Jordan R Hansford, Neevika Manoharan, Gauthier Bouche, Vijay Ramaswamy, Nada Jabado, Eric Bouffet
{"title":"ONC201 (Dordaviprone): review of evidence to date in diffuse midline glioma, hope or hype?","authors":"Jordan R Hansford, Neevika Manoharan, Gauthier Bouche, Vijay Ramaswamy, Nada Jabado, Eric Bouffet","doi":"10.1080/14728214.2024.2426649","DOIUrl":"10.1080/14728214.2024.2426649","url":null,"abstract":"","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"1-5"},"PeriodicalIF":2.7,"publicationDate":"2024-11-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142643379","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}
Leonardo Stella, Clemence Hollande, Yasmina Ben Merabet, Hugo Fakhouri, Vincent Leclerc, Francesca Romana Ponziani, Mohamed Bouattour
{"title":"Promising PD-1 antagonists for liver cancer: an evaluation of phase II and III results.","authors":"Leonardo Stella, Clemence Hollande, Yasmina Ben Merabet, Hugo Fakhouri, Vincent Leclerc, Francesca Romana Ponziani, Mohamed Bouattour","doi":"10.1080/14728214.2024.2430493","DOIUrl":"https://doi.org/10.1080/14728214.2024.2430493","url":null,"abstract":"<p><strong>Introduction: </strong>Hepatocellular carcinoma (HCC) is the most common primary liver cancer and a significant cause of cancer-related morbidity and mortality. Limited treatment options for advanced stages emphasize the need for effective therapies.</p><p><strong>Areas covered: </strong>This review examines immune checkpoint inhibitors (ICIs), specifically PD-1, PD-L1, and CTLA-4 inhibitors, as novel treatments for advanced HCC. The review includes data from phase II and III clinical trials that evaluate ICI combinations with tyrosine kinase inhibitors (TKIs) and anti-angiogenic agents, as well as locoregional treatments such as Transarterial Chemoembolization (TACE). Clinical outcomes, including progression-free survival and overall response rates, were analyzed along with the incidence and management of immune-related adverse events (irAEs). A systematic literature review method was applied to ensure the inclusion of comprehensive, high-quality studies.</p><p><strong>Expert opinion: </strong>ICI-based therapies and their combinations are reshaping the treatment landscape for advanced HCC by providing improved outcomes and potentially extending patient survival. Although promising, these therapies require further refinement to optimize sequencing and treatment selection, especially for patients with different levels of liver function and disease stages. Managing adverse effects effectively is essential for maximizing the benefits of these therapies in clinical practice. Continued research is needed to support the development of personalized approaches that can better tailor these treatments to individual patient needs, ultimately enhancing the overall effectiveness and safety of HCC management.</p>","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":""},"PeriodicalIF":2.7,"publicationDate":"2024-11-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142643385","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}
Heli A Patel, Linh Tran, Ralina Karagenova, Steven R Feldman
{"title":"Monoclonal antibodies in phase II and III trials for moderate to severe atopic dermatitis.","authors":"Heli A Patel, Linh Tran, Ralina Karagenova, Steven R Feldman","doi":"10.1080/14728214.2024.2416114","DOIUrl":"10.1080/14728214.2024.2416114","url":null,"abstract":"<p><strong>Introduction: </strong>Atopic dermatitis (AD) is a prevalent chronic inflammatory skin disease that significantly affects quality of life and mental health, especially in children. Traditional treatments include chemotherapeutics, topical corticosteroids, and immunomodulatory agents, but recent advances have introduced novel monoclonal antibody therapies. Through this comprehensive review paper, we aim to discuss these therapeutic options and their role in treating atopic dermatitis.</p><p><strong>Areas covered: </strong>A comprehensive search of the NIH Clinical Trials database was conducted from September 2023 to January 2024, focusing on phase 2 and 3 trials for AD treatments. Trials were filtered using keywords such as 'atopic dermatitis,' 'monoclonal antibody,' and 'phase 2/3.' Out of 25 trials analyzed, 11 were in phase 2 and 14 in phase 3. Only U.S.-based trials comparing novel therapies to placebo were included. In addition to the clinical trial database, we utilized the companies' websites and relevant abstracts to gather the latest results.</p><p><strong>Expert opinion: </strong>Currently investigated monoclonal antibodies have the ability to transform management by targeting specific mediators implicated in the inflammatory pathway of AD. The results of Phase II and III trials for monoclonal antibodies demonstrated strong therapeutic potential with significant reductions in EASI scores and represent a promising new targeted treatment option.