BioDrugs最新文献

{"title":"Long-Term Efficacy and Safety of Stapokibart in Adults with Moderate-to-Severe Atopic Dermatitis: An Open-Label Extension, Nonrandomized Clinical Trial.","authors":"Yan Zhao, Jing-Yi Li, Bin Yang, Yang-Feng Ding, Li-Ming Wu, Li-Tao Zhang, Jin-Yan Wang, Qian-Jin Lu, Chun-Lei Zhang, Fu-Ren Zhang, Xiao-Hong Zhu, Yu-Mei Li, Xiao-Hua Tao, Qing-Chun Diao, Lin-Feng Li, Jian-Yun Lu, Xiao-Yong Man, Fu-Qiu Li, Xiu-Juan Xia, Jiao-Ran Song, Ying-Min Jia, Li-Bo Zhang, Bo Chen, Jian-Zhong Zhang","doi":"10.1007/s40259-024-00668-z","DOIUrl":"10.1007/s40259-024-00668-z","url":null,"abstract":"<p><strong>Background: </strong>Stapokibart/CM310, a humanized monoclonal antibody targeting the interleukin-4 receptor α chain, has shown promising treatment benefits in patients with moderate-to-severe atopic dermatitis in previous phase II clinical trials.</p><p><strong>Objective: </strong>We aimed to evaluate the long-term efficacy and safety of stapokibart in adults with moderate-to-severe atopic dermatitis.</p><p><strong>Methods: </strong>Enrolled patients who previously completed parent trials of stapokibart received a subcutaneous stapokibart 600-mg loading dose, then 300 mg every 2 weeks up to 52 weeks. Efficacy outcomes included the proportions of patients with ≥ 50%/75%/90% improvements from baseline of parent trials in the Eczema Area and Severity Index, Investigator's Global Assessment, and weekly average of the daily Peak Pruritus Numerical Rating Scale.</p><p><strong>Results: </strong>In total, 127 patients were enrolled, and 110 (86.6%) completed the study. At week 52, the Eczema Area and Severity Index-50/75/90 response rates were 96.3%, 87.9%, and 71.0%, respectively. An Investigator's Global Assessment 0/1 with a ≥ 2-point reduction was achieved in 39.3% of patients at week 16, increasing to 58.9% at week 52. The proportions of patients with ≥ 3-point and ≥ 4-point reductions in the weekly average of daily Peak Pruritus Numerical Rating Scale scores were 80.2% and 62.2%, respectively, at week 52. Improvement in patients' quality of life was sustained over a 52-week treatment period. Treatment-emergent adverse events occurred in 88.2% of patients, with an exposure-adjusted event rate of 299.2 events/100 patient-years. Coronavirus disease 2019, upper respiratory tract infection, and conjunctivitis were the most common treatment-emergent adverse events.</p><p><strong>Conclusions: </strong>Long-term treatment with stapokibart for 52 weeks showed high efficacy and good safety profiles, supporting its use as a continuous long-term treatment option for atopic dermatitis.</p><p><strong>Clinical trial registration: </strong>ClinicalTrials.gov identifier: NCT04893707 (15 May, 2021).</p>","PeriodicalId":9022,"journal":{"name":"BioDrugs","volume":" ","pages":"681-689"},"PeriodicalIF":5.4,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141854701","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":"Prospects of Synergy: Local Interventions and CAR T Cell Therapy in Solid Tumors.","authors":"Anne Holtermann, Mila Gislon, Martin Angele, Marion Subklewe, Michael von Bergwelt-Baildon, Kirsten Lauber, Sebastian Kobold","doi":"10.1007/s40259-024-00669-y","DOIUrl":"10.1007/s40259-024-00669-y","url":null,"abstract":"<p><p>Chimeric antigen receptor T cell therapy has been established in the treatment of various B cell malignancies. However, translating this therapeutic effect to treat solid tumors has been challenging because of their inter-tumoral as well as intratumoral heterogeneity and immunosuppressive microenvironment. Local interventions, such as surgery, radiotherapy, local ablation, and locoregional drug delivery, can enhance chimeric antigen receptor T cell therapy in solid tumors by improving tumor infiltration and reducing systemic toxicities. Additionally, ablation and radiotherapy have proven to (re-)activate systemic immune responses via abscopal effects and reprogram the tumor microenvironment on a physical, cellular, and chemical level. This review highlights the potential synergy of the combined approaches to overcome barriers of chimeric antigen receptor T cell therapy and summarizes recent studies that may pave the way for new treatment regimens.</p>","PeriodicalId":9022,"journal":{"name":"BioDrugs","volume":" ","pages":"611-637"},"PeriodicalIF":5.4,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11358237/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141854702","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}

