Expert opinion on investigational drugs最新文献_第4页

SOT201: cis-acting PD-1/IL-15 mutein-based immunocytokine under investigational study in solid tumors. SOT201：基于顺式作用PD-1/IL-15蛋白的免疫细胞因子在实体瘤中的研究

IF 4.3 2区医学

Expert opinion on investigational drugs Pub Date : 2026-06-01 Epub Date: 2026-06-03 DOI: 10.1080/13543784.2026.2683841

Vladyslav Mazhara, Irfan Baki Kilic, Irena Adkins, Radek Spisek, Marek Kovar

{"title":"SOT201: cis-acting PD-1/IL-15 mutein-based immunocytokine under investigational study in solid tumors.","authors":"Vladyslav Mazhara, Irfan Baki Kilic, Irena Adkins, Radek Spisek, Marek Kovar","doi":"10.1080/13543784.2026.2683841","DOIUrl":"10.1080/13543784.2026.2683841","url":null,"abstract":"Introduction: The systemic administration of cytokines is constrained by their pleiotropic activity, dose-dependent toxicities, and short serum half-life, limiting their therapeutic window in cancer treatment. To overcome these challenges, strategies that restrict cytokine signaling to defined immune cell subsets within the tumor microenvironment have been developed to enhance efficacy while minimizing off-target effects. Among these, antibody-cytokine fusion proteins represent a rational design platform that enables selective and localized cytokine delivery to specific immune populations.Areas covered: This report outlines the design principles underlying tumor-associated antigen-targeted and cis-delivered IL-2- and IL-15-based immunocytokine platforms, with particular emphasis on the PD-1-directed cis-signaling strategy. Preclinical data on SOT201 are summarized, highlighting how its affinity optimized IL-15 mutein promote selective proliferation and enhanced effector function of PD-1+ CD8+ T cells.Expert opinion: Cis-acting immunocytokines represent a promising class of advanced therapeutics that selectively direct cytokine payload to tumor infiltrating lymphocytes. This strategy has been shown to induce durable antitumor immunity and, in some cases, promote immune memory formation while limiting systemic toxicity. Ongoing clinical evaluation and rational combination approaches will ultimately define its therapeutic positioning in precision cancer immunotherapy.","PeriodicalId":12313,"journal":{"name":"Expert opinion on investigational drugs","volume":" ","pages":"393-400"},"PeriodicalIF":4.3,"publicationDate":"2026-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148137099","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

Evaluating the investigational drug landscape for ESR1-mutated, estrogen receptor-positive, HER2-negative metastatic breast cancer. 评估esr1突变、雌激素受体阳性、HER2阴性转移性乳腺癌的研究药物前景。

IF 4.3 2区医学

Expert opinion on investigational drugs Pub Date : 2026-06-01 Epub Date: 2026-06-16 DOI: 10.1080/13543784.2026.2686701

Marie Kroemer, Morgane Lopez, Laura Mansi, Elsa Curtit

{"title":"Evaluating the investigational drug landscape for ESR1-mutated, estrogen receptor-positive, HER2-negative metastatic breast cancer.","authors":"Marie Kroemer, Morgane Lopez, Laura Mansi, Elsa Curtit","doi":"10.1080/13543784.2026.2686701","DOIUrl":"10.1080/13543784.2026.2686701","url":null,"abstract":"Introduction: Estrogen receptor-positive, HER2-negative (ER+/HER2-) metastatic breast cancer represents a major therapeutic challenge due to the frequent development of endocrine resistance. This review focuses on ESR1 mutations as key drivers of resistance and examines the evolving therapeutic strategies designed to overcome this mechanism.Area covered: The authors discuss current standards of care in the first- and second-line settings, with a focus on molecularly guided therapeutic strategies. The literature was identified through a targeted search of PubMed and ClinicalTrials.gov, focusing on English-language publications from peer-reviewed journals with additional reference to current ESMO and ASCO clinical practice guidelines. Particular attention was given to emerging oral ER-targeting agents. Key clinical trial data supporting ctDNA-based monitoring of ESR1 mutations and treatment adaptation were also reviewed.Expert opinion: Routine assessment of ESR1 mutations using liquid biopsy should be integrated into clinical practice to enable dynamic, molecularly guided treatment optimization. The expanding arsenal of ER-targeted therapies supports personalized sequencing strategies that move beyond rigid temporal cutoffs and improve outcomes in ET‑resistant disease.","PeriodicalId":12313,"journal":{"name":"Expert opinion on investigational drugs","volume":" ","pages":"383-391"},"PeriodicalIF":4.3,"publicationDate":"2026-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148197887","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

