Aikaterini Tsiogka, Eleni Paschou, Dimitra Koumaki, E. Vakirlis, S. Gregoriou
{"title":"Interleukin antagonists for atopic dermatitis: a new era of therapy.","authors":"Aikaterini Tsiogka, Eleni Paschou, Dimitra Koumaki, E. Vakirlis, S. Gregoriou","doi":"10.1080/13543784.2024.2347294","DOIUrl":"https://doi.org/10.1080/13543784.2024.2347294","url":null,"abstract":"INTRODUCTION\u0000Over the last decade, increasing understanding of the immunopathogenesis of atopic dermatitis (AD) enabled the recognition of multiple therapeutic targets and subsequently the development of novel, highly effective systemic treatments, including interleukin (IL)-antagonists. To date, the IL-4Ra-inhibitor dupilumab, and the IL-13 inhibitor tralokinumab, have gained regulatory approval in Europe for the treatment of moderate-to-severe AD, while more than 70 new therapeutics are currently in development.\u0000\u0000\u0000AREAS COVERED\u0000In this review, we address the role of ILs in the pathogenesis of AD and provide an overview of the novel and investigational IL-antagonists, as regards their efficacy and safety on moderate-to-severe AD.\u0000\u0000\u0000EXPERT OPINION\u0000Current data have established IL-4 and IL-13 inhibitors as effective and safe for the treatment of moderate-to-severe AD, as regards the rapid control of flares as well as the long-term remission of the disease. Data regarding the efficacy and safety of other IL-inhibitors, including those targeting IL-31, IL-22, IL-33, IL-36 and IL-18, are accumulating. There is still an unmet need for real-world-evidence studies and head-to-head studies for both currently available and future agents in AD treatment. Establishing predictive biomarkers of treatment response in a disorder of such considerable heterogenicity might help physicians pursue a patient-tailored therapeutic response.","PeriodicalId":12313,"journal":{"name":"Expert opinion on investigational drugs","volume":null,"pages":null},"PeriodicalIF":6.1,"publicationDate":"2024-04-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140661194","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}
R. Pirchio, R. S. Auriemma, A. Vergura, R. Pivonello, A. Colao
{"title":"Investigational drugs for the Treatment of Acromegaly: new agents to transform therapy.","authors":"R. Pirchio, R. S. Auriemma, A. Vergura, R. Pivonello, A. Colao","doi":"10.1080/13543784.2024.2343056","DOIUrl":"https://doi.org/10.1080/13543784.2024.2343056","url":null,"abstract":"INTRODUCTION\u0000Disease control is essential to decrease morbidity burden and mortality in acromegaly patients. In the last decades, the availability of new drugs increased the rate of disease control. However, up to 55% of patients remain uncontrolled despite available treatment strategies in real-world data. The reasons for this finding may include poor adherence, inadequate tolerability, therapeutic inertia, and high costs. Since acromegaly is a chronic disease and medical therapy is usually life-long, patient's adherence to treatment is fundamental in both achieving and maintaining disease control. Less invasive routes of administration could improve adherence and concur to increase disease control rate.\u0000\u0000\u0000AREAS COVERED\u0000The aim of current review is to provide a detailed update about investigational drugs for acromegaly treatment currently under investigation as paltusotine, ONO-5788, AP102, GT-02037, ISIS 766,720, CAM2024, Lanreotide PRF, DP1038, MTD201, solid dose injection of octreotide.\u0000\u0000\u0000EXPERT OPINION\u0000Medical therapy of acromegaly is an evolving field. Current studies are addressing patient's needing for both new molecules and less invasive routes of administration for already existing drugs. It cannot be ruled out that drugs currently used for other disease such as cancers could be considered in the future for the treatment of acromegaly.","PeriodicalId":12313,"journal":{"name":"Expert opinion on investigational drugs","volume":null,"pages":null},"PeriodicalIF":6.1,"publicationDate":"2024-04-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140668463","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}
Giulio Evangelisti, Simone Ferrero, Umberto Perrone, Claudio Gustavino, Eugenio Volpi, Alberto Izzotti, Fabio Barra
{"title":"Experimental and new investigational drugs for the treatment of uterine fibroids","authors":"Giulio Evangelisti, Simone Ferrero, Umberto Perrone, Claudio Gustavino, Eugenio Volpi, Alberto Izzotti, Fabio Barra","doi":"10.1080/13543784.2024.2343786","DOIUrl":"https://doi.org/10.1080/13543784.2024.2343786","url":null,"abstract":"Uterine fibroids, the most prevalent benign tumors among reproductive-age women, pose treatment challenges that range from surgical interventions to medical therapies for symptom control. Progestin...","PeriodicalId":12313,"journal":{"name":"Expert opinion on investigational drugs","volume":null,"pages":null},"PeriodicalIF":6.1,"publicationDate":"2024-04-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140573646","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}