{"title":"Treatment Targets Should Influence Choice of Infliximab Dose Intensification Strategy in Inflammatory Bowel Disease: A Pharmacokinetic Simulation Study.","authors":"Ashish Srinivasan, Daniel van Langenberg, Peter De Cruz, Jonathan Segal, Abhinav Vasudevan, Richard N Upton","doi":"10.1007/s40259-024-00673-2","DOIUrl":"10.1007/s40259-024-00673-2","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;The optimal infliximab dose intensification strategy to address loss of response associated with subtherapeutic infliximab trough levels remains uncertain, as does whether post-intensification trough and treatment targets should influence this decision.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Objectives: &lt;/strong&gt;This pharmacokinetic simulation study aimed to identify infliximab dose intensification strategies capable of achieving post-intensification infliximab trough thresholds associated with clinical and objective treatment targets in Crohn's disease and ulcerative colitis.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;A validated pharmacokinetic infliximab model, applied to 200 simulated patients, identified those with subtherapeutic (&lt; 3.00 mg/L) trough levels after 30 weeks of standard (5 mg/kg 8-weekly) dosing, and subsequently applied 10 dose intensification strategies over a further 32 weeks. The proportion of simulations achieving 32-week post-intensification infliximab trough levels associated with endoscopic remission (ulcerative colitis &gt; 7.50 mg/L, Crohn's disease &gt; 9.70 mg/L) was the primary outcome, with perianal fistula healing (Crohn's disease &gt; 10.10 mg/L) and clinical improvement (ulcerative colitis &gt; 3.70 mg/L, Crohn's disease &gt; 7.00mg/L) evaluated as secondary outcomes. All outcomes were stratified by intensity of dose intensification, with standard (≤ 10 mg/kg 8-weekly or 5 mg/kg 4-weekly; n = 5) and intensive (&gt; 10 mg/kg 8-weekly or 5 mg/kg 4-weekly; n = 5) dosing strategies defined, respectively.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;The median pre-intensification infliximab trough level was 0.91 mg/L (interquartile range 1.37). Intensive dosing strategies were more likely to achieve infliximab trough concentrations associated with endoscopic remission (ulcerative colitis 36.48% vs. 10.80%, Crohn's disease 25.98 vs. 4.68%), perianal fistula healing (24.52% vs. 4.36%) and clinical improvement (ulcerative colitis 61.90% vs. 34.86%, Crohn's disease 40.32 vs. 12.08%) than standard intensification strategies (all p &lt; 0.01). When controlling for cumulative (mg/kg) infliximab dose over 32 weeks, strategies that concurrently dose increased and interval shortened achieved the highest infliximab trough levels (all p &lt; 0.01).&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Conclusion: &lt;/strong&gt;This simulation-based analysis highlights the potential of using post-intensification infliximab trough thresholds associated with aspirational treatment targets in Crohn's disease and ulcerative colitis to guide choice of infliximab dose intensification strategy. Intensive dose intensification strategies, particularly those that concurrently dose increase and interval shorten, appear to achieve higher infliximab levels than standard dose intensification strategies. This may be particularly important in the pursuit of stringent endpoints, such as endoscopic remission and fistula healing, which have been consistently associated with higher infliximab trough levels. These findings require va","PeriodicalId":9022,"journal":{"name":"BioDrugs","volume":" ","pages":"691-702"},"PeriodicalIF":5.4,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11358351/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142016304","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}

{"title":"Targeting Neurological Aspects of Mucopolysaccharidosis Type II: Enzyme Replacement Therapy and Beyond.","authors":"Alessandra Zanetti, Rosella Tomanin","doi":"10.1007/s40259-024-00675-0","DOIUrl":"10.1007/s40259-024-00675-0","url":null,"abstract":"<p><p>Mucopolysaccharidosis type II (MPS II) is a rare, pediatric, neurometabolic disorder due to the lack of activity of the lysosomal hydrolase iduronate 2-sulfatase (IDS), normally degrading heparan sulfate and dermatan sulfate within cell lysosomes. The deficit of activity is caused by mutations affecting the IDS gene, leading to the pathological accumulation of both glycosaminoglycans in the lysosomal compartment and in the extracellular matrix of most body districts. Although a continuum of clinical phenotypes is described, two main forms are commonly recognized-attenuated and severe-the latter being characterized by an earlier and faster clinical progression and by a progressive impairment of central nervous system (CNS) functions. However, attenuated forms have also been recently described as presenting some neurological