Alpha-1 antitrypsin: therapeutic applications beyond serine protease inhibition. α -1抗胰蛋白酶：超越丝氨酸蛋白酶抑制的治疗应用。

IF 4.3 2区医学

Expert opinion on investigational drugs Pub Date : 2026-06-01 Epub Date: 2026-06-03 DOI: 10.1080/13543784.2026.2681588

Martina Toldo, Trushaben Patel, Michele Golino, Eugenio Refrigeri, Jazmin Kelly, Antonio Abbate, Eleonora Mezzaroma, Stefano Toldo

{"title":"Alpha-1 antitrypsin: therapeutic applications beyond serine protease inhibition.","authors":"Martina Toldo, Trushaben Patel, Michele Golino, Eugenio Refrigeri, Jazmin Kelly, Antonio Abbate, Eleonora Mezzaroma, Stefano Toldo","doi":"10.1080/13543784.2026.2681588","DOIUrl":"10.1080/13543784.2026.2681588","url":null,"abstract":"Introduction: Alpha-1 antitrypsin (AAT) is an acute-phase glycoprotein belonging to the serine protease inhibitor (SERPIN) superfamily. An advanced understanding of functional and post-translational modifications highlighted a broad range of AAT effects on inflammation, metabolism, and cell survival. AAT's SERPIN activity is important to inhibit overactivation of several endogenous proteases, a crucial function evidenced by the fact that mutant AAT isoforms can promote lung and liver disease. However, recent research highlighted that AAT's physiological and pathophysiological functions extend beyond SERPIN function. AAT isolated from healthy donors' plasma is approved for augmentation therapy and remains the only disease-modifying therapy approved for patients with severe AAT deficiency.Area covered: We searched PubMed and Google Scholar to review the current literature on AAT function, the pathophysiology of AAT deficiency, and the growing evidence of additional biological effects independent of protease inhibition.Expert opinion: Preclinical and clinical studies highlighted that AAT is more than a SERPIN. AAT can regulate cell function, inflammation, and response to injury. AAT showed a disease-modifying effect in cellular, animal, and human studies. In addition, novel recombinant isoforms or peptides that mimic some of AAT's properties, are the focus of preclinical and clinical research as an alternative to plasma-derived AAT.","PeriodicalId":12313,"journal":{"name":"Expert opinion on investigational drugs","volume":" ","pages":"413-427"},"PeriodicalIF":4.3,"publicationDate":"2026-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148028631","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

Darigabat and subtype-selective GABA-A modulation: pharmacology, clinical development, and translational challenges. 达利加巴特和亚型选择性GABA-A调节：药理学、临床发展和转化挑战。

IF 4.3 2区医学

Expert opinion on investigational drugs Pub Date : 2026-06-01 Epub Date: 2026-06-16 DOI: 10.1080/13543784.2026.2687030