Irene Capelli, Sarah Lerario, Francesca Ciurli, Gian Marco Berti, Valeria Aiello, Michele Provenzano, Gaetano Manna
{"title":"Investigational agents for autosomal dominant polycystic kidney disease: preclinical and early phase study insights","authors":"Irene Capelli, Sarah Lerario, Francesca Ciurli, Gian Marco Berti, Valeria Aiello, Michele Provenzano, Gaetano Manna","doi":"10.1080/13543784.2024.2342327","DOIUrl":"https://doi.org/10.1080/13543784.2024.2342327","url":null,"abstract":"Autosomal Dominant Polycystic Kidney Disease (ADPKD) is the most common inherited kidney condition caused by a single gene mutation. It leads patients to kidney failure in more than 50% of cases by...","PeriodicalId":12313,"journal":{"name":"Expert opinion on investigational drugs","volume":null,"pages":null},"PeriodicalIF":6.1,"publicationDate":"2024-04-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140573645","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}
{"title":"Sodium/Bile acid co-transporter inhibitors currently in preclinical or early clinical development for the treatment of primary biliary cholangitis","authors":"Abhishek Gairola, Aaron Wetten, Jessica Dyson","doi":"10.1080/13543784.2024.2343789","DOIUrl":"https://doi.org/10.1080/13543784.2024.2343789","url":null,"abstract":"Pruritus is common and often undertreated in patients with primary biliary cholangitis (PBC). Existing treatments largely have an aging and low-quality evidence base, and studies included only smal...","PeriodicalId":12313,"journal":{"name":"Expert opinion on investigational drugs","volume":null,"pages":null},"PeriodicalIF":6.1,"publicationDate":"2024-04-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140573650","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}
{"title":"Investigational regenerative medicine for non-traumatic osteonecrosis of the femoral head: a survey of registered clinical trials.","authors":"Yutaka Kuroda, Toshiyuki Kawai, Yaichiro Okuzu, Yugo Morita, Shuichi Matsuda","doi":"10.1080/13543784.2024.2326622","DOIUrl":"10.1080/13543784.2024.2326622","url":null,"abstract":"<p><strong>Introduction: </strong>Osteonecrosis of the femoral head (ONFH) is a refractory disease requiring joint replacement in young patients. Regenerative therapies have been developed.</p><p><strong>Areas covered: </strong>This study surveyed clinical trials on regenerative medicine for ONFH. We extracted clinical trials on non-traumatic ONFH from the websites of five publicly available major registries (EuropeanUnion Clinical Trials Register ([EU-CTR],ClinicalTrials.gov, Chinese ClinicalTrial Registry [ChiCTR], University Hospital Medical InformationNetwork - Clinical Trial Registry [UMIN-CTR] and Australian New Zealand Clinical Trials Registry [ANZCTR]).The trials were classified into six categories based on purpose: surgical treatment, non-drug conservative treatment, conservative drug treatment, therapeutic strategy, diagnosis and pathogenesis, and regenerative therapy.) We extracted 169 clinical trials on ONFH. Of these, 37 were on regenerative medicine, including 29 on cell therapy. Surgical treatment was the most common treatment, followed by regenerative therapy.There were 9 clinical trials registered in the EU-CTR, with 5 on regenerative medicine; 79 trials registered on ClinicalTrials.gov, with 24 on regenerativemedicine; 54 trials registered in the ChiCTR, with 6 on regenerative medicine.</p><p><strong>Expert opinion: </strong>The focus of the joint-preserving surgery has shifted to regenerative therapy based on using cell therapy in early-stage ONFH. The global standardisation of regenerative therapy is still ongoing.</p>","PeriodicalId":12313,"journal":{"name":"Expert opinion on investigational drugs","volume":null,"pages":null},"PeriodicalIF":6.1,"publicationDate":"2024-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140021305","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}
Amelia K Mardon, Lucy Whitaker, Toobah Farooqi, Jane Girling, Claire Henry, Carolyn Ee, Jordan Tewhaiti-Smith, Mike Armour
