Expert opinion on investigational drugs最新文献_第3页

Early psoriatic arthritis: clinical and therapeutic challenges. 早期银屑病关节炎：临床和治疗难题。

IF 4.9 2区医学

Expert opinion on investigational drugs Pub Date : 2024-09-01 Epub Date: 2024-08-07 DOI: 10.1080/13543784.2024.2383421

Francesco Caso, Luisa Costa, Matteo Megna, Mario Cascone, Francesco Maione, Roberto Giacomelli, Raffaele Scarpa, Piero Ruscitti

{"title":"Early psoriatic arthritis: clinical and therapeutic challenges.","authors":"Francesco Caso, Luisa Costa, Matteo Megna, Mario Cascone, Francesco Maione, Roberto Giacomelli, Raffaele Scarpa, Piero Ruscitti","doi":"10.1080/13543784.2024.2383421","DOIUrl":"10.1080/13543784.2024.2383421","url":null,"abstract":"Introduction: Psoriatic arthritis (PsA) is a chronic immunoinflammatory disease of the enthesis and adjacent synovium, skin, and nail, which early diagnosis may be crucial for starting a prompt therapeutic intervention. Theoretically, early treatment offers the advantage of acting on the reduction of the articular damage progression since initial phases of the disease.Areas covered: This review explores the challenges of clinical-diagnostic aspects and the underlying pathophysiology of early PsA phases, as well as the evidence evaluating the impact of early intervention on disease outcomes.Expert opinion: Main instruments for early PsA diagnosis include recognizing synovial-entheseal inflammatory signs at onset, improving screening PsA high-risk subjects, and increasing disease knowledge of physicians and patients with psoriasis or familial history. PsA continues to significantly impact on the Quality of Life of patients affected by the disease, making necessary to deeply study clinical manifestations, risk factors, and underlying immunoinflammatory mechanisms, as well as to identify biomarkers for early identification. Additionally, it remains a need to increase more evidence on understanding how early treatment of PsA and of psoriasis might influence the course of the disease.","PeriodicalId":12313,"journal":{"name":"Expert opinion on investigational drugs","volume":" ","pages":"945-965"},"PeriodicalIF":4.9,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141747889","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

Investigational new drug approval of DA-1241: what we know about GPR119 targeting for MASH therapy? DA-1241获批新药研究：我们对GPR119靶向治疗MASH了解多少？

IF 4.9 2区医学

Expert opinion on investigational drugs Pub Date : 2024-09-01 Epub Date: 2024-08-07 DOI: 10.1080/13543784.2024.2388592

Khaled Al Smadi, Ammar Qureshi, Besher Ashouri, Zeid Kayali

引用次数: 0

Factor IX stimulants in preclinical and early phase trials for hemophilia B treatment. 用于 B 型血友病治疗的临床前和早期试验中的因子 IX 促效剂。

IF 4.9 2区医学

Expert opinion on investigational drugs Pub Date : 2024-09-01 Epub Date: 2024-08-11 DOI: 10.1080/13543784.2024.2388565

Massimo Franchini, Daniele Focosi

{"title":"Factor IX stimulants in preclinical and early phase trials for hemophilia B treatment.","authors":"Massimo Franchini, Daniele Focosi","doi":"10.1080/13543784.2024.2388565","DOIUrl":"10.1080/13543784.2024.2388565","url":null,"abstract":"Introduction: Hemophilia B is a X-linked rare inherited bleeding disorder characterized by coagulation factor IX (FIX) deficiency. Therapy for hemophilia B is aimed at replacing the FIX deficiency by means of several plasma-derived or recombinant FIX products. The recent availability of recombinant FIX concentrates with a prolonged FIX half-life represented a great technological advance, permitting more spaced drug infusions and reducing treatment burden among hemophilia B patients.Areas covered: This review summarizes the main preclinical and phase 1/2 studies investigating the innovative hemostatic products for hemophilia B replacement therapy.Expert opinion: The significant recent technological advantages in the treatment of hemophilia B has led to the development of innovative FIX products aimed at further extending FIX half-life and using increasingly effective and convenient modes of administration. These novel hemostatic agents, currently in the preclinical or early clinical phase of development, carry the potential of improving patients' health status and quality of life. Continuous research is anyway needed to offer such patients a concrete chance of conducting a normal existence, like to non-affected age-matched individuals.","PeriodicalId":12313,"journal":{"name":"Expert opinion on investigational drugs","volume":" ","pages":"939-944"},"PeriodicalIF":4.9,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141888910","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

