanemab, donanemab, and remternetug, and lessons learned from the failure of previous anti-Aβ monoclonal antibodies RCTs, provided critical evidence to support the role of Aβ in AD pathogenesis. The loss of free Aβ instead of an Aβ toxicity may promote AD neuropathology. Cerebrospinal fluid analyses (i.e. increases in Aβ) may indicate a potential benefit of anti-Aβ monoclonal antibodies in AD and downstream biomarkers should be considered for providing comprehension in cognitive and clinical efficacy of future AD RCTs.
Navigating the landscape of immune checkpoint inhibitors and novel immunotherapies in melanoma: long-term outcomes, progress, and challenges.
Cheruvu S, McMahon D, Larkin J
Expert Opin Biol Ther · 2025 Mar · PMID 39895540 · Publisher ↗
INTRODUCTION: 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...
INTRODUCTION: 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. AREAS COVERED: 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. EXPERT OPINION: 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.
Gene therapy for choroideremia: progress, potential and pitfalls.
Abdalla Elsayed MEA, Cehajic-Kepetanovic J, MacLaren RE
Expert Opin Biol Ther · 2025 Mar · PMID 39893699 · Full text
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...
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 long-term 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.
How should we approach salvage therapy in advanced renal cell carcinoma after first-line immunotherapy combinations?
Sackstein P, Atkins MB
Expert Opin Biol Ther · 2025 Mar · PMID 39868704 · Publisher ↗
Abstract loading — click title to view on PubMed.
An evaluation of mepolizumab as an add-on maintenance treatment for severe eosinophilic asthma.
Zhu Z, Chen X, Luo Y … +3 more , Feng R, Zhou Z, Chen R
Expert Opin Biol Ther · 2025 Mar · PMID 39861980 · Publisher ↗
INTRODUCTION: 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 mepolizuma...
INTRODUCTION: 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. AREAS COVERED: 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. EXPERT OPINION: 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.
Treating episodic migraine with precision: the evolving landscape of targeted therapies driven by insights in disease biology.
Fleischmann R, Strauß S, Reuter U
Expert Opin Biol Ther · 2025 Mar · PMID 39831521 · Publisher ↗
INTRODUCTION: 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, inclu...
INTRODUCTION: 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. AREAS COVERED: 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. EXPERT OPINION: 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.
Biosimilars in pediatric rheumatology: innovations, challenges, and opportunities.
Öksel A, Sönmez HE, Şahin N
Expert Opin Biol Ther · 2025 · PMID 39798157 · Publisher ↗
INTRODUCTION: Biosimilars are biologic medications designed to closely replicate the properties of previously approved biologic disease-modifying anti-rheumatic drugs (bDMARDs). They offer a cost-effective alternative on...
INTRODUCTION: Biosimilars are biologic medications designed to closely replicate the properties of previously approved biologic disease-modifying anti-rheumatic drugs (bDMARDs). They offer a cost-effective alternative once the original product's patent has expired. AREAS COVERED: In pediatric rheumatology, the use of biosimilars began in 2013 with the launch of the infliximab biosimilar. Since then, more biosimilars, including etanercept, rituximab, and adalimumab, have been introduced, providing additional treatment options for children with rheumatic diseases. This article explores the role of biosimilars in pediatric rheumatology, particularly in juvenile idiopathic arthritis, focusing on their development, safety, and efficacy, as well as the challenges associated with their clinical adoption. It also addresses the importance of education in improving understanding of biosimilars. EXPERT OPINION: The article provides insights into their safety, effectiveness, and economic impact by reviewing current literature to help healthcare professionals make informed decisions for treating pediatric rheumatic diseases. Education for both patients and healthcare providers, effective communication, and expectation management play a critical role in ensuring appropriate treatment continuity.
The potential of antibody-drug conjugates for effective therapy in diffuse large B-cell lymphoma.
Ahmed G, Hamadani M, Al-Juhaishi T
Expert Opin Biol Ther · 2025 · PMID 39798075 · Publisher ↗
INTRODUCTION: Antibody-drug conjugates (ADCs) are a rapidly evolving class of anti-cancer drugs with a significant impact on management of hematological malignancies including diffuse large B-cell lymphoma (DLBCL). ADCs...