{"title":"Investigational drugs for the treatment of dysmenorrhea.","authors":"Amelia K Mardon, Lucy Whitaker, Toobah Farooqi, Jane Girling, Claire Henry, Carolyn Ee, Jordan Tewhaiti-Smith, Mike Armour","doi":"10.1080/13543784.2024.2326627","DOIUrl":"10.1080/13543784.2024.2326627","url":null,"abstract":"<p><strong>Introduction: </strong>Dysmenorrhea is the most common cause of gynecological pain among women that has considerable impact on quality of life and psychosocial wellbeing. Non-steroidal anti-inflammatory drugs (NSAIDs) and hormonal therapies are most commonly used to treat dysmenorrhea. However, given these drugs are often associated with bothersome side effects and are less effective when there is an underlying cause contributing to dysmenorrhea (e.g. endometriosis), a patient-centered approach to managing dysmenorrhea is important. Various new drugs are currently being investigated for the treatment of primary and secondary dysmenorrhea.</p><p><strong>Areas covered: </strong>This review provides an updated overview on new therapeutic targets and investigational drugs for the treatment of primary and secondary dysmenorrhea. The authors describe the clinical development and implications of these drugs.</p><p><strong>Expert opinion: </strong>Among the investigative drugs discussed in this review, anti-inflammatories show the most promising results for the treatment of dysmenorrhea. However, given some trials have considerable methodological limitations, many drugs cannot be currently recommended. Research focused on understanding the mechanisms involved in menstruation and its associated symptoms will be important to identify new therapeutic targets for dysmenorrhea. Further robust clinical trials are required to better understand the efficacy and safety of investigational drugs for treating primary and secondary dysmenorrhea.</p>","PeriodicalId":12313,"journal":{"name":"Expert opinion on investigational drugs","volume":null,"pages":null},"PeriodicalIF":6.1,"publicationDate":"2024-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140021304","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}
{"title":"Novel preclinical developments of the primary sclerosing cholangitis treatment landscape.","authors":"Aalam Sohal, Kris V Kowdley","doi":"10.1080/13543784.2024.2330738","DOIUrl":"10.1080/13543784.2024.2330738","url":null,"abstract":"<p><strong>Introduction: </strong>Primary sclerosing cholangitis (PSC) is a chronic cholestatic liver disease associated with inflammation, fibrosis, and destruction of intra- and extrahepatic bile ducts. Despite substantial recent advances in our understanding of PSC, the only proven treatment of PSC is liver transplantation. There is an urgent unmet need to find medical therapies for this disorder.</p><p><strong>Areas covered: </strong>Multiple drugs are currently under evaluation as therapeutic options for this disease. This article summarizes the literature on the various novel therapeutic options that have been investigated and are currently under development for the treatment of PSC.</p><p><strong>Expert opinion: </strong>In the next decade, more than one drug will likely be approved for the treatment of the disease, and we will be looking at combination therapies for the optimal management of the disease.</p>","PeriodicalId":12313,"journal":{"name":"Expert opinion on investigational drugs","volume":null,"pages":null},"PeriodicalIF":6.1,"publicationDate":"2024-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140119224","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}
{"title":"Investigational new drugs for the treatment of chronic renal failure: an overview of the literature.","authors":"Chiara Terzo, Guido Gembillo, Valeria Cernaro, Elisa Longhitano, Vincenzo Calabrese, Chiara Casuscelli, Luigi Peritore, Domenico Santoro","doi":"10.1080/13543784.2024.2326624","DOIUrl":"10.1080/13543784.2024.2326624","url":null,"abstract":"<p><strong>Introduction: </strong>Chronic kidney disease (CKD) is widespread throughout the world, with a high social and health impact. It is considered a 'silent killer' for its sudden onset without symptoms in the early stages of the disease. The main goal of nephrologists is to slow the progression of kidney disease and treat the associated symptoms with a range of new medications.</p><p><strong>Areas covered: </strong>The aim of this systematic review is to analyze the new investigational drugs for the treatment of chronic renal failure. Data were obtained from the available scientific literature and from the ClinicalTrials.gov website.</p><p><strong>Expert opinion: </strong>Among the drugs currently being researched, SGLT2 inhibitors appear to be the most promising drugs for the treatment of CKD, has they have slower progression of CKD and protection of cardiorenal function. An important role in the future of CKD treatment is played by autologous cell-therapy, which appears to be a new frontier in the treatment of CKD. Other therapeutic strategies are currently being investigated and have been shown to slow the progression of CKD. However, further studies are needed to determine whether these approaches may offer benefits in slowing the progression of CKD in the near future.</p>","PeriodicalId":12313,"journal":{"name":"Expert opinion on investigational drugs","volume":null,"pages":null},"PeriodicalIF":6.1,"publicationDate":"2024-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140012533","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}