</p>","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"1-8"},"PeriodicalIF":2.7,"publicationDate":"2024-10-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142399909","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 immunotherapies in the Hodgkin lymphoma armamentarium.","authors":"Michael A Spinner, Ranjana H Advani","doi":"10.1080/14728214.2024.2349083","DOIUrl":"10.1080/14728214.2024.2349083","url":null,"abstract":"<p><strong>Introduction: </strong>Brentuximab vedotin and PD-1 inhibitors have improved outcomes for classic Hodgkin lymphoma (cHL), but better therapies are needed for patients who relapse after these agents. Based on an improved understanding of cHL biology, there is a robust pipeline of novel therapies in development. In this review, we highlight emerging immunotherapeutic agents and combinations for cHL.</p><p><strong>Areas covered: </strong>We review clinical trials of novel PD-1/PD-L1 inhibitors beyond FDA-approved agents, checkpoint inhibitors targeting CTLA-4, LAG-3, TIM-3, TIGIT, and CD47/SIRPα, PD-1 inhibitor combinations with immunomodulatory agents and epigenetic modifying therapies, antibody-drug conjugates, bispecific antibodies, and cellular therapies including anti-CD30 CAR-T and allogeneic NK cell therapy. We review the key safety and efficacy data from published phase 1-2 studies and highlight trials in progress, including the first phase 3 trial for PD-1 inhibitor-refractory cHL.</p><p><strong>Expert opinion: </strong>Many novel immunotherapies hold great promise in cHL. Rational combinations with existing agents and next-generation antibody and CAR-T constructs may improve response rates and durability. Identifying biomarkers of response to these immunotherapies and using more sensitive tools to assess response, such as circulating tumor DNA, may further inform treatment decisions and enable a precision medicine approach in the future.</p>","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"263-275"},"PeriodicalIF":2.7,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140862631","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}
Hui Jue Wang, Arun Chinna-Meyyappan, Oriel J Feldman, Krista L Lanctôt
{"title":"Emerging therapies for treatment of agitation, psychosis, or apathy in Alzheimer's disease.","authors":"Hui Jue Wang, Arun Chinna-Meyyappan, Oriel J Feldman, Krista L Lanctôt","doi":"10.1080/14728214.2024.2363215","DOIUrl":"10.1080/14728214.2024.2363215","url":null,"abstract":"<p><strong>Introduction: </strong>Agitation, psychosis, and apathy are prevalent and highly distressing neuropsychiatric symptoms (NPS) of Alzheimer's disease (AD) that have been linked to numerous negative outcomes, including increased mortality, worsened cognitive decline, and caregiver burden. Current treatments for AD-associated agitation, namely atypical antipsychotics, provide some benefits but may increase the risk of serious adverse events and death. Meanwhile, no pharmacotherapies have been approved by regulatory agencies for the treatment of psychosis or apathy in AD. Over the past decade, many new and repurposed drugs have emerged as potential therapeutic options for managing these challenging NPS.</p><p><strong>Areas covered: </strong>This review aims to provide a comprehensive summary of pharmacotherapies that have recently been investigated in phase 2 and 3 clinical trials for the treatment of agitation, psychosis, or apathy in AD.</p><p><strong>Expert opinion: </strong>Novel atypical antipsychotics, serotonergic antidepressants, cannabinoids, and dextromethorphan combination drugs have shown promising results for alleviating agitation. Pimavanserin appears to be the most effective emerging therapy for psychosis, while methylphenidate has demonstrated good efficacy for apathy. Further research on biomarkers of NPS severity and treatment response, as well as continued improvements in methodological approaches are needed to advance the field.</p>","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"289-303"},"PeriodicalIF":2.7,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141186324","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}
Tristan Frau, Myriam El Khatib, Brune De Dreuille, Lore Billiauws, Alexandre Nuzzo, Francisca Joly