Investigative agents for atrial fibrillation: agonists and stimulants, progress and expectations. 心房颤动的研究药物：激动剂和兴奋剂、进展和期望。

IF 4.9 2区医学

Expert opinion on investigational drugs Pub Date : 2024-09-01 Epub Date: 2024-08-08 DOI: 10.1080/13543784.2024.2388583

Ruben Casado-Arroyo, Marco Bernardi, Pierre Sabouret, Giuseppe Franculli, Juan Tamargo, Luigi Spadafora, Nicolas Lellouche, Giuseppe Biondi-Zoccai, Peter P Toth, Maciej Banach

{"title":"Investigative agents for atrial fibrillation: agonists and stimulants, progress and expectations.","authors":"Ruben Casado-Arroyo, Marco Bernardi, Pierre Sabouret, Giuseppe Franculli, Juan Tamargo, Luigi Spadafora, Nicolas Lellouche, Giuseppe Biondi-Zoccai, Peter P Toth, Maciej Banach","doi":"10.1080/13543784.2024.2388583","DOIUrl":"10.1080/13543784.2024.2388583","url":null,"abstract":"Introduction: Atrial fibrillation (AF) is the most common type of cardiac arrhythmia. Its prevalence has increased due to worldwide populations that are aging in combination with the growing incidence of risk factors associated. Recent advances in our understanding of AF pathophysiology and the identification of nodal players involved in AF-promoting atrial remodeling highlights potential opportunities for new therapeutic approaches.Areas covered: This detailed review summarizes recent developments in the field antiarrhythmic drugs in the field AF.Expert opinion: The current situation is far than optimal. Despite clear unmet needs in drug development in the field of AF treatment, the current development of new drugs is absent. The need for a molecule with absence of cardiac and non-cardiac toxicity in the short and long term is a limitation in the field. Improvement in the understanding of AF genetics, pathophysiology, molecular alterations, big data and artificial intelligence with the objective to provide a personalized AF treatment will be the cornerstone of AF treatment in the coming years.","PeriodicalId":12313,"journal":{"name":"Expert opinion on investigational drugs","volume":" ","pages":"967-978"},"PeriodicalIF":4.9,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141888911","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

Investigational CXCR4 inhibitors in early phase development for the treatment of hematological malignancies. 用于治疗血液恶性肿瘤的处于早期研发阶段的在研 CXCR4 抑制剂。

IF 4.9 2区医学

Expert opinion on investigational drugs Pub Date : 2024-09-01 Epub Date: 2024-08-08 DOI: 10.1080/13543784.2024.2388567

Enrica Antonia Martino, Antonella Bruzzese, Caterina Labanca, Francesco Mendicino, Eugenio Lucia, Virginia Olivito, Gaia Stanzione, Annamaria Zimbo, Stefano Pozzi, Antonino Neri, Fortunato Morabito, Ernesto Vigna, Massimo Gentile