involvement, although less deep, such as deficits of attention and hearing loss. The main treatment for the disease is represented by enzyme replacement therapy (ERT), applied in several countries since 2006, which, albeit showing partial efficacy on some peripheral organs, exhibited a very poor efficacy on bones and heart, and a total inefficacy on CNS impairment, due to the inability of the recombinant enzyme to cross the blood-brain barrier (BBB). Together with ERT, whose design enhancements, performed in the last few years, allowed a possible brain penetration of the drug through the BBB, other therapeutic approaches aimed at targeting CNS involvement in MPS II were proposed and evaluated in the last decades, such as intrathecal ERT, intracerebroventricular ERT, ex vivo gene therapy, or adeno-associated viral vector (AAV) gene therapy. The aim of this review is to summarize the main clinical aspects of MPS II in addition to current therapeutic options, with particular emphasis on the neurological ones and on the main CNS-targeted therapeutic approaches explored through the years.</p>","PeriodicalId":9022,"journal":{"name":"BioDrugs","volume":" ","pages":"639-655"},"PeriodicalIF":5.4,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11358193/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142035148","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}

{"title":"Correction to: Demonstration of Physicochemical and Functional Similarity of the Biosimilar BAT1806/BIIB800 to Reference Tocilizumab.","authors":"Yujie Liu, Jianhua Xie, Zhuxiang Li, Xiong Mei, Di Cao, Shengfeng Li, Linda Engle, Suli Liu, Hans C Ebbers, Cuihua Liu","doi":"10.1007/s40259-024-00672-3","DOIUrl":"10.1007/s40259-024-00672-3","url":null,"abstract":"","PeriodicalId":9022,"journal":{"name":"BioDrugs","volume":" ","pages":"725"},"PeriodicalIF":5.4,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11358319/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142016303","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}

{"title":"Systematic Review of Genetic Substrate Reduction Therapy in Lysosomal Storage Diseases: Opportunities, Challenges and Delivery Systems.","authors":"Marina Beraza-Millor, Julen Rodríguez-Castejón, Ana Del Pozo-Rodríguez, Alicia Rodríguez-Gascón, María Ángeles Solinís","doi":"10.1007/s40259-024-00674-1","DOIUrl":"10.1007/s40259-024-00674-1","url":null,"abstract":"<p><strong>Background: </strong>Genetic substrate reduction therapy (gSRT), which involves the use of nucleic acids to downregulate the genes involved in the biosynthesis of storage substances, has been investigated in the treatment of lysosomal storage diseases (LSDs).</p><p><strong>Objective: </strong>To analyze the application of gSRT to the treatment of LSDs, identifying the silencing tools and delivery systems used, and the main challenges for its development and clinical translation, highlighting the contribution of nanotechnology to overcome them.</p><p><strong>Methods: </strong>A systematic review following the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) reporting guidelines was performed. PubMed, Scopus, and Web of Science databases were used for searching terms related to LSDs and gene-silencing strategies and tools.</p><p><strong>Results: </strong>Fabry, Gaucher, and Pompe diseases and mucopolysaccharidoses I and III are the only LSDs for which gSRT has been studied, siRNA and lipid nanoparticles being the silencing strategy and the delivery system most frequently employed, respectively. Only in one recently published study was CRISPR/Cas9 applied to treat Fabry disease. Specific tissue targeting, availability of relevant cell and animal LSD models, and the rare disease condition are the main challenges with gSRT for the treatment of these diseases. Out of the 11 studies identified, only two gSRT studies were evaluated in animal models.</p><p><strong>Conclusions: </strong>Nucleic acid therapies are expanding the clinical tools and therapies currently available for LSDs. Recent advances in CRISPR/Cas9 technology and the growing impact of nanotechnology are expected to boost the clinical translation of gSRT in the near future, and not only for LSDs.</p>","PeriodicalId":9022,"journal":{"name":"BioDrugs","volume":" ","pages":"657-680"},"PeriodicalIF":5.4,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11358353/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142035057","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}