{"title":"Emerging drugs for the treatment of short bowel syndrome.","authors":"Tristan Frau, Myriam El Khatib, Brune De Dreuille, Lore Billiauws, Alexandre Nuzzo, Francisca Joly","doi":"10.1080/14728214.2024.2357567","DOIUrl":"10.1080/14728214.2024.2357567","url":null,"abstract":"<p><strong>Introduction: </strong>SBS is a rare and disabling condition. The standard management is based on diet optimization with parenteral supplementation. In addition, glucagon-like peptide-2 (GLP-2)analogs, have shown promising results as disease-modifying therapies for SBS.</p><p><strong>Areas covered: </strong>Short bowel syndrome (SBS) is defined as a reduction in functional intestinal length to less than 200 cm, leading to intestinal failure (IF) leading to malnutrition and parenteral support dependency. This review discusses the current management of SBS-CIFpatients, the place of GLP-2 analog treatment in terms of efficacy, safety and availability, and the new perspectives opened by the use of enterohormones.</p><p><strong>Expert opinion: </strong>Clinical trials and real-world experience demonstrated that Teduglutide reduces dependence on parenteral support and has a place in the management of patients with SBS-CIF. The use of Teduglutide should be discussed in patients stabilized after resection and its introduction requires the advice of an expert center capable of assessing the benefit-risk ratio. The complex, individualized management of SBS-C IF requires theexpertise of a specialized IF center which a multidisciplinary approach. The arrival of new treatments will call for new therapeutic strategies, and the question of how to introduce and monitor them will represent a new therapeutic challenge.</p>","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"277-288"},"PeriodicalIF":2.7,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140957158","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}
Joshua Winograd, Nikit Venishetty, Alia Codelia-Anjum, Naeem Bhojani, Dean Elterman, Kevin C Zorn, Alexis Te, Bilal Chughtai
{"title":"Emerging drugs for the treatment of benign prostatic hyperplasia: a 2023 update.","authors":"Joshua Winograd, Nikit Venishetty, Alia Codelia-Anjum, Naeem Bhojani, Dean Elterman, Kevin C Zorn, Alexis Te, Bilal Chughtai","doi":"10.1080/14728214.2024.2363213","DOIUrl":"10.1080/14728214.2024.2363213","url":null,"abstract":"<p><strong>Introduction: </strong>Benign prostatic hyperplasia (BPH) is a condition that affects over 50% of men as they enter their fifth decade of life, often leading to lower urinary tract symptoms (LUTS). Primary treatment options include alpha blockers, 5-alpha reductase inhibitors, and phosphodiesterase-5 inhibitors. However, these medications can have some side effects, and there is a noticeable dearth of information addressing the long-term use of these medications. Thus, the exploration of all treatment modalities helps ensure patients receive personalized and effective care. Consequently, the primary objective of this review is to identify potential emerging medications for the treatment of BPH.</p><p><strong>Areas covered: </strong>We conducted an extensive review of articles discussing pharmacotherapy for BPH spanning the last 15 years. Our information gathering process involved Scopus, PubMed-MEDLINE, Cochrane, Wiley Online Library Google Scholar, ClinicalTrials.gov, and the PharmaProjects database. This approach ensures that readers gain an in-depth knowledge of the existing therapeutic agents as well as promising avenues for managing BPH.</p><p><strong>Expert opinion: </strong>BPH treatment targets a patient's specific constellation of symptoms. Therefore, a broad knowledge base encompassing various treatment options is paramount in ensuring optimal treatment. Looking forward, the emphasis on personalization promises to reshape the landscape of BPH treatment and improve patient outcomes.</p>","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"205-217"},"PeriodicalIF":2.7,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141261337","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}
Liyang Pan, Lydia Nagib, Sujata Ganguly, Arumugam Moorthy, Hasan Tahir
{"title":"A comprehensive review of phase 2/3 trials in osteoarthritis: an expert opinion.","authors":"Liyang Pan, Lydia Nagib, Sujata Ganguly, Arumugam Moorthy, Hasan Tahir","doi":"10.1080/14728214.2024.2386174","DOIUrl":"10.1080/14728214.2024.2386174","url":null,"abstract":"<p><strong>Introduction: </strong>Osteoarthritis (OA) is a chronic, degenerative, and debilitating disease associated with significant long-term morbidity and disability. The pathogenesis of OA is not completely understood but involves an interplay between environmental risk factors, joint mechanics, abnormal pain pathways and upregulation of inflammatory signaling pathways. Current therapeutic options for patients are limited to conservative management, minimal pharmacological options or surgical management, with significant caveats to all approaches.</p><p><strong>Areas covered: </strong>In this review, we have set out to investigate current phase II/III clinical trials by undertaking a PubMed search. Examined clinical trials have explored a myriad of potential therapeutics from conventional disease-modifying anti-rheumatic drugs and biologics usually used in the treatment of inflammatory arthritides, to more novel approaches targeting inflammatory pathways implicated in OA, cartilage degeneration or pain pathways.