Luigi Francesco Iannone, Marina Romozzi, Luca Pani, Fabio Tascedda

{"title":"Darigabat and subtype-selective GABA-A modulation: pharmacology, clinical development, and translational challenges.","authors":"Luigi Francesco Iannone, Marina Romozzi, Luca Pani, Fabio Tascedda","doi":"10.1080/13543784.2026.2687030","DOIUrl":"10.1080/13543784.2026.2687030","url":null,"abstract":"Introduction: Darigabat is a selective γ-aminobutyric acid type A (GABA-A) receptor positive allosteric modulator targeting α2, α3, and α5 subunits, developed to preserve anxiolytic and anticonvulsant effects while reducing α1-mediated sedation and cognitive adverse events. Subtype-selective modulation represents a strategy to improve the safety of GABAergic therapies.Areas covered: A literature search in PubMed, Embase, CENTRAL, and ClinicalTrials.gov up to June 2025 identified 20 preclinical and clinical studies. Darigabat demonstrated dose-proportional pharmacokinetics, high brain penetration, and overall acceptable tolerability, with dose-dependent central nervous system adverse effects at higher exposures. Preclinical models showed anticonvulsant and anxiolytic activity. However, clinical trials reported heterogeneous and inconsistent efficacy across indications.Expert opinion: Darigabat supports the feasibility of subtype-selective GABA-A modulation. Early-phase studies in healthy volunteers suggested improved acute tolerability compared with nonselective benzodiazepines. In contrast, repeated-dose studies in patients revealed dose-dependent central nervous system adverse effects, including somnolence, psychomotor slowing, and cognitive impairment, indicating that α1-sparing reduces but does not eliminate these effects. Current evidence does not support a clear therapeutic role in chronic central nervous system disorders. Future research should focus on exposure-response relationships, identification of responsive subgroups, and biomarker integration. The ability to retain efficacy with improved safety remains uncertain.","PeriodicalId":12313,"journal":{"name":"Expert opinion on investigational drugs","volume":" ","pages":"449-462"},"PeriodicalIF":4.3,"publicationDate":"2026-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148197899","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 impact of novel BET inhibitors on the treatment armamentarium of myelofibrosis. 新型bet抑制剂对骨髓纤维化治疗方案的影响。

IF 4.3 2区医学

Expert opinion on investigational drugs Pub Date : 2026-06-01 Epub Date: 2026-06-08 DOI: 10.1080/13543784.2026.2677662

Matteo Bellia, Clara Deambrogi, Abdurraouf Mokhtar Mahmoud, Andrea Patriarca

{"title":"The impact of novel BET inhibitors on the treatment armamentarium of myelofibrosis.","authors":"Matteo Bellia, Clara Deambrogi, Abdurraouf Mokhtar Mahmoud, Andrea Patriarca","doi":"10.1080/13543784.2026.2677662","DOIUrl":"10.1080/13543784.2026.2677662","url":null,"abstract":"Introduction: Myelofibrosis (MF) is a clonal myeloproliferative neoplasm driven by dysregulated JAK-STAT signaling and epigenetic alterations, characterized by chronic inflammation, marrow fibrosis, and limited disease-modifying treatment options. Bromodomain and extra-terminal (BET) proteins regulate transcriptional programs implicated in MF pathogenesis, making BET inhibition a rational therapeutic strategy.Areas covered: This review discusses the biological rationale for BET inhibition in MF and critically appraises clinical development from January 2015 to November 2025. We summarize preclinical evidence and clinical trial data of BET inhibitors, including pelabresib, INCB057643, BMS-986158, and other investigational agents, both as monotherapy and in combination with JAK inhibitors. The literature search included international clinical trial registries (ICTRP, Clinical trial.gov), databases, conference abstracts, and peer-reviewed publications, focusing on efficacy, safety, and potential disease-modifying effects.Expert opinion: BET inhibitors represent a promising investigational class with the potential to complement JAK inhibition by targeting inflammatory, fibrotic, and proliferative pathways. Pelabresib has demonstrated clinically meaningful activity in phase 2-3 trials, supporting its potential role in first-line combination therapy. However, hematologic toxicities, durability of responses, and the impact on survival and leukemic transformation remain unresolved. Biomarker-driven patient selection and rational combination strategies will be essential to optimize the clinical integration of BET inhibitors in MF.","PeriodicalId":12313,"journal":{"name":"Expert opinion on investigational drugs","volume":" ","pages":"401-412"},"PeriodicalIF":4.3,"publicationDate":"2026-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148175756","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, tolerability, and pharmacokinetics of HL-300, a novel topical pan-JAK inhibitor, in healthy Chinese adults: a first-in-human, randomized, double-blind, placebo-controlled phase I trial. 一种新型外用Pan-JAK抑制剂HL-300在中国健康成人中的安全性、耐受性和药代动力学：一项首次人体、随机、双盲、安慰剂对照的I期试验

