Expert Opinion on Biological Therapy最新文献

The future of cellular therapy for the treatment of renal cell carcinoma. 细胞疗法治疗肾细胞癌的未来。

IF 3.6 3区医学

Expert Opinion on Biological Therapy Pub Date : 2024-11-04 DOI: 10.1080/14712598.2024.2418321

Nada Chaoul, Eleonora Lauricella, Andrea Giglio, Gabriella D'Angelo, Carlo Ganini, Mauro Cives, Camillo Porta

{"title":"The future of cellular therapy for the treatment of renal cell carcinoma.","authors":"Nada Chaoul, Eleonora Lauricella, Andrea Giglio, Gabriella D'Angelo, Carlo Ganini, Mauro Cives, Camillo Porta","doi":"10.1080/14712598.2024.2418321","DOIUrl":"10.1080/14712598.2024.2418321","url":null,"abstract":"Introduction: Systemic treatment options for renal cell carcinoma (RCC) have expanded considerably in recent years, and both tyrosine kinase inhibitors and immune checkpoint inhibitors, alone or in combination, have entered the clinical arena. Adoptive cell immunotherapies have recently revolutionized the treatment of cancer and hold the promise to further advance the treatment of RCC.Areas covered: In this review, we summarize the latest preclinical and clinical development in the field of adoptive cell immunotherapy for the treatment of RCC, focusing on lymphokine-activated killer (LAK) cells, cytokine-induced killer (CIK) cells, tumor-infiltrating T cells (TILs), TCR-engineered T cells, chimeric antigen receptor (CAR) T cells, and dendritic cell vaccination strategies. Perspectives on emerging cellular products including CAR NK cells, CAR macrophages, as well as γδ T cells are also included.Expert opinion: So far, areas of greater therapeutic success of adoptive cell therapies include the adjuvant administration of CIK cells and the transfer of anti-CD70 CAR T cells in patients with metastatic RCC. Bench to bedside and back research will be needed to overcome current limitations of adoptive cell therapies in RCC, primarily aiming at improving the safety of immune cell products, optimizing their antitumor activity and generating off-the-shelf products ready for clinical use.","PeriodicalId":12084,"journal":{"name":"Expert Opinion on Biological Therapy","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2024-11-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142557430","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

Will zilebesiran, an RNA interference therapy, be effective, safe, and improve the treatment of hypertension? RNA干扰疗法齐来贝西兰是否有效、安全，并能改善高血压的治疗？

IF 3.6 3区医学

Expert Opinion on Biological Therapy Pub Date : 2024-11-04 DOI: 10.1080/14712598.2024.2425343

Sheila A Doggrell

{"title":"Will zilebesiran, an RNA interference therapy, be effective, safe, and improve the treatment of hypertension?","authors":"Sheila A Doggrell","doi":"10.1080/14712598.2024.2425343","DOIUrl":"https://doi.org/10.1080/14712598.2024.2425343","url":null,"abstract":"Introduction: Less than half of the subjects with hypertension have been diagnosed and treated, with only 21% having their blood pressure under control. Many of the subjects find it difficult to adhere to daily antihypertensives. Zilebesiran reduces hepatic angiotensinogen messenger RNA levels to inhibit the renin-angiotensin-aldosterone system and is being developed as a long-acting anti-hypertensive agent.Areas covered: KARDIA-1; a phase 2 clinical trial of zilebesiran in mild to moderate hypertension. Most doses of zilebesiran (150-600 mg) modestly reduced blood pressure from baseline to month 3. Adverse events included hyperkalemia and kidney failure.Expert opinion: The main problem with zilebesiran is that it only has a modest effect on blood pressure, and it is likely to have to be used as add-on therapy, which will probably reduce any benefits on adherence it has. It was also difficult to reliably interpret the results of KARDIA-1 as blood pressure went up significantly in the placebo group. KARDIA-1 did not answer previous concerns about zilebesiran; (i) what happens during volume depletion, sepsis, and pregnancy when angiotensinogen is inhibited long-term or (ii) will it be effective in a high sodium diet.","PeriodicalId":12084,"journal":{"name":"Expert Opinion on Biological Therapy","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2024-11-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142568246","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

Recent developments and industry interest in gene therapy for Duchenne muscular dystrophy. 杜兴氏肌肉萎缩症基因疗法的最新进展和业界兴趣。

IF 3.6 3区医学

Expert Opinion on Biological Therapy Pub Date : 2024-11-03 DOI: 10.1080/14712598.2024.2422998