INTRODUCTION: Antibody-drug conjugates (ADCs) are a rapidly evolving class of anti-cancer drugs with a significant impact on management of hematological malignancies including diffuse large B-cell lymphoma (DLBCL). ADCs combine a cytotoxic drug (a.k.a. payload) attached through a linker to a monoclonal antibody specific to a particular cancer antigen. Payloads include microtubule disruptors or DNA damaging chemicals. After attaching to the antigen, the ADCs are internalized, and the payload is dissociated from ADC by lysozymes and delivered to the intended site for exerting cytotoxic effects. This unique molecular design permits a better balance of efficacy and safety. Loncastuximab tesirine and polatuzumab vedotin are two ADCs approved in the U.S.A. for treatment of DLBCL. AREAS COVERED: This review covers the efficacy and safety data of these two drugs. We will review new ADC-based combination regimens and novel constructs in development. EXPERT OPINION: ADCs have made a significant impact in improving outcomes of DLBCL patients. Both polatuzumab vedotin and loncastuximab tesirine are established as useful therapeutics options, with polatuzumab vedotin currently approved in first line and relapsed/refractory setting, while loncastuximab tesirine is approved in relapsed setting. ADCs are effective with tolerable safety profile and currently many more ADCs are undergoing clinical trials.
Should CAR-T cell therapy be considered a standard of care for patients with refractory diffuse large B-cell lymphoma in second line treatment?
Perez-Lamas L, Sandoval-Sus J, Chavez JC
Expert Opin Biol Ther · 2025 · PMID 39784146 · Publisher ↗
INTRODUCTION: CAR-T therapy has transformed the treatment landscape for relapsed/refractory diffuse large B-cell lymphomas (DLBCL). AREAS COVERED: This article reviews the existing evidence for using CAR-T therapy as a s...
INTRODUCTION: CAR-T therapy has transformed the treatment landscape for relapsed/refractory diffuse large B-cell lymphomas (DLBCL). AREAS COVERED: 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. EXPERT OPINION: 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.
Integrating gene therapy into the treatment paradigm for non-muscle invasive bladder cancer.
Steinmetz AR, Jazayeri B, Pierce M … +4 more , Mokkapati S, McConkey D, Li R, Dinney CP
Expert Opin Biol Ther · 2025 · PMID 39779686 · Publisher ↗
INTRODUCTION: Approximately 75% of bladder cancer cases are non-muscle invasive at diagnosis. Drug development for non-muscle invasive bladder cancer (NMIBC) has historically lagged behind that of other malignancies. No...
INTRODUCTION: Approximately 75% of bladder cancer cases are non-muscle invasive at diagnosis. Drug development for non-muscle invasive bladder cancer (NMIBC) has historically lagged behind that of other malignancies. No treatment has demonstrated the ability to overcome drug resistance that ultimately leads to recurrence and progression. Gene therapy is emerging as a promising option for patients with NMIBC. AREAS COVERED: This review summarizes the clinical application of gene therapy in NMIBC management and discusses recent clinical trials involving the adenoviral vector-based treatment nadofaragene firadenovec, and the oncolytic serotype 5 adenovirus, cretostimogene grenadenorepvec. Nadofaragene received approval by the Food and Drug Administration in December 2022, and cretostimogene has been granted Fast Track Designation and Breakthrough Therapy Designation. Ongoing trials are investigating strategies to augment efficacy and durability of these therapies. EXPERT OPINION: Gene therapy may overcome resistance mechanisms of other NMIBC treatments, and data suggest a role for combination therapy with additive or synergistic agents. Significant differences in trial design limit comparability of agents across trials, highlighting the need for critical assessment of published findings. While initial investigations were in high-risk patients who recur despite frontline therapy with Bacillus Calmette-Guerin (BCG), there is growing interest in BCG-naïve and intermediate-risk populations.
Clinical updates in mesenchymal stromal cell therapy for osteoarthritis treatment.