IF 4.3 2区医学

Expert opinion on investigational drugs Pub Date : 2026-06-01 Epub Date: 2026-06-09 DOI: 10.1080/13543784.2026.2687028

Hui Qiu, Xingfei Zhang, Jie Huang, Lin Ye, Chan Zou, Jinlian Xie, Kunhong Deng, Qian Wu, Xiulin He, Congxin Liang, Yong Liu, Guang Yang, Guoping Yang, Chengxian Guo

{"title":"Safety, tolerability, and pharmacokinetics of HL-300, a novel topical pan-JAK inhibitor, in healthy Chinese adults: a first-in-human, randomized, double-blind, placebo-controlled phase I trial.","authors":"Hui Qiu, Xingfei Zhang, Jie Huang, Lin Ye, Chan Zou, Jinlian Xie, Kunhong Deng, Qian Wu, Xiulin He, Congxin Liang, Yong Liu, Guang Yang, Guoping Yang, Chengxian Guo","doi":"10.1080/13543784.2026.2687028","DOIUrl":"10.1080/13543784.2026.2687028","url":null,"abstract":"Introduction: HL-300 is a novel topical pan-JAK inhibitor targeting JAK1, JAK2, JAK3, and TYK2 for atopic dermatitis (AD). This first-in-human study evaluated its safety, tolerability, and pharmacokinetics (PK) in healthy Chinese adults.Methods: In a randomized, double-blind, placebo-controlled phase I trial, 36 subjects received single or multiple topical doses of HL-300 (0.25%, 0.5%, or 1.0%) or placebo. Safety and PK parameters were assessed.Results: Following single dosing, HL-300 showed slow absorption, with mean Cmax ranging from 82.77 to 537.60 pg/mL and median Tmax of 24-36 h. After multiple dosing, steady state was reached by Day 6, with a Cmax,ss of 3059 pg/mL. Accumulation ratios for AUC and Cmax were 40.93 and 18.9, respectively. Most treatment-emergent adverse events were mild (Grade 1), and no serious adverse events were reported. .Conclusion: HL-300 was generally safe and well tolerated after single and multiple topical administrations, showing low systemic exposure after single dosing and predictable PK with steady-state attainment after repeated dosing. These findings support further clinical development and dose optimization in patients with AD.","PeriodicalId":12313,"journal":{"name":"Expert opinion on investigational drugs","volume":" ","pages":"463-475"},"PeriodicalIF":4.3,"publicationDate":"2026-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148197908","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 journey of targeted drug conjugates in solid tumors: moving beyond antibody drug conjugates. 实体肿瘤中靶向药物偶联物的旅程：超越抗体药物偶联物。

IF 4.3 2区医学

Expert opinion on investigational drugs Pub Date : 2026-06-01 Epub Date: 2026-06-14 DOI: 10.1080/13543784.2026.2683842

