Expert opinion on investigational drugs最新文献_第5页

Lepodisiran, an siRNA targeting lipoprotein(a) for the potential future treatment of cardiovascular disease. Lepodisiran 是一种靶向脂蛋白（a）的 siRNA，未来可能用于治疗心血管疾病。

IF 4.9 2区医学

Expert opinion on investigational drugs Pub Date : 2024-07-01 Epub Date: 2024-05-08 DOI: 10.1080/13543784.2024.2352129

P Mihika S Fernando, Amanda J Hooper, John R Burnett

引用次数: 0

Will new investigational drugs change the way we treat Charcot-Marie-Tooth disease? 新的研究药物是否会改变我们治疗黑热病的方法？

IF 4.9 2区医学

Expert opinion on investigational drugs Pub Date : 2024-07-01 Epub Date: 2024-05-08 DOI: 10.1080/13543784.2024.2352635

Amedeo De Grado, Chiara Pisciotta, Paola Saveri, Davide Pareyson

引用次数: 0

Investigational agents for polymyalgia rheumatica treatment: assessing the critical needs for future development. 治疗多发性风湿病的研究药物：评估未来发展的关键需求。

IF 4.9 2区医学

Expert opinion on investigational drugs Pub Date : 2024-07-01 Epub Date: 2024-06-16 DOI: 10.1080/13543784.2024.2366847

Luca Iorio, Roberto Padoan, Milena Bond, Christian Dejaco

{"title":"Investigational agents for polymyalgia rheumatica treatment: assessing the critical needs for future development.","authors":"Luca Iorio, Roberto Padoan, Milena Bond, Christian Dejaco","doi":"10.1080/13543784.2024.2366847","DOIUrl":"10.1080/13543784.2024.2366847","url":null,"abstract":"Introduction: Polymyalgia rheumatica (PMR) is an inflammatory rheumatic disorder characterized by pain and stiffness in the shoulder and pelvic girdles, constitutional symptoms, and elevated acute-phase reactants. Glucocorticoids (GCs) remain the first-choice treatment for PMR, but relapses are common. Identification of steroid-sparing agents is therefore of utmost importance.Areas covered: The efficacy of conventional immunosuppressive drugs is controversial. The use of interleukin (IL)-6 receptor inhibitors proved to be effective and safe in treating PMR patients. Currently, there are 12 ongoing clinical trials exploring potential treatments such as leflunomide, low-dose IL-2, rituximab, abatacept, secukinumab, Janus kinase inhibitors, and selective inhibitors like SPI-62 and ABBV 154.Expert opinion: The high efficacy of IL-6 R receptor inhibitors as well as the numerous drug trials currently recruiting suggest that several therapeutic options will be available in the near future. Accurate diagnosis and early stratification of PMR patients according to the giant cell arteritis-PMR Spectrum Disease 'GPSD' and potential risk factors for relapsing disease or GC-related adverse events are crucial to identify patients who would benefit most from GC-sparing agents. The development of internationally accepted definitions for remission and relapse is urgently needed. Early referral strategies to specialist settings would improve disease stratification and personalized treatment.","PeriodicalId":12313,"journal":{"name":"Expert opinion on investigational drugs","volume":" ","pages":"671-676"},"PeriodicalIF":4.9,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141327409","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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

The role of extracellular vesicles in immune cell exhaustion and resistance to immunotherapy. 细胞外囊泡在免疫细胞衰竭和免疫疗法抗药性中的作用。

IF 4.9 2区医学

Expert opinion on investigational drugs Pub Date : 2024-07-01 Epub Date: 2024-05-30 DOI: 10.1080/13543784.2024.2360209

Ava Aghakhani, Parmida Sadat Pezeshki, Nima Rezaei

{"title":"The role of extracellular vesicles in immune cell exhaustion and resistance to immunotherapy.","authors":"Ava Aghakhani, Parmida Sadat Pezeshki, Nima Rezaei","doi":"10.1080/13543784.2024.2360209","DOIUrl":"10.1080/13543784.2024.2360209","url":null,"abstract":"Introduction: Extracellular vesicles (EVs) are membrane-bound nanoparticles for intercellular communication. Subtypes of EVs, namely exosomes and microvesicles transfer diverse, bioactive cargo to their target cells and eventually interfere with immune responses. Despite being a promising approach, cancer immunotherapy currently faces several challenges including immune resistance. EVs secreted from various sources in the tumor microenvironment provoke immune cell exhaustion and lower the efficacy of immunological treatments, such as CAR T cells and immune checkpoint inhibitors.Areas covered: This article goes through the mechanisms of action of various types of EVs in inhibiting immune response and immunotherapies, and provides a comprehensive review of EV-based treatments.Expert opinion: By making use of the distinctive features of EVs, natural or modified EVs are innovatively utilized as novel cancer therapeutics. They are occasionally coupled with currently established treatments to overcome their inadequacies. Investigating the properties and interactions of EVs and EV-based treatments is crucial for determining future steps in cancer therapeutics.","PeriodicalId":12313,"journal":{"name":"Expert opinion on investigational drugs","volume":" ","pages":"721-740"},"PeriodicalIF":4.9,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141096860","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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