{"title":"Investigational CXCR4 inhibitors in early phase development for the treatment of hematological malignancies.","authors":"Enrica Antonia Martino, Antonella Bruzzese, Caterina Labanca, Francesco Mendicino, Eugenio Lucia, Virginia Olivito, Gaia Stanzione, Annamaria Zimbo, Stefano Pozzi, Antonino Neri, Fortunato Morabito, Ernesto Vigna, Massimo Gentile","doi":"10.1080/13543784.2024.2388567","DOIUrl":"10.1080/13543784.2024.2388567","url":null,"abstract":"Introduction: CXCR4/CXCL12 axis regulates cell proliferation, survival, and differentiation, as well as the homing and mobilization of hematopoietic stem cells (HSCs) from bone marrow niches to the peripheral blood. Furthermore, CXCR4 and CXCL12 are key mediators of cross-talk between hematological malignancies and their microenvironments. CXCR4 overexpression drives disease progression, boosts tumor cell survival, and promotes chemoresistance, leading to poor prognosis.Areas covered: In light of these discoveries, scientific investigations, and clinical trials have underscored the therapeutic promise found in small-molecule antagonists like plerixafor, peptides/peptidomimetics, such as BKT140, monoclonal antibodies like PF-06747143 and ulocuplumab, as well as microRNAs. Their efficacy is evident in reducing tumor burden, inducing apoptosis and sensitizing malignant cells to conventional chemotherapies. This overview delves into the pathogenic role of the CXC4/CXCL12 axis in hematological neoplasms and examines the clinical application of key CXCR4 antagonists.Expert opinion: The information collectively emphasizes the potential of CXCR4 antagonists as a therapeutic strategy for hematologic malignancies, showcasing advancements in preclinical and clinical studies. As these therapeutic strategies progress through clinical trials, their potential to reshape the prognosis of hematologic malignancies will become increasingly apparent.","PeriodicalId":12313,"journal":{"name":"Expert opinion on investigational drugs","volume":" ","pages":"915-924"},"PeriodicalIF":4.9,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141878562","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

Medical treatments for abdominal aortic aneurysm: an overview of clinical trials. 腹主动脉瘤的药物治疗：临床试验概述。

IF 4.9 2区医学

Expert opinion on investigational drugs Pub Date : 2024-09-01 Epub Date: 2024-07-11 DOI: 10.1080/13543784.2024.2377747

Jinyi Chen, Lanting Hu, Zhenjie Liu

{"title":"Medical treatments for abdominal aortic aneurysm: an overview of clinical trials.","authors":"Jinyi Chen, Lanting Hu, Zhenjie Liu","doi":"10.1080/13543784.2024.2377747","DOIUrl":"10.1080/13543784.2024.2377747","url":null,"abstract":"Introduction: Abdominal aortic aneurysm is a progressive, segmental, abdominal aortic dilation associated with a high mortality rate. Abdominal aortic aneurysms with diameters larger than 55 mm are associated with a high risk of rupture, and the most effective treatment options are surgical repair. Close observation and lifestyle adjustments are recommended for smaller abdominal aortic aneurysms with lower rupture risk. The development of medical therapies that limit or prevent the progression, expansion, and eventual rupture of abdominal aortic aneurysms remains an unmet clinical need.Areas covered: This review provides an overview of completed and ongoing clinical trials examining the efficacies of various drug classes, including antibiotics, antihypertensive drugs, hypolipidemic drugs, hypoglycemic drugs, and other potential therapies for abdominal aortic aneurysms. A search of PubMed, Web of Science, Clinical Trials, and another six clinical trial registries was conducted in January 2024.Expert opinion: None of the drugs have enough evidence to indicate that they can effectively inhibit the dilation of abdominal aortic aneurysm. More clinical trial data is required to support the efficacy of propranolol. Future research should also explore different drug delivery mechanisms, such as nanoparticles, to elevate drug concentration at the aneurysm wall.","PeriodicalId":12313,"journal":{"name":"Expert opinion on investigational drugs","volume":" ","pages":"979-992"},"PeriodicalIF":4.9,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141558496","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