Stephen J Walsh, Francesca Wood, Leslie R DeMars, Gavin Bennett

{"title":"The journey of targeted drug conjugates in solid tumors: moving beyond antibody drug conjugates.","authors":"Stephen J Walsh, Francesca Wood, Leslie R DeMars, Gavin Bennett","doi":"10.1080/13543784.2026.2683842","DOIUrl":"10.1080/13543784.2026.2683842","url":null,"abstract":"Introduction: Antibody drug conjugates (ADCs) selectively deliver cytotoxins to tumor cells using highly specific monoclonal antibodies. While ADCs are a standard treatment paradigm in oncology, safety- and efficacy-related challenges remain. Additional platforms are needed to deliver more effective targeted therapy with differentiated safety profiles.Areas covered: This narrative review discusses the preclinical and clinical journey of ADCs, their shortcomings, and avenues for refinement. Development of novel antibody fragment-drug conjugates, small molecule-drug conjugates (SMDCs), peptide drug conjugates (PDCs), and Bicycle drug conjugate (BDC) molecules are discussed based on targeted literature searches for each technology described; searches were not bound by specific terms or dates in PubMed or ClinicalTrials.gov.Expert opinion: Targeted delivery of cytotoxins will continue to be a powerful tool against cancer; however, we believe novel SMDCs, PDCs, and BDC molecules will help circumvent current challenges surrounding cytotoxin-containing ADCs and provide differentiated treatment options in solid tumors, including those particularly difficult to treat. We expect promising preclinical and clinical data emerging for SMDCs, PDCs, and BDC molecules will translate into approvals, such as BDC molecules that are under pivotal randomized phase II clinical investigation. These novel treatments will increase survival and quality of life for patients with solid tumors.","PeriodicalId":12313,"journal":{"name":"Expert opinion on investigational drugs","volume":" ","pages":"429-447"},"PeriodicalIF":4.3,"publicationDate":"2026-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148156140","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

GV1001: repurposing a telomerase-derived peptide for neurological therapeutics. GV1001：重新利用端粒酶衍生肽用于神经治疗。

IF 4.3 2区医学

Expert opinion on investigational drugs Pub Date : 2026-05-01 Epub Date: 2026-05-18 DOI: 10.1080/13543784.2026.2674652

Kyoungho Suk, Won-Ha Lee

{"title":"GV1001: repurposing a telomerase-derived peptide for neurological therapeutics.","authors":"Kyoungho Suk, Won-Ha Lee","doi":"10.1080/13543784.2026.2674652","DOIUrl":"10.1080/13543784.2026.2674652","url":null,"abstract":"Introduction: Neurodegenerative and demyelinating diseases represent major unmet medical needs, with existing therapeutics demonstrating limited efficacy and significant safety concerns. Drug repurposing offers accelerated development timelines and established safety profiles. GV1001, a 16-amino acid telomerase-derived peptide originally developed as a cancer vaccine, has emerged as a promising multi-mechanism neuroprotective agent.Areas covered: This review examines preclinical evidence demonstrating GV1001 efficacy across Alzheimer's disease, stroke, experimental autoimmune encephalomyelitis, and depression models. Convergent mechanisms include gonadotropin-releasing hormone receptor-mediated neuroprotection, mitochondrial stabilization, modulation of glial functional states away from tissue-damaging inflammation, and promotion of remyelination. Clinical translation through Phase 2 trials in Alzheimer's disease demonstrated statistically significant cognitive improvements consistent with neurotrophic benefit, with excellent safety profiles lacking amyloid-related imaging abnormalities.Expert opinion: The network pharmacology approach of GV1001-simultaneously addressing multiple pathological processes-positions it as a differentiated alternative to single-target therapeutics. The dual anti-inflammatory and pro-remyelination profile of the peptide addresses critical unmet needs in progressive multiple sclerosis. Phase 3 confirmation, biomarker-driven patient stratification, and combination therapy investigations represent critical development priorities. Successful development may help validate multi-mechanism approaches in neurodegeneration, potentially catalyzing paradigm shifts from reductionist single-target strategies.","PeriodicalId":12313,"journal":{"name":"Expert opinion on investigational drugs","volume":" ","pages":"369-381"},"PeriodicalIF":4.3,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147927995","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

Discontinued therapies for sickle cell disease: status and future directions. 停止治疗镰状细胞病：现状和未来方向。

IF 4.3 2区医学

Expert opinion on investigational drugs Pub Date : 2026-05-01 Epub Date: 2026-04-23 DOI: 10.1080/13543784.2026.2663116