Safety, pharmacokinetics and pharmacodynamics of SHR-1703, an innovative long-acting anti-interleukin-5 monoclonal antibody, in healthy subjects: a randomized, double-blind, dose-escalation, placebo-controlled phase I study. 创新型长效抗白细胞介素-5 单克隆抗体 SHR-1703 在健康受试者中的安全性、药代动力学和药效学：一项随机、双盲、剂量递增、安慰剂对照的 I 期研究。

IF 4.9 2区医学

Expert opinion on investigational drugs Pub Date : 2024-07-01 Epub Date: 2024-06-03 DOI: 10.1080/13543784.2024.2361065

Ling Yang, Yuan Fang, Yuan Luo, Meng Fu, Kai Shen, Zhu Luo

{"title":"Safety, pharmacokinetics and pharmacodynamics of SHR-1703, an innovative long-acting anti-interleukin-5 monoclonal antibody, in healthy subjects: a randomized, double-blind, dose-escalation, placebo-controlled phase I study.","authors":"Ling Yang, Yuan Fang, Yuan Luo, Meng Fu, Kai Shen, Zhu Luo","doi":"10.1080/13543784.2024.2361065","DOIUrl":"10.1080/13543784.2024.2361065","url":null,"abstract":"Objective: SHR-1703 is a novel humanized IgG1 monoclonal antibody with high IL-5 affinity and prolonged half-life, aiming to control eosinophil-related diseases. The study intended to evaluate pharmacokinetics, pharmacodynamics, immunogenicity, safety, and tolerability of SHR-1703 in healthy subjects.Methods: A single-center, randomized, double-blind, placebo-controlled, single-dose escalation phase I study was conducted. 42 subjects were allocated to sequentially receive single subcutaneous injection of 20, 75, 150, 300, and 400 mg SHR-1703 or placebo.Results: After administration, SHR-1703 was slowly absorbed with median Tmax ranging from 8.5 to 24.5 days. Mean t1/2 in 150 to 400 mg doses was 86 to 100 days. Cmax and AUC increased in nearly dose-proportional pattern over range of 75 to 400 mg SHR-1703. After receiving SHR-1703, peripheral blood eosinophils (EOS) greatly decreased from baseline, which showed no significant change from baseline in placebo group. Magnitude and duration of reduction of EOS rose with increased dosing of SHR-1703. In 400 mg dose, remarkable efficacy of reducing EOS maintained up to approximately 6 months post single administration. Moreover, SHR-1703 exhibited low immunogenicity (2.9%), favorable safety, and tolerability in healthy subjects.Conclusion: Pharmacokinetics, pharmacodynamics, immunogenicity, safety, and tolerability of SHR-1703 support further clinical development of SHR-1703 in eosinophil-associated diseases.Clinical trial registration: The study was registered on the ClinicalTrials.gov (identifier: NCT04480762).","PeriodicalId":12313,"journal":{"name":"Expert opinion on investigational drugs","volume":" ","pages":"741-752"},"PeriodicalIF":4.9,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141157178","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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

Clinical application and potential pluripotent effects of hepatocyte growth factor in spinal cord injury regeneration. 肝细胞生长因子在脊髓损伤再生中的临床应用和潜在多能效应。

IF 4.9 2区医学

Expert opinion on investigational drugs Pub Date : 2024-07-01 Epub Date: 2024-05-29 DOI: 10.1080/13543784.2024.2360191

Shogo Hashimoto, Narihito Nagoshi, Masaya Nakamura, Hideyuki Okano

{"title":"Clinical application and potential pluripotent effects of hepatocyte growth factor in spinal cord injury regeneration.","authors":"Shogo Hashimoto, Narihito Nagoshi, Masaya Nakamura, Hideyuki Okano","doi":"10.1080/13543784.2024.2360191","DOIUrl":"10.1080/13543784.2024.2360191","url":null,"abstract":"Introduction: Spinal cord injury (SCI) is a condition in which the spinal cord parenchyma is damaged by various factors. The mammalian central nervous system has been considered unable to regenerate once damaged, but recent progress in basic research has gradually revealed that injured neural cells can indeed regenerate. Drug therapy using novel agents is being actively investigated as a new treatment for SCI. One notable treatment method is regeneration therapy using hepatocyte growth factors (HGF).Area covered: HGF has pluripotent neuroregenerative actions, as indicated by its neuroprotective and regenerative effects on the microenvironment and damaged cells, respectively. This review examines these effects in various phases of SCI, from basic research to clinical studies, and the application of this treatment to other diseases.Expert opinion: In regenerative medicine for SCI, drug therapies have tended to be more likely to be developed compared to cell replacement treatment. Nevertheless, there are still challenges to be addressed for these clinical applications due to a wide variety of pathology and animal experimental models of basic study, but HGF could be an effective treatment for SCI with expanded application.","PeriodicalId":12313,"journal":{"name":"Expert opinion on investigational drugs","volume":" ","pages":"713-720"},"PeriodicalIF":4.9,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141086716","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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

Therapeutic potential of cannabidiol (CBD) in the treatment of cardiovascular diseases. 大麻二酚（CBD）在治疗心血管疾病方面的治疗潜力。