Zanzalintinib (XL092): a next-generation tyrosine kinase inhibitor-comprehensive review of early safety & efficacy data. 赞扎林替尼（XL092）：新一代酪氨酸激酶抑制剂--早期安全性和有效性数据的全面回顾。

IF 4.9 2区医学

Expert opinion on investigational drugs Pub Date : 2024-09-01 Epub Date: 2024-08-07 DOI: 10.1080/13543784.2024.2388571

James Yu, Robin Park, Alireza Tojjari, Arezoo Sadeghipour, Ali Saeed, Anwaar Saeed

{"title":"Zanzalintinib (XL092): a next-generation tyrosine kinase inhibitor-comprehensive review of early safety & efficacy data.","authors":"James Yu, Robin Park, Alireza Tojjari, Arezoo Sadeghipour, Ali Saeed, Anwaar Saeed","doi":"10.1080/13543784.2024.2388571","DOIUrl":"10.1080/13543784.2024.2388571","url":null,"abstract":"Introduction: Zanzalintinib (XL092) is a next-generation anti-VEGFR-related multi-targeted TKI that exhibits immunomodulatory effects.Areas covered: This review explores preclinical and clinical data, along with the future directions associated with zanzalintinib and its combination with immune checkpoint inhibitors (ICIs).Expert opinion: In addition to its anti-VEGFR activity, zanzalintinib demonstrates potential synergistic effects with ICIs through its immunomodulatory impact, attributed to its inhibition of MET and TAM kinases. Recent preclinical studies provide compelling evidence supporting this synergistic potential. Furthermore, a recent phase 1 dose escalation study confirmed the tolerability of the zanzalintinib and anti-PDL1 combination without major safety concerns.Multiple ongoing clinical trials are investigating the combination of zanzalintinib and ICIs across various solid tumor types, including phase 3 studies for renal cell carcinoma, colorectal, and head and neck cancer. These trials aim to elucidate the therapeutic role of this new-generation TKI and ICI combination.However, the identification of reliable predictive biomarkers for the zanzalintinib and ICI combination presents significant challenges. Given the intricate nature of their mechanistic rationale and the difficulties in identifying reliable biomarkers for combined anti-angiogenesis and ICI therapies, addressing this challenge remains a priority for ongoing and future research.","PeriodicalId":12313,"journal":{"name":"Expert opinion on investigational drugs","volume":" ","pages":"887-895"},"PeriodicalIF":4.9,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141888912","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

An overview of conventional and investigational phosphodiesterase 5 inhibitors for treating erectile dysfunction and other conditions 用于治疗勃起功能障碍和其他疾病的传统和试验性磷酸二酯酶 5 抑制剂概述

IF 6.1 2区医学

Expert opinion on investigational drugs Pub Date : 2024-08-03 DOI: 10.1080/13543784.2024.2388569

Ezzat A. Ismail, Ahmed I. El-Sakka

引用次数: 0

Spleen tyrosine kinase (SYK): an emerging target for the assemblage of small molecule antitumor agents 脾脏酪氨酸激酶（SYK）：小分子抗肿瘤药物组合的新靶点

IF 6.1 2区医学

Expert opinion on investigational drugs Pub Date : 2024-08-03 DOI: 10.1080/13543784.2024.2388559

Charanjit Kaur, Amandeep Thakur, Ke-Chi Liou, Neralla Vijayakameswara Rao, Kunal Nepali

引用次数: 0

VERVE-101, a CRISPR base-editing therapy designed to permanently inactivate hepatic PCSK9 and reduce LDL-cholesterol. VERVE-101 是一种 CRISPR 碱基编辑疗法，旨在永久性失活肝脏 PCSK9 并降低低密度脂蛋白胆固醇。

IF 4.9 2区医学

Expert opinion on investigational drugs Pub Date : 2024-08-01 Epub Date: 2024-06-21 DOI: 10.1080/13543784.2024.2369747

Amanda J Hooper, Xuan L Tang, John R Burnett

引用次数: 0