Akshay Sharma, Shruthi Suryaprakash, Liza-Marie Johnson, Yoram Unguru

{"title":"Discontinued therapies for sickle cell disease: status and future directions.","authors":"Akshay Sharma, Shruthi Suryaprakash, Liza-Marie Johnson, Yoram Unguru","doi":"10.1080/13543784.2026.2663116","DOIUrl":"10.1080/13543784.2026.2663116","url":null,"abstract":"Introduction: Over the past decade, the therapeutic landscape for sickle cell disease (SCD) has expanded beyond hydroxyurea to targeted small molecules, monoclonal antibodies, and transformative cellular and gene therapies. However, not every investigational approach survives the rigor of randomized trials, post-marketing surveillance, and commercial realities. High-profile discontinuations and trial failures have important implications for a disease community with longstanding unmet needs and a historical mistrust of biomedical research.Areas covered: This review analyzes the proximate causes of discontinuation of SCD therapies, highlighting recurrent themes such as overreliance on surrogate endpoints, trial design limitations, post-marketing safety surveillance gaps, enrollment challenges, and corporate reprioritization, and draws lessons for future drug development. We place the most consequential cases in context and discuss how next-generation agents, trial design innovations, biomarker-based strategies, and equitable deployment might reduce the likelihood and impact of future discontinuations. Finally, we propose pragmatic recommendations to maximize patient benefit while minimizing avoidable harms.Expert opinion: Recent advances in sickle cell therapies bring real promise alongside setbacks. Failures are part of progress, but better trial design, transparency, patient partnership, and shared data can reduce harm, strengthen trust, and ensure innovation translates into meaningful, durable patient benefit.","PeriodicalId":12313,"journal":{"name":"Expert opinion on investigational drugs","volume":" ","pages":"341-349"},"PeriodicalIF":4.3,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13453127/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147728830","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

New insights into the management of Behçet syndrome. behet综合征管理的新见解。

IF 4.3 2区医学

Expert opinion on investigational drugs Pub Date : 2026-05-01 Epub Date: 2026-05-18 DOI: 10.1080/13543784.2026.2676066

Sinem Nihal Esatoglu, Yesim Ozguler, Gulen Hatemi

{"title":"New insights into the management of Behçet syndrome.","authors":"Sinem Nihal Esatoglu, Yesim Ozguler, Gulen Hatemi","doi":"10.1080/13543784.2026.2676066","DOIUrl":"10.1080/13543784.2026.2676066","url":null,"abstract":"Introduction: Behçet syndrome (BS) is a multisystem variable-vessel vasculitis characterized by recurrent mucocutaneous manifestations and severe organ involvement affecting the eyes, blood vessels, nervous system, and gastrointestinal tract. Although therapeutic advances over the past two decades have significantly improved outcomes, important clinical questions remain regarding optimal treatment selection, sequencing, durability, and long-term safety.Areas covered: This narrative review summarizes current evidence on established and emerging therapies for BS, focusing on unresolved aspects of management. We discuss comparative data between infliximab and adalimumab, the evolving role of cyclophosphamide in major vascular involvement, treatment strategies in colchicine-resistant mucocutaneous and joint disease, the use of combination therapy with conventional immunosuppressives, and considerations for tapering or discontinuing biologics. We also review available evidence for alternative biologic and targeted therapies, including interleukin (IL)-1, IL-6, IL-12/23, IL-17 inhibitors, JAK inhibitors, and novel phosphodiesterase-4 inhibitors, as well as investigational agents. This review is based on a targeted literature search and the authors' expertise, including recent work for the updated 2025 EULAR recommendations for the management of BS.Expert opinion: Monoclonal anti-TNF antibodies remain the most supported biologic option across all disease manifestations. Emerging therapies show promise, but their long-term efficacy and safety require confirmation in controlled trials.","PeriodicalId":12313,"journal":{"name":"Expert opinion on investigational drugs","volume":" ","pages":"329-339"},"PeriodicalIF":4.3,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147962747","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