Hidenori Moriyama, Toshifumi Yokota

引用次数: 0

Stem cell therapy for type-2 diabetes: keeping the pedal to the metal to deliver translation to the clinic. 干细胞疗法治疗 2 型糖尿病：坚持不懈地向临床转化。

IF 3.6 3区医学

Expert Opinion on Biological Therapy Pub Date : 2024-10-31 DOI: 10.1080/14712598.2024.2422358

Ning Yang, LaTonya J Hickson, Lilach O Lerman

引用次数: 0

Optimization of radiation target volume for locally advanced esophageal cancer in the immunotherapy era. 在免疫疗法时代优化局部晚期食管癌的放射靶体积。

IF 3.6 3区医学

Expert Opinion on Biological Therapy Pub Date : 2024-10-26 DOI: 10.1080/14712598.2024.2423009

Jian Zheng, Zhunhao Zheng, Tian Zhang, Xi Chen, Qingsong Pang, Ping Wang, Cihui Yan, Wencheng Zhang

{"title":"Optimization of radiation target volume for locally advanced esophageal cancer in the immunotherapy era.","authors":"Jian Zheng, Zhunhao Zheng, Tian Zhang, Xi Chen, Qingsong Pang, Ping Wang, Cihui Yan, Wencheng Zhang","doi":"10.1080/14712598.2024.2423009","DOIUrl":"https://doi.org/10.1080/14712598.2024.2423009","url":null,"abstract":"Introduction: Locally advanced esophageal cancer (EC) has poor prognosis. Preliminary clinical studies have demonstrated the synergistic efficacy of radiotherapy combined with immunotherapy in EC. Adjusting the radiotherapy target volume to protect immune function favors immunotherapy. However, there is no clear consensus on the exact definition of the EC target volume.Areas covered: Preclinical studies have provided a wealth of information on immunotherapy combined with different radiotherapy modalities, and several clinical studies have evaluated the impact of immunotherapy combined with radiotherapy on locally advanced EC. Here, we illustrate the rational target volume delineation for radiotherapy in terms of patient prognosis, pattern of radiotherapy failure, treatment-related toxicities, tumor-draining lymph nodes, and systemic immunity and summarize the clinical trials of radiotherapy combined with immunotherapy in EC.Expert opinion: We recommend applying involved-field irradiation (IFI) instead of elective nodal irradiation (ENI) for irradiated fields when immunotherapy is combined with chemoradiotherapy (CRT) for locally advanced EC. We expect that this target design will be evaluated in clinical trials to further explore more precise diagnostic modalities, long-term toxic responses, and quality of survival, and stratification factors for personalized treatment, and to provide more treatment benefits for patients.","PeriodicalId":12084,"journal":{"name":"Expert Opinion on Biological Therapy","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2024-10-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142497637","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 evaluation of ravulizumab for the treatment of neuromyelitis optica spectrum disorder. 评估雷珠单抗治疗神经脊髓炎视网膜频谱紊乱症的效果。

IF 3.6 3区医学

Expert Opinion on Biological Therapy Pub Date : 2024-10-26 DOI: 10.1080/14712598.2024.2423002

Denis T Balaban, Michael Levy, Ray Borrow, Monique R Anderson

{"title":"An evaluation of ravulizumab for the treatment of neuromyelitis optica spectrum disorder.","authors":"Denis T Balaban, Michael Levy, Ray Borrow, Monique R Anderson","doi":"10.1080/14712598.2024.2423002","DOIUrl":"https://doi.org/10.1080/14712598.2024.2423002","url":null,"abstract":"Introduction: Following the CHAMPION-NMOSD trial, the FDA recently granted approval for ravulizumab, a humanized monoclonal antibody against complement C5 protein in AQP-4 seropositive neuromyelitis optica spectrum disorder (NMOSD). Similar to eculizumab, ravulizumab offers near complete prevention of NMOSD relapses, but has a longer half-life, providing decreased infusion frequency and increased convenience for patients. While targeting the complement pathway has clear advantages, patients are at risk for infection with encapsulated organisms, in particular Neisseria meningitidis.Areas covered: In this paper, we discuss the details of the CHAMPION-NMOSD trial and discuss challenges in meningitis prevention and strategies for switching therapies.Expert opinion: Ravulizumab improves on eculizumab's success as a highly effective NMOSD therapy by decreasing infusion frequency, thereby increasing patient convenience. We predict ravulizumab will eventually replace eculizumab, but may not have a similar impact on inebelizumab or satralizumab. Patients taking C5 complement inhibitors have an increased risk of serious meningococcal infections, such as invasive meningococcal disease (IMD), and have incomplete protection against IMD despite immunization. Thus, we recommend that in addition to standard pre-immunizations against Neisseria meningitidis, patients should also be assessed for starting on appropriate antibiotic prophylaxis against IMD, based on local resistance patterns.","PeriodicalId":12084,"journal":{"name":"Expert Opinion on Biological Therapy","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2024-10-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142497635","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

