Drug discoveries & therapeutics最新文献

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Comparison of salivary Mucin 5B (MUC5B) secretion between heat-not-burn tobacco users and non-smokers. 热不燃烟草使用者与非吸烟者唾液粘蛋白5B (MUC5B)分泌的比较。
Drug discoveries & therapeutics Pub Date : 2026-03-04 Epub Date: 2026-02-12 DOI: 10.5582/ddt.2025.01115
Yukihiro Mori, Mamoru Tanaka, Hana Kozai, Yuka Aoyama, Morihiro Ito
{"title":"Comparison of salivary Mucin 5B (MUC5B) secretion between heat-not-burn tobacco users and non-smokers.","authors":"Yukihiro Mori, Mamoru Tanaka, Hana Kozai, Yuka Aoyama, Morihiro Ito","doi":"10.5582/ddt.2025.01115","DOIUrl":"10.5582/ddt.2025.01115","url":null,"abstract":"<p><p>The use of heat-not-burn (HNB) tobacco is rapidly increasing, particularly in Japan; however, its health effects, especially on innate immunity in the oral mucosa, remain unclear. This cross-sectional study aimed to compare the secretion levels of mucin 5B (MUC5B), a mucin that forms a protective barrier role on the oral mucosal surface, between HNB tobacco smokers and non-smokers. MUC5B levels in stored saliva samples from 208 males (147 in the non-smoking group, 32 in the HNB tobacco group, 15 in the paper cigarette group, and 14 in the dual-use group) were measured using enzyme-linked immunoassay (ELISA). The primary outcome was the MUC5B secretion rate (μg/min = MUC5B concentration × saliva secretion rate). Significant differences in MUC5B secretion rates were observed among the four groups, with multiple comparisons showing significantly lower rates in the HNB tobacco and dual-use groups than in the non-smoking group (p = 0.042 and p < 0.001, respectively). The observed decrease in salivary MUC5B secretion is a concern, as it may comprise oral hygiene and immune function. From a public health perspective, these findings provide a basis to discourage the use and combined use of HNB tobacco.</p>","PeriodicalId":520606,"journal":{"name":"Drug discoveries & therapeutics","volume":" ","pages":"85-90"},"PeriodicalIF":0.0,"publicationDate":"2026-03-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146169224","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Development of ginger extract-loaded self-nanoemulsifying drug delivery system for enhanced solubility of ginger extract. 姜提取物自纳米乳化给药系统的研制提高了姜提取物的溶解度。
Drug discoveries & therapeutics Pub Date : 2026-03-04 Epub Date: 2026-02-13 DOI: 10.5582/ddt.2025.01121
Maung Maung Than, Chuda Chittasupho, Songwut Yotsawimonwat, Kantaporn Kheawfu
{"title":"Development of ginger extract-loaded self-nanoemulsifying drug delivery system for enhanced solubility of ginger extract.","authors":"Maung Maung Than, Chuda Chittasupho, Songwut Yotsawimonwat, Kantaporn Kheawfu","doi":"10.5582/ddt.2025.01121","DOIUrl":"10.5582/ddt.2025.01121","url":null,"abstract":"<p><p>The well-known medicinal plant ginger (Zingiber officinale Roscoe) has numerous health benefits, but its key bioactive compound, 6-gingerol, suffers from poor water solubility and stability. This study aimed to enhance the oral delivery of ginger extract by formulating a self-nanoemulsifying drug delivery system (SNEDDS) using a design of experiments (DoE) approach. Ginger rhizomes were extracted by ultrasound-assisted extraction, with a 10-min extraction time yielding the highest 6-gingerol content. An I-optimal mixture design was then applied to develop SNEDDS formulations using castor oil, Cremophor RH40, various co-surfactants (Span 20 or Span 80), and co-solvents (polyethyleneglycol 400 (PEG 400) or ethanol). The optimized SNEDDS readily self-emulsified in gastric medium, producing nano-sized droplets (42.5-78.1 nm) with low polydispersity (0.12-0.58) within 10 min. The ginger extract-loaded SNEDDS (G-SNEDDS) achieved high encapsulation efficiencies, exceeding 90% for both 6-gingerol and 6-shogaol, and significantly enhanced the in vitro release of 6-gingerol, reaching cumulative release levels of approximately 90-100% over 48 h, compared to only 67% from the unformulated extract. Transmission electron microscopy (TEM) confirmed the formation of uniform, spherical nanoemulsion droplets. Short-term stability testing indicated that the optimized formulation remained physically stable, as evidenced by minimal changes in droplet size, and preserved most of the 6-gingerol content under ambient storage conditions; however, exposure to elevated temperatures accelerated the conversion of 6-gingerol to 6-shogaol. Overall, the optimized SNEDDS significantly enhanced the solubility, dissolution, and storage stability of ginger extract, offering a promising strategy to improve the oral bioavailability of the therapeutically active constituents present in ginger.</p>","PeriodicalId":520606,"journal":{"name":"Drug discoveries & therapeutics","volume":" ","pages":"26-41"},"PeriodicalIF":0.0,"publicationDate":"2026-03-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146204721","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Equivalence in the modernization of Chinese medicine: A multi-dimensional analysis of active components, processing methods, and clinical outcomes in formula granules. 中药现代化中的等效:配方颗粒有效成分、炮制方法和临床效果的多维分析。
Drug discoveries & therapeutics Pub Date : 2026-03-04 Epub Date: 2026-02-27 DOI: 10.5582/ddt.2025.01134
Bo Xie, Xi Qu, Huijing Shi, Yi Zhou, Dan Deng, Yuanru Liu, Rong Jiang, Ling Wang
