{"title":"Resiquimod: a new topical immune-response modifier for the treatment of actinic keratosis.","authors":"Morgan A Farr, Tejas P Joshi, Daniel J Lewis","doi":"10.1080/14728214.2021.2004694","DOIUrl":"https://doi.org/10.1080/14728214.2021.2004694","url":null,"abstract":"<p><p>Resiquimod is a Toll-like receptor (TLR)-7 and TLR-8 agonist that, like imiquimod, belongs to the class of imidazoquinolines, small organic molecules with potent antiviral and anticancer activity. Resiquimod activates myeloid and plasmacytoid dendritic cells while also promoting release of cytokines as interleukin-6, tumor necrosis factor-α and interferon-γ more effectively than imiquimod. Given these immunologic effects, resiquimod is emerging as a new topical pharmacotherapy for actinic keratosis (AK). In the pivotal trial by Stockfleth et al. complete clinical clearance in all the resiquimod-treated arms was more significant than placebo varying from 56% to 74%. Partial clinical clearance, or disappearance of >75% of AKs, was also significantly higher in the resiquimod arms varying from 75% to 87%. Overall, resiquimod is a new molecule that remains a promising therapy for AK, although additional studies are needed to further evaluate its efficacy.</p>","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":"26 4","pages":"433-434"},"PeriodicalIF":3.4,"publicationDate":"2021-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39601255","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}
{"title":"Emerging therapies for Achondroplasia: changing the rules of the game.","authors":"Smitha Kumble, Ravi Savarirayan","doi":"10.1080/14728214.2021.2005577","DOIUrl":"https://doi.org/10.1080/14728214.2021.2005577","url":null,"abstract":"<p><strong>Introduction: </strong>Achondroplasia is the most common genetic cause of disproportionate short stature, affecting over 360,000 individuals. Serious complications contributing to significant morbidity in affected individuals include cranio-cervical junction compression and obstructive sleep apnea. Current clinically available treatments are predominantly symptomatic and associated with variable outcomes. We summarize the new precision investigational products that are currently in Phase 2 and Phase 3 clinical trials for the treatment of individuals with achondroplasia.</p><p><strong>Areas covered: </strong>Fibroblast growth factor receptor 3 (<i>FGFR3</i>), a membrane-spanning tyrosine kinase receptor, binds various fibroblast growth factors (FGF) to regulate the normal process of endochondral bone growth. Gain of <i>FGFR3</i> function in individuals with achondroplasia results in inhibition of normal endochondral ossification. A greater understanding of these molecular pathways through animal models has led to the development of several targeted therapies being tested in children, which we discuss in this review.</p><p><strong>Expert opinion: </strong>The last decade has been game-changing in terms of new precision therapies for children with achondroplasia that have the potential to fundamentally change the natural history of this condition. The next decade will see how these therapies compare, if they might be used in combination, and evaluate the balance of their long-term benefits and harms.</p>","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":"26 4","pages":"425-431"},"PeriodicalIF":3.4,"publicationDate":"2021-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39696930","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}
{"title":"An update on emerging drugs for the treatment of hypercholesterolemia.","authors":"Adam J Nelson, Kristen Bubb, Stephen J Nicholls","doi":"10.1080/14728214.2021.2009801","DOIUrl":"https://doi.org/10.1080/14728214.2021.2009801","url":null,"abstract":"<p><strong>Introduction: </strong>Elevated levels of low-density lipoprotein (LDL) cholesterol have been unequivocally demonstrated to play a causal role in atherosclerotic cardiovascular disease. The last thirty years have witnessed a generation of clinical trials that have demonstrated a reduction in cardiovascular risk with the use of increasing intensive lipid lowering regimens involving statin therapy in combination with other agents. However, many patients fail to achieve treatment mandated LDL cholesterol goals. This highlights the need to develop additional approaches to lower LDL cholesterol levels.</p><p><strong>Areas covered: </strong>(i) Contemporary data highlighting the atherogenicity of LDL cholesterol and cardiovascular benefits of current lipid lowering therapies. (ii) Importance of statin intolerance and inability to achieve LDL cholesterol goals in driving ongoing cardiovascular risk. (iii) Emergence of new therapeutic agents designed to achieve more effective lowering of LDL cholesterol.