Evaluation of orphan maintained biological medicinal products in the European Union between 2018 to 2023: a regulatory perspective. 2018 年至 2023 年欧盟孤儿维护生物药品评估：监管视角。

IF 3.6 3区医学

Expert Opinion on Biological Therapy Pub Date : 2024-10-25 DOI: 10.1080/14712598.2024.2422360

Paolo Caferra, Thomas Fraisse, Maria Letizia Trincavelli, Laura Marchetti, Anna Maria Piras

{"title":"Evaluation of orphan maintained biological medicinal products in the European Union between 2018 to 2023: a regulatory perspective.","authors":"Paolo Caferra, Thomas Fraisse, Maria Letizia Trincavelli, Laura Marchetti, Anna Maria Piras","doi":"10.1080/14712598.2024.2422360","DOIUrl":"https://doi.org/10.1080/14712598.2024.2422360","url":null,"abstract":"Objectives: Orphan medicinal products (OMPs) authorized in the European Union (EU) benefit from market exclusivity, fee waivers, and national incentives. Maintaining orphan status during the marketing authorization application (MAA) requires meeting eligibility criteria, especially demonstrating significant benefit (SB), which is challenging. This study identifies key features linked to successful orphan status maintenance for biological OMPs approved in the EU between 2018 and 2023.Methods: Data from European public assessment reports and orphan maintenance assessment reports were analyzed.Results: Among the 50 biological OMPs granted orphan designation, 68.0% had to demonstrate SB over existing treatments, with 91.2% leveraging the clinically relevant advantage area, utilizing better clinical efficacy (83.8%) and subpopulation (38.7%) sub-domains. However, 32% did not need to demonstrate SB due to a lack of alternative treatments, most of which were ultra-orphan drugs. Advanced therapy medicinal products and monoclonal antibodies were the most numerous OMP categories, whereas oncology and immunomodulation were the preferred therapeutic areas.Conclusion: The Orphan Regulation plays a critical role in advancing treatments for rare diseases, fostering innovation while addressing unmet medical needs. Nonetheless, the insufficient return on investment criterion remains underused, whereas refining major contribution to patient care guidelines and incorporating real-world evidence may enhance regulatory evaluations.","PeriodicalId":12084,"journal":{"name":"Expert Opinion on Biological Therapy","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2024-10-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142497636","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

Assessment of cell-binding capacity of shed rAAV particles after gene therapy vector administration: implications for environmental risk and hygiene recommendations. 评估基因治疗载体使用后脱落的 rAAV 粒子与细胞的结合能力：对环境风险和卫生建议的影响。

IF 3.6 3区医学

Expert Opinion on Biological Therapy Pub Date : 2024-10-22 DOI: 10.1080/14712598.2024.2418961

Tobias Fleischmann

{"title":"Assessment of cell-binding capacity of shed rAAV particles after gene therapy vector administration: implications for environmental risk and hygiene recommendations.","authors":"Tobias Fleischmann","doi":"10.1080/14712598.2024.2418961","DOIUrl":"10.1080/14712598.2024.2418961","url":null,"abstract":"Background: Currently, adeno-associated viruses (AAVs) are the most commonly used in vivo gene therapy (GT) vector platform. Risks posed to the environment, including the public, have not been well studied in the past. There is uncertainty concerning the necessary level of biocontainment and appropriate hygiene behavior for the handling of secreta/excreta of GT patients during the shedding phase.Research design and methods: Here, feces and urine samples from non-human primates, treated with an AAV9-based vector at 2 × 1013 vector genomes per kilogram body weight (vg/kg), were analyzed for vector presence and subsequently analyzed for their capacity to bind to cells.Results: Both sample types contained particles which bound to cells at concentrations in the range of ~104 (and higher) vg/mL of culture medium. Novel control rAAV vector displayed a ~2-3 orders of magnitude higher affinity to cells than shed particles.Conclusions: The lower binding capacity of the shed vector particles speaks in favor of a more relaxed containment and hygiene approach in the context of GT. It is recommended that current hygiene and contact-avoidance-based containment measures after GT administration are reduced. The results also support the efforts to achieve a simplification of the regulatory review process of medicinal genetically modified organisms.","PeriodicalId":12084,"journal":{"name":"Expert Opinion on Biological Therapy","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2024-10-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142461283","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 systematic review of the efficacy and safety of anti-amyloid beta monoclonal antibodies in treatment of Alzheimer's disease. 抗淀粉样蛋白 beta 单克隆抗体治疗阿尔茨海默病的疗效和安全性系统综述。