{"title":"A Population Pharmacokinetic Model of Tocilizumab in Kidney Transplant Patients Treated for Chronic Active Antibody-Mediated Rejection: Comparison of Plasma Exposure Between Intravenous and Subcutaneous Administration Schemes.","authors":"Capucine Arrivé, Caroline Bazzoli, Thomas Jouve, Johan Noble, Lionel Rostaing, Françoise Stanke-Labesque, Zoubir Djerada","doi":"10.1007/s40259-024-00676-z","DOIUrl":"10.1007/s40259-024-00676-z","url":null,"abstract":"<p><strong>Background: </strong>Tocilizumab prevents the clinical worsening of chronic active antibody-mediated rejection (CAAMR) in kidney transplant recipients. Following a global shortage of the intravenous pharmaceutical form in 2022, patients were switched from monthly intravenous administration of 8 mg/kg to weekly subcutaneous injection of 162 mg, raising the question of bioequivalence between these schemes of administration.</p><p><strong>Aims: </strong>We aimed to compare the areas under the curve (AUC) of tocilizumab in virtual simulations of populations treated with the two administration schemes and to identify the covariates that could contribute to pharmacokinetic variability of tocilizumab in kidney transplant patients with CAAMR who received tocilizumab as salvage treatment.</p><p><strong>Methods: </strong>This retrospective monocentric study included 43 kidney transplant patients (202 tocilizumab concentrations) with CAAMR treated with intravenous or subcutaneous tocilizumab between December 2020 and January 2023. We developed a population pharmacokinetic model using nonlinear mixed effects modeling and identified the covariates that could contribute to tocilizumab AUC variability. Monte Carlo simulations were then performed to assess the subcutaneous and intravenous tocilizumab AUC for 0-28 days (M1), 56-84 days (M3), 140-168 days (M6), and 308-336 days (M12). Bioequivalence was defined by SC/IV AUC geometric mean ratios (GMRs) between 0.80 and 1.25.</p><p><strong>Results: </strong>A two-compartment model with parallel linear and nonlinear elimination best described the concentration-time data. Significant covariates for tocilizumab clearance were body weight, urinary albumin-to-creatinine ratio (ACR), and inflammation status [C-reactive protein (CRP) ≥ 5 mg/L]. The GMR values and their 90% confidence intervals at M3, M6, and M12 were within the 0.8-1.25 margin for equivalence. Conversely, the 90% prediction intervals of the GMR were much wider than the 90% confidence intervals and did not fall within 0.8 and 1.25.</p><p><strong>Conclusions: </strong>From month 3 of treatment, the subcutaneous and intravenous tocilizumab administration schemes provided average bioequivalent pharmacokinetic exposure at the population level but not at the individual level. Body weight, inflammation, ACR, and administration scheme should be considered to personalize the dose of tocilizumab for patients with CAAMR. Further studies are required to determine the target of tocilizumab exposure in kidney transplant patients with CAAMR.</p>","PeriodicalId":9022,"journal":{"name":"BioDrugs","volume":" ","pages":"703-716"},"PeriodicalIF":5.4,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141987368","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":"Insulin Icodec: First Approval.","authors":"Hannah A Blair","doi":"10.1007/s40259-024-00670-5","DOIUrl":"10.1007/s40259-024-00670-5","url":null,"abstract":"<p><p>Insulin icodec (AWIQLI^®) is an ultra-long-acting basal insulin analogue that is being developed by Novo Nordisk for the treatment of diabetes mellitus. Administered once weekly as a subcutaneous injection, insulin icodec is designed to improve treatment adherence and glycaemic control relative to once-daily insulin analogues. On 7 March 2024, insulin icodec was approved in Switzerland for the treatment of diabetes mellitus in adults. Insulin icodec was approved in Canada on 12 March 2024 for the once-weekly treatment of adults with diabetes mellitus to improve glycaemic control and received EU approval in May 2024 for the treatment of diabetes mellitus in adults. This article summarizes the milestones in the development of insulin icodec leading to this first approval for diabetes mellitus.</p>","PeriodicalId":9022,"journal":{"name":"BioDrugs","volume":" ","pages":"717-724"},"PeriodicalIF":5.4,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141730995","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":"Barriers and Enablers Affecting the Uptake of Biosimilar Medicines Viewed Through the Lens of Actor Network Theory: A Systematic Review.","authors":"Chad Rieger, Judith A Dean, Lisa Hall, Paola Vasquez, Gregory Merlo","doi":"10.1007/s40259-024-00659-0","DOIUrl":"10.1007/s40259-024-00659-0","url":null,"abstract":"<p><strong>Background and objective: </strong>Biosimilars represent an opportunity to realise savings against the costs of innovative medicines. Despite efforts made by stakeholders, there are numerous barriers to the uptake of biosimilars. To realise the promise of biosimilars reducing costs, barriers must be identified, understood, and overcome, and enablers magnified. The aim of this systematic review is to summarise the enablers and barriers affecting uptake of biosimilars through the application of a classification system to organise them into healthcare professional (HCP), patient, or systemic categories.