Im GI
Expert Opin Biol Ther · 2025 · PMID 39710894 · Publisher ↗
INTRODUCTION: Osteoarthritis (OA) is a common chronic musculoskeletal disease with heterogeneous clinical manifestations and variable responses to different treatments. Unfortunately, there is no effective disease modify...
INTRODUCTION: Osteoarthritis (OA) is a common chronic musculoskeletal disease with heterogeneous clinical manifestations and variable responses to different treatments. Unfortunately, there is no effective disease modifying therapy at present that can alter the natural course of the disease. Cell therapy based on mesenchymal stromal cells (MSCs) may offer an attractive therapeutic option for OA with their multiple modes of action, particularly immune-regulatory and regenerative capacities. AREAS COVERED: In this narrative review, updates on mode of action based on patient's data, factors that can influence the efficacy of MSC treatment, current status in clinical application of MSCs as seen from randomized, controlled OA trials are introduced as well as the author's perspectives in the future of MSCs as OA therapeutics. EXPERT OPINION: Symptomatic relief is not sufficient to justify the high cost associated with culture-expanded stem cells. Its advantages and efficacy over simple and low risk/cost modalities should be seriously reevaluated. Also, as the short-term strategy, efforts should be made to lower the cost of MSC therapy. In the future, multiomics technology may help to predict that subgroup of patients who will favorably respond to stem cell treatment, which would enhance the cost effectiveness and therapeutic benefit of MSC therapy.
Benralizumab: from tissue distribution to eosinophilic cytotoxicity up to potential immunoregulation.
Vultaggio A, Bergantini L, Crimi C … +5 more , Matucci A, Menzella F, Schroeder JWV, Senna G, Cameli P
Expert Opin Biol Ther · 2025 · PMID 39708290 · Publisher ↗
INTRODUCTION: 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...
INTRODUCTION: 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. AREAS COVERED: 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. EXPERT OPINION: 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.
An evaluation of sugemalimab for the treatment of relapsed or refractory extranodal natural killer T-cell lymphoma.
Feng Y, Liu X, Yu J … +7 more , Song Z, Li L, Qiu L, Zhou S, Qian Z, Wang X, Zhang H
Expert Opin Biol Ther · 2025 Jan · PMID 39702924 · Publisher ↗
INTRODUCTION: Relapsed or refractory extranodal natural killer/T-cell lymphoma (R/R ENKTL) is a rare and aggressive subtype arising from natural killer or cytotoxic T-cells, predominantly affecting the nasal cavity and p...
INTRODUCTION: Relapsed or refractory extranodal natural killer/T-cell lymphoma (R/R ENKTL) is a rare and aggressive subtype arising from natural killer or cytotoxic T-cells, predominantly affecting the nasal cavity and paranasal sinuses, lacking a standardized therapeutic approach. Sugemalimab, a fully human, full-length anti-PD-L1 immunoglobulin G4 (IgG4) monoclonal antibody (mAb), has been investigated in a Single-Arm, Multicenter, Phase II Study (GEMSTONE-201). The results demonstrated significant efficacy, favorable tolerability, and manageable adverse reactions of sugemalimab in R/R ENKTL. This study summarizes and compares the efficacy and safety profile of sugemalimab with several other PD-1/PD-L1 inhibitors in R/R ENKTL patients. AREA COVERED: We included a Phase II study (GEMSTONE-201) of sugemalimab in R/R ENKTL. EXPERT OPINION: The clinical trials have demonstrated superior efficacy of sugemalimab, evidenced by a complete response rate (CRR) of 35.9% and an overall response rate (ORR) of 44.9%. In comparison with other immune checkpoint inhibitors (ICIs), sugemalimab shows a notably higher CRR. Additionally, sugemalimab exhibits a manageable safety profile. Further evaluation of sugemalimab is required based on its efficacy and safety in real-world patient populations. Should sugemalimab be included in medical insurance in the future, it could potentially benefit a larger number of patients with R/R ENKTL.
About
- Frequency
- Sun
- Papers found
- 200
- RSS feed
- Subscribe