IF 3.6 3区医学

Expert Opinion on Biological Therapy Pub Date : 2024-10-21 DOI: 10.1080/14712598.2024.2416947

Akanksha Chhabra, Siddhant Solanki, Prithvi Saravanabawan, Arun Venkiteswaran, NagaTarang Nimmathota, Nishi Manojkumar Modi

{"title":"A systematic review of the efficacy and safety of anti-amyloid beta monoclonal antibodies in treatment of Alzheimer's disease.","authors":"Akanksha Chhabra, Siddhant Solanki, Prithvi Saravanabawan, Arun Venkiteswaran, NagaTarang Nimmathota, Nishi Manojkumar Modi","doi":"10.1080/14712598.2024.2416947","DOIUrl":"https://doi.org/10.1080/14712598.2024.2416947","url":null,"abstract":"Introduction: Alzheimer's disease can cause dementia through brain matter degradation. This study investigates the monoclonal antibody usage for AD treatment, following PRISMA 2020 guidelines, and aims to discern the monoclonal antibody that offers the optimal balance of efficacy and safety for individuals with AD.Methods: A systematic search was conducted across databases such as PubMed, Cochrane Library, and clinical trial registries for randomized controlled trials. The quality of studies was assessed using the Cochrane risk of bias 2 tool. Cognitive function and daily activities were evaluated using MMSE, ADAS-Cog, and CDR-SB test data.Results: According to CDR-SB measurements, lecanemab showed effectiveness in reducing brain amyloid and cognitive decline, with a change from baseline of 1.21. Aducanumab resulted in a decrease of -0.39 (-22%). Bapineuzumab showed no significant benefit, with scores of 2.4 (2.8). Gantenerumab, scoring 1.69 (1.37, 2.01), reduces amyloid, particularly in early Alzheimer's stages. Crenezumab was ineffective, with a score of 3.61.Conclusion: The findings provide various perspectives. Lecanemab showed the most promise in brain amyloid reduction and decelerating cognitive decline compared to the other therapies. Further research is needed, highlighting the necessity of AD therapeutic research to alter AD's trajectory and provide reliable treatment.Protocol registration: www.crd.york.ac.uk/prospero identifier is CRD42024504358.","PeriodicalId":12084,"journal":{"name":"Expert Opinion on Biological Therapy","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2024-10-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142461282","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 evaluation of mirikizumab for the treatment of ulcerative colitis. 米利珠单抗治疗溃疡性结肠炎的评估。

IF 3.6 3区医学

Expert Opinion on Biological Therapy Pub Date : 2024-10-16 DOI: 10.1080/14712598.2024.2412650

Christopher White, Peter M Irving

{"title":"An evaluation of mirikizumab for the treatment of ulcerative colitis.","authors":"Christopher White, Peter M Irving","doi":"10.1080/14712598.2024.2412650","DOIUrl":"10.1080/14712598.2024.2412650","url":null,"abstract":"Introduction: Treatment of ulcerative colitis (UC) aims to reduce symptoms and complications by decreasing intestinal inflammation. A proportion of patients do not respond to, do not tolerate, or are inappropriate candidates for current therapies. Interleukin (IL)-23 is a therapeutic target and mirikizumabis the first p19-targeted IL-23 antibody approved for the treatment of moderately to severely active UC.Areas covered: This review summarizes the pro-inflammatory effects of IL-23 and outlines the pharmacokinetics of mirikizumab. It provides a synopsis of the available phase II and phase III evidence for the efficacy and safety of mirikizumab in UC.Expert opinion: The mirikizumab clinical development program demonstrated its superiority over placebo and its favorable safety profile in the treatment of UC. Its positioning in therapeutic algorithms remains to be fully understood but mirikizumab has proven efficacy in both advanced therapy (AT)-naïve and AT-experienced patients. The inclusion in the license of extended induction for non-responders as well as rescue intravenous dosing allows for flexibility in patient with limited primary response and secondary loss of response.","PeriodicalId":12084,"journal":{"name":"Expert Opinion on Biological Therapy","volume":null,"pages":null},"PeriodicalIF":3.6,"publicationDate":"2024-10-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142364934","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