</p><p><strong>Methods: </strong>A systematic literature search was performed in PubMed, Scopus, CINAHL, eConlit, and Embase. Included were primary research studies published in English between Jan 2017 through June 2023 focused on enablers and barriers affecting uptake of biosimilars. Excluded studies comprised comparisons of biosimilar efficacy and safety versus the reference biologic. One reviewer extracted data that included classification of barriers or enablers, the sub-classification, and the identification of the degree of agency associated with the actor through their role and associations as a mediator within their network, through the application of Actor Network Theory. The data were validated by a second reviewer (PV).</p><p><strong>Results: </strong>Of the 94 studies included, 59 were cross-sectional, 20 were qualitative research, 12 were cohort studies, and three were economic evaluations. Within the review, 51 of the studies included HCP populations and 35 included patients. Policies and guidelines were the most cited group of enablers, overall. Systemic enablers were addressed in 29 studies. For patients, the most frequently cited enabler was positive framing of a biosimilar, while for HCPs, cost benefit was the most frequently noted enabler. The most frequently discussed systemic barrier to biosimilar acceptance was lack of effective policies or guidelines, followed by lack of financial incentives, while the most significant barriers for HCPs and patients, respectively, were their lack of general knowledge about biosimilars and concerns about safety and efficacy. Systemic actors and HCPs most frequently acted with broad degree of agency as mediators, while patient most frequently acted with a narrow degree of agency as mediators within their networks.</p><p><strong>Conclusions: </strong>Barriers and enablers affecting uptake of biosimilars are interconnected within networks, and can be divided into systemic, HCP, and patient categories. Understanding the agency of actors within networks may allow for more comprehensive and effective approaches. Systemic enablers in the form of policies appear to be the most effective overall levers in affecting uptake of biosimilars, with policy makers advised to give careful consideration to appropriately educating HCPs and positively framing biosimilars for patients.</p>","PeriodicalId":9022,"journal":{"name":"BioDrugs","volume":" ","pages":"541-555"},"PeriodicalIF":5.4,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11247062/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141327204","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}

{"title":"Stem Cells and Stem Cell-Derived Factors for the Treatment of Inflammatory Bowel Disease with a Particular Focus on Perianal Fistulizing Disease: A Minireview on Future Perspectives.","authors":"Amy L Lightner, Peter M Irving, Graham M Lord, Aline Betancourt","doi":"10.1007/s40259-024-00661-6","DOIUrl":"10.1007/s40259-024-00661-6","url":null,"abstract":"<p><p>Inflammatory bowel disease remains a difficult disease to effectively treat, especially fistulizing Crohn's disease. Perianal fistulas in the setting of Crohn's disease remain an area of unmet need with significant morbidity in this patient population. Up to one third of Crohn's patients will have perianal fistulizing disease and current medical and surgical interventions are of limited efficacy. Thus, most patients experience significant morbidity, narcotic use, and loss of employment and end up with multiple surgical interventions. Mesenchymal stem cells (MSCs) have shown efficacy in phase 3 clinical trials, but considerable infrastructure challenges make MSCs limited with regard to scalability in clinical practice. Extracellular vesicles, being derived from MSCs and capturing the secretome functionality of MSCs, offer similar physiological utility regarding mechanism, while also providing an off the shelf regenerative medicine product that could be widely used in daily clinical practice.</p>","PeriodicalId":9022,"journal":{"name":"BioDrugs","volume":" ","pages":"527-539"},"PeriodicalIF":5.4,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11247053/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141445324","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}