</p><p><strong>Expert opinion: </strong>Effective lowering of LDL cholesterol plays a critical role in approaches to the prevention of cardiovascular disease. A greater number of patients will require combinations of agents to achieve optimal lipid control. Accordingly, new agents will be required to provide sufficient choice for patients at high cardiovascular risk.</p>","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":"26 4","pages":"363-369"},"PeriodicalIF":3.4,"publicationDate":"2021-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39942558","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}
{"title":"Emerging drugs for the treatment of postsurgical pain.","authors":"Esra Kutlu Yalcin, Jorge Araujo-Duran, Alparslan Turan","doi":"10.1080/14728214.2021.2009799","DOIUrl":"https://doi.org/10.1080/14728214.2021.2009799","url":null,"abstract":"<p><strong>Introduction: </strong>Postoperative pain is a distressful experience and remains to be a significant concern after surgery. Current agents either fail to prevent or minimize postoperative pain or cause a series of adverse effects, addiction, or abuse. Opioids have been the gold standard in the treatment of postoperative pain despite their well-described adverse effects. Many new agents with different mechanisms of action have been recently introduced to address this issue.</p><p><strong>Areas covered: </strong>This current review summarizes the list of new and emerging drugs investigated for their efficacy in controlling the postoperative pain and decreasing the need for rescue opioid use, adverse effect profile, abuse, and addiction potential.</p><p><strong>Expert opinion: </strong>Opioids have unrivaled analgesic efficacy. However adverse effects of opioids led to the search for better options. In mild pain most of the emerging drugs have been shown to control postoperative pain and decrease the use of rescue opioid, however fail to control pain after major surgeries causing severe pain. Specific agents such as Oliceridine, new local anesthetics, etc., are effective in controlling severe pain and hold a promise to replace opioids in the near future.</p>","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":"26 4","pages":"371-384"},"PeriodicalIF":3.4,"publicationDate":"2021-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39674960","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}
Lauren Mason, Saad Jafri, Isabelle Dortonne, John D Sheppard
{"title":"Emerging therapies for dry eye disease.","authors":"Lauren Mason, Saad Jafri, Isabelle Dortonne, John D Sheppard","doi":"10.1080/14728214.2021.2011858","DOIUrl":"https://doi.org/10.1080/14728214.2021.2011858","url":null,"abstract":"<p><strong>Introduction: </strong>Dry Eye Disease (DED) is defined as a multifactorial disease of the ocular surface characterized by a loss of homeostasis of the tear film, and a vicious cycle of inflammation on the ocular surface. Despite its high prevalence and standing as one of the most common eye conditions seen by practitioners, the current treatment options available to patients have not proven adequate.</p><p><strong>Areas covered: </strong>This review will discuss the burden of DED, its pathophysiology, as well as emerging therapies. These therapies include immunosuppressants, immunomodulators, anti-inflammatory drugs, and corticosteroids. The mechanisms of these drugs will be discussed, as well as their phase of development and results from recent clinical trials. The literature search was performed using PubMed, Cochrane Library, Web of Science, ClinicalTrials.gov, and the Springer AdisInsight database.</p><p><strong>Expert opinion: </strong>The optimal therapy for DED is associated with improved bioavailability, minimal ocular side effects, and effective dosing. The ideal treatment has not yet been established, but this paper outlines a number of promising therapies. Continued development of therapies targeting the inflammation cascade, as well as the establishment of objective markers to quantify DED severity, are important aspects in the progression of treatment.</p>","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":"26 4","pages":"401-413"},"PeriodicalIF":3.4,"publicationDate":"2021-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39678717","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}