</p><p><strong>Expert opinion: </strong>Unfortunately, most completed phase II/III clinical trials have shown little impact on patient pain scores, with the exception of the traditional DMARD methotrexate and Sprifermin. Methotrexate has been shown to be beneficial when used in the correct patient cohort (MRI proven synovitis). Sprifermin has the longest follow-up data of 5 years and has been shown to reduce loss of MRI-measured cartilage thickness and pain scores.</p>","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"1-13"},"PeriodicalIF":2.7,"publicationDate":"2024-08-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141859482","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":"Current and emerging drug treatment strategies to tackle sickle cell anemia.","authors":"Yogindra Persaud, Alexis Leonard, Parul Rai","doi":"10.1080/14728214.2024.2379260","DOIUrl":"10.1080/14728214.2024.2379260","url":null,"abstract":"<p><strong>Introduction: </strong>Since its discovery in the early 1900s, sickle cell disease (SCD) has contributed significantly to the scientific understanding of hemoglobin and hemoglobinopathies. Despite this, now almost a century later, optimal medical management and even curative options remain limited. Encouragingly, in the last decade, there has been a push toward advancing the care for individuals with SCD and a diversifying interest in options to manage this disorder.</p><p><strong>Areas covered: </strong>Here, we review the current state of disease modifying therapies for SCD including fetal hemoglobin inducers, monoclonal antibodies, anti-inflammatory modulators, and enzyme activators. We also discuss current curative strategies with specific interest in transformative gene therapies.</p><p><strong>Expert opinion: </strong>SCD is a chronic, progressive disease that despite a century of clinical description, only now is seeing a growth and advance in therapeutic options to improve the lifespan and quality of life for individuals with SCD. We anticipate newly designed and even repurposed therapies that may work as a single agent or combination agents to tackle the progression of SCD. The vast majority of individuals living with SCD are unlikely to receive gene therapy, therefore improved disease management is critical even for those that may ultimately chose to pursue a potentially curative strategy.</p>","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"1-20"},"PeriodicalIF":2.7,"publicationDate":"2024-07-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141579372","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}
William C Upshaw, Lenise G Soileau, Nicholas R Storey, Kassady A Perkinson, Patrick M Luther, Noah J Spillers, Christopher L Robinson, Benjamin C Miller, Shahab Ahmadzadeh, Omar Viswanath, Sahar Shekoohi, Alan D Kaye
{"title":"An extract of phase II and III trials on recent developments in managing neuropathic pain syndromes: diabetic peripheral neuropathy, trigeminal neuralgia, and postherpetic neuralgia.","authors":"William C Upshaw, Lenise G Soileau, Nicholas R Storey, Kassady A Perkinson, Patrick M Luther, Noah J Spillers, Christopher L Robinson, Benjamin C Miller, Shahab Ahmadzadeh, Omar Viswanath, Sahar Shekoohi, Alan D Kaye","doi":"10.1080/14728214.2024.2323193","DOIUrl":"10.1080/14728214.2024.2323193","url":null,"abstract":"<p><strong>Introduction: </strong>Neuropathic pain (NP) conditions involve lesions to the somatosensory nervous system leading to chronic and debilitating pain. Many patients suffering from NP utilize pharmacological treatments with various drugs that seek to reduce pathologic neuronal states. However, many of these drugs show poor efficacy as well as cause significant adverse effects. Because of this, there is a major need for the development of safer and more efficacious drugs to treat NP.</p><p><strong>Areas covered: </strong>In this review, we analyzed current treatments being developed for a variety of NP conditions. Specifically, we sought drugs in phase II/III clinical trials with indications for NP conditions. Various databases were searched including Google Scholar, PubMed, and clinicaltrials.gov.</p><p><strong>Expert opinion: </strong>All the mentioned targets for treatments of NP seem to be promising alternatives for existing treatments that often possess poor side effect profiles for patients. However, gene therapy potentially offers the unique ability to inject a plasmid containing growth factors leading to nerve growth and repair. Because of this, gene therapy appears to be the most intriguing new treatment for NP.</p>","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"103-112"},"PeriodicalIF":2.7,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139971526","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}