IF 4.9 2区医学

Expert opinion on investigational drugs Pub Date : 2024-07-01 Epub Date: 2024-05-09 DOI: 10.1080/13543784.2024.2351513

Nadia Martinez Naya, Jazmin Kelly, Austin Hogwood, Antonio Abbate, Stefano Toldo

{"title":"Therapeutic potential of cannabidiol (CBD) in the treatment of cardiovascular diseases.","authors":"Nadia Martinez Naya, Jazmin Kelly, Austin Hogwood, Antonio Abbate, Stefano Toldo","doi":"10.1080/13543784.2024.2351513","DOIUrl":"10.1080/13543784.2024.2351513","url":null,"abstract":"Introduction: Cannabidiol (CBD) is the primary non-psychoactive chemical derived from Cannabis Sativa, and its growing popularity is due to its potential therapeutic properties while avoiding the psychotropic effects of other phytocannabinoids, such as tetrahydrocannabinol (THC). Numerous pre-clinical studies in cellular and animal models and human clinical trials have demonstrated a positive impact of CBD on physiological and pathological processes. Recently, the FDA approved its use for the treatment of seizures, and clinical trials to test the efficacy of CBD in myocarditis and pericarditis are ongoing.Areas covered: We herein reviewed the current literature on the reported effects of CBD in the cardiovascular system, highlighting the physiological effects and the outcomes of using CBD as a therapeutic tool in pathological conditions to address this significant global health concern.Expert opinion: The comprehensive examination of the literature emphasizes the potential of CBD as a therapeutic option for treating cardiovascular diseases through its anti-inflammatory, vasodilatory, anti-fibrotic, and antioxidant properties in different conditions such as diabetic cardiomyopathy, myocarditis, doxorubicin-induced cardiotoxicity, and ischemia-reperfusion injury.","PeriodicalId":12313,"journal":{"name":"Expert opinion on investigational drugs","volume":" ","pages":"699-712"},"PeriodicalIF":4.9,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140855239","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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

Therapies in preclinical and in early clinical development for the treatment of urinary tract infections: from pathogens to therapies. 治疗尿路感染的临床前和早期临床开发疗法：从病原体到疗法。

IF 4.9 2区医学

Expert opinion on investigational drugs Pub Date : 2024-07-01 Epub Date: 2024-05-09 DOI: 10.1080/13543784.2024.2351509

Virginia Fuochi, Salvatore Furnari, Laura Trovato, Maddalena Calvo, Pio Maria Furneri

{"title":"Therapies in preclinical and in early clinical development for the treatment of urinary tract infections: from pathogens to therapies.","authors":"Virginia Fuochi, Salvatore Furnari, Laura Trovato, Maddalena Calvo, Pio Maria Furneri","doi":"10.1080/13543784.2024.2351509","DOIUrl":"10.1080/13543784.2024.2351509","url":null,"abstract":"Introduction: Urinary tract infections (UTIs) are a prevalent health challenge characterized by the invasion and multiplication of microorganisms in the urinary system. The continuous exploration of novel therapeutic interventions is imperative. Advances in research offer hope for revolutionizing the management of UTIs and improving the overall health outcomes for individuals affected by these infections.Areas covered: This review aimed to provide an overview of existing treatments for UTIs, highlighting their strengths and limitations. Moreover, we explored and analyzed the latest therapeutic modalities under clinical development. Finally, the review offered a picture into the potential implications of these therapies on the future landscape of UTIs treatment, discussing possible advancements and challenges for further research.Expert opinion: Comprehensions into the pathogenesis of UTIs have been gleaned from foundational basic science studies, laying the groundwork for the exploration of novel therapeutic interventions. The primary source of evidence originates predominantly from animal studies conducted on murine models. Nevertheless, the lack of clinical trials interferes the acquisition of robust evidence in humans. The challenges presented by the heterogeneity and virulence of uropathogens add an additional layer of complexity, posing an obstacle that scientists and clinicians are actively grappling with in their pursuit of effective solutions.","PeriodicalId":12313,"journal":{"name":"Expert opinion on investigational drugs","volume":" ","pages":"677-698"},"PeriodicalIF":4.9,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140854852","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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

The role of phosphodiesterase 9A inhibitors in heart failure. 磷酸二酯酶 9A 抑制剂在心力衰竭中的作用。

IF 4.9 2区医学

Expert opinion on investigational drugs Pub Date : 2024-06-01 Epub Date: 2024-05-08 DOI: 10.1080/13543784.2024.2349813

Saam Foroshani, Avrohom Karp, Wilbert S Aronow, Gregg M Lanier

引用次数: 0

Frontotemporal dementia: from genetics to therapeutic approaches. 额颞叶痴呆症：从遗传学到治疗方法。

IF 4.9 2区医学

Expert opinion on investigational drugs Pub Date : 2024-06-01 Epub Date: 2024-05-09 DOI: 10.1080/13543784.2024.2349286

Francesca R Buccellato, Marianna D'Anca, Gianluca Martino Tartaglia, Massimo Del Fabbro, Daniela Galimberti

引用次数: 0