{"title":"Emerging therapies for AML with myelodysplasia-related changes: slowly but surely moving the needle.","authors":"Davis F Phillips, Joshua F Zeidner","doi":"10.1080/14728214.2021.1950689","DOIUrl":"https://doi.org/10.1080/14728214.2021.1950689","url":null,"abstract":"<p><p><b>Introduction</b>: Patients with acute myeloid leukemia with myelodysplasia-related changes (AML-MRC) have historically poor outcomes with conventional chemotherapy regimens. Current treatment strategies focus on intensive induction therapy followed by allogeneic stem cell transplant or a less intensive approach with hypomethylating agents with or without venetoclax. CPX-351 is a liposomal formulation of cytarabine and daunorubicin that has been shown to significantly improve response rates and survival compared with 7 + 3 (continuous infusion cytarabine plus anthracyclines). Despite the approval of CPX-351 for AML-MRC, overall prognosis remains poor with an unmet need to develop novel therapeutic strategies for this patient population.<b>Areas covered</b>: This article reviews the data for existing therapeutic options for patients with AML-MRC and the emerging therapies undergoing clinical trial development for this patient population.<b>Expert opinion</b>: The development of CPX-351 as a more effective induction therapeutic backbone for patients with AML-MRC presents an opportunity to investigate novel combination regimens in order to further improve outcomes. Promising emerging therapeutic modalities include immunotherapeutic strategies, small-molecule inhibitors and targeted agents. Unfortunately, there have been few clinical trials focusing on patients with AML-MRC with reliance instead on subgroup analyses. Clinical trials focused specifically on this patient population are urgently needed.</p>","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":"26 3","pages":"245-257"},"PeriodicalIF":3.4,"publicationDate":"2021-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/14728214.2021.1950689","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39154565","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}
{"title":"Emerging drugs for the prevention of migraine.","authors":"Oyindamola Ogunlaja, Nazia Karsan, Peter Goadsby","doi":"10.1080/14728214.2021.1956463","DOIUrl":"https://doi.org/10.1080/14728214.2021.1956463","url":null,"abstract":"<p><strong>Introduction: </strong>Migraine is a common and disabling neurological disorder. A greater understanding of the pathophysiological mechanisms underlying migraine has led to the availability of specific new drugs targeting calcitonin gene-related peptide (CGRP). The success of the CGRP inhibitors validates research efforts into migraine-specific therapies.</p><p><strong>Areas covered: </strong>There are additional promising therapeutic targets that will be covered in this paper, focusing on the pain phase. They include pituitary adenylate cyclase-activating polypeptide (PACAP), the orexinergic system, the nitric oxide signaling pathway specifically neuronal nitric oxide synthase inhibitors (nNOSi), and metabotropic glutamate receptor 5 (mGluR5).</p><p><strong>Expert opinion: </strong>Based on currently available research; the targets discussed in this paper are all on equal footing with each other in terms of their potential as effective novel migraine therapies. There is a need for more clinical trials to pinpoint which of these potential drug targets will be effective for migraine preventio.</p>","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":"26 3","pages":"271-280"},"PeriodicalIF":3.4,"publicationDate":"2021-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/14728214.2021.1956463","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39185706","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}
{"title":"Emerging glucagon-like peptide 1 receptor agonists for the treatment of obesity.","authors":"Mathies M Jepsen, Mikkel B Christensen","doi":"10.1080/14728214.2021.1947240","DOIUrl":"https://doi.org/10.1080/14728214.2021.1947240","url":null,"abstract":"<p><p><b>Introduction</b>: Obesity is a growing threat to public health, increasing risks of numerous diseases and mortality, and impairing quality of life. If current trends continue, more than 1.1 billion individuals will have obesity in 2030, corresponding to almost 2.5 times the number of adults currently living with diabetes. There is a strong interest in developing obesity treatments based on glucagon-like peptide-1 (GLP-1) agonism, which have proved to limit morbidity and mortality in type 2 diabetes.<b>Areas covered</b>: This review provides an overview of current compounds containing GLP-1 receptor agonism in clinical development for obesity, with mono-activity at the GLP-1 receptor (PF-0688296, glutazumab, semaglutide) or engaging one or more other endogenous hormonal systems involved in energy balance and metabolism, including glucagon, oxyntomodulin, glucose-dependent inhibitory peptide and amylin (CT-868, CT-388, AMG 133, tirzepatide, NNC9204-1177, JNJ-54,728,518, SAR425899, pegapamodutide, MK8521, cotadutide, efinopegdutide, BI-456,906, cagrilintide + semaglutide 2,4 mg, HM15211, NNC9204-1706).<b>Expert opinion</b>: Many novel compounds employing GLP-1 receptor agonism are in clinical development. Semaglutide is farthest in clinical development and will presumably become a benchmark for this class of novel anti-obesity compounds.</p>","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":"26 3","pages":"231-243"},"PeriodicalIF":3.4,"publicationDate":"2021-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/14728214.2021.1947240","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39111214","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}
{"title":"The future of antibody therapy in chronic lymphocytic leukemia.","authors":"Jennifer L Crombie, Jennifer R Brown","doi":"10.1080/14728214.2021.1966414","DOIUrl":"https://doi.org/10.1080/14728214.2021.1966414","url":null,"abstract":"<p><strong>Introduction: </strong>Outcomes in chronic lymphocytic leukemia (CLL) have been dramatically improved with the addition of anti-CD20 antibodies to chemotherapy, defining a new standard of care for many years. More recently, therapies targeting fundamental signaling and anti-apoptotic pathways within the CLL cell have demonstrated dramatic clinical responses, including in patients with high-risk prognostic markers, thus emerging as preferred therapy for many patients. While the addition of anti-CD20 antibodies to traditional chemotherapy resulted in significant improvements in outcomes, the role of monoclonal antibodies in the era of targeted agents remains an active area of investigation. Furthermore, since the advent of next-generation anti-CD20 antibodies, the role of specific anti-CD20 antibodies remains an open question.</p><p><strong>Areas covered: </strong>In this review, we highlight the important role that anti-CD20 antibody therapy has had in the field of CLL, both when used with chemotherapy and in combination with targeted therapy, as well as the current studies that are further exploring this treatment paradigm in the modern era.</p><p><strong>Expert opinion: </strong>While anti-CD20 antibodies have played a pivotal role in the treatment of CLL, additional studies will be required to determine the optimal application of these therapies in combination with targeted therapy.</p>","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":"26 3","pages":"323-336"},"PeriodicalIF":3.4,"publicationDate":"2021-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39307845","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}
{"title":"Emerging therapeutics in Huntington's disease.","authors":"Robert Wiggins, Andrew Feigin","doi":"10.1080/14728214.2021.1962285","DOIUrl":"https://doi.org/10.1080/14728214.2021.1962285","url":null,"abstract":"<p><strong>Introduction: </strong>Huntington's disease is a neurodegenerative disease that is characterized by motor dysfunction, behavioral/psychiatric symptoms, and cognitive impairment. Because of the lack of availability of curative or disease modifying treatments, much of clinical practice in HD care to date has focused on symptomatic treatment. Recent work has created optimism surrounding possible emerging disease modifying therapeutics. HD is a developing therapeutic field with diverse and promising emerging therapies.</p><p><strong>Areas covered: </strong>A PubMed literature review was completed to discover pertinent reviews and analyses. ClinicalTrials.gov was referenced to find updated information about ongoing and planned trials. Lastly, because of the rapidly evolving nature of HD treatments, drug manufacturer websites and press releases were reviewed to provide current information surrounding recently reported trial results.</p><p><strong>Expert opinion: </strong>Recent setbacks involving antisense oligonucleotide research should not diminish enthusiasm and hope for the many other novel therapies currently being pursued. We remain optimistic about the many promising emerging therapies for HD, and we expect that growing knowledge about the pathophysiology of the underlying disease and constant advances in biotechnology will lead to therapies that have a meaningful impact in the lives of patients, their families, and those who care for them.</p>","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":"26 3","pages":"295-302"},"PeriodicalIF":3.4,"publicationDate":"2021-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/14728214.2021.1962285","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39230740","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}