{"title":"Equivalence in the modernization of Chinese medicine: A multi-dimensional analysis of active components, processing methods, and clinical outcomes in formula granules.","authors":"Bo Xie, Xi Qu, Huijing Shi, Yi Zhou, Dan Deng, Yuanru Liu, Rong Jiang, Ling Wang","doi":"10.5582/ddt.2025.01134","DOIUrl":"10.5582/ddt.2025.01134","url":null,"abstract":"<p><p>The feasibility of substituting traditional decoction pieces with Chinese herbal formula granules fundamentally hinges on the equivalence of their active components. Formula granules face complex compositional changes during manufacturing: volatile oils suffer substantial losses (retention rates below 30% in aromatic herbs), thermolabile glycosides undergo degradation during high-temperature processing, and Maillard reactions generate novel compounds whose pharmacological contributions remain unclear. Although advanced analytical technologies such as ultra-performance liquid chromatography quadrupole time-of-flight mass spectrometry (UPLC-Q-TOF-MS) can identify thousands of components, existing quality control standards still rely on 1-3 pharmacopeial markers, and fingerprint similarity criteria lack uniformity (0.80-0.95), inadequately reflecting the true quality of multi-component systems. Bioequivalence studies demonstrate that formula granules generally exhibit 60-90% performance of traditional decoctions in vitro, with comparable area under the curve (AUC) and Cmax values for certain components but significant discrepancies for others. Critically, high chemical similarity cannot guarantee clinical therapeutic equivalence-the logical chain from\"component equivalence\" to \"therapeutic equivalence\" remains unestablished. Clinical research is sparse, with high-quality randomized controlled trials (RCTs) representing less than 5% of studies and head-to-head comparisons particularly scarce. Future research must develop volatile component preservation technologies, establish comprehensive synergistic effect evaluation methodologies, and most importantly, conduct large-scale clinical trials. Without these efforts, the scientific credibility and international acceptance of formula granules will remain questionable.</p>","PeriodicalId":520606,"journal":{"name":"Drug discoveries & therapeutics","volume":" ","pages":"12-25"},"PeriodicalIF":0.0,"publicationDate":"2026-03-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147329082","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Protein persulfidation: The missing link in Alzheimer's disease defense mechanisms. 蛋白质过硫化:阿尔茨海默病防御机制的缺失环节。
Drug discoveries & therapeutics Pub Date : 2026-03-04 Epub Date: 2026-02-08 DOI: 10.5582/ddt.2026.01004
Ya-Nan Ma, Xiaoxi Huang, Ying Xia, Peipei Song, Xiqi Hu
{"title":"Protein persulfidation: The missing link in Alzheimer's disease defense mechanisms.","authors":"Ya-Nan Ma, Xiaoxi Huang, Ying Xia, Peipei Song, Xiqi Hu","doi":"10.5582/ddt.2026.01004","DOIUrl":"10.5582/ddt.2026.01004","url":null,"abstract":"<p><p>Despite decades of research dominated by the amyloid-beta hypothesis, clinical treatment of Alzheimer's disease (AD) has yet to achieve a decisive breakthrough. This editorial advances an alternative pathological paradigm: the collapse of endogenous hydrogen sulfide (H₂S) signaling represents a central failure point in the brain's intrinsic defense mechanisms against AD. We dissect the molecular cascade triggered by cystathionine γ-lyase (CSE) deficiency, focusing on how reduced persulfidation of glycogen synthase kinase 3β (GSK3β) directly promotes Tau hyperphosphorylation and subsequent neuronal injury. A critical message of this commentary is the need to dispel the oversimplified notion that sulfide supplementation alone can confer neuroprotection. Because H₂S works within a narrow therapeutic window and has complex hormetic effects, untargeted dietary or environmental exposure cannot match the spatiotemporal precision of endogenous signaling. Instead, it may increase the risk of toxicity. By integrating analyses of transsulfuration metabolism, mitochondrial function, and nutritional status, we propose a precision medicine framework centered on brain-targeted delivery technologies and metabolic correction strategies to selectively restore compromised H₂S signaling networks. This conceptual shift marks a new direction in AD research, shifting the focus from clearing toxic protein aggregates to restoring endogenous neuronal resilience.</p>","PeriodicalId":520606,"journal":{"name":"Drug discoveries & therapeutics","volume":" ","pages":"1-6"},"PeriodicalIF":0.0,"publicationDate":"2026-03-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146145418","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Analgesic effects of Goreisan in patients with glossodynia: A preliminary exploratory study. 葛瑞森对舌痛患者的镇痛作用:一项初步的探索性研究。
Drug discoveries & therapeutics Pub Date : 2026-03-04 Epub Date: 2026-02-14 DOI: 10.5582/ddt.2025.01128
Takao Ayuse, Shuntaro Sato, Ichiro Okayasu, Mizuki Tachi-Yoshida, Jun Sato, Hironori Saisu, Yoko Yamazaki, Hiroko Imura
{"title":"Analgesic effects of Goreisan in patients with glossodynia: A preliminary exploratory study.","authors":"Takao Ayuse, Shuntaro Sato, Ichiro Okayasu, Mizuki Tachi-Yoshida, Jun Sato, Hironori Saisu, Yoko Yamazaki, Hiroko Imura","doi":"10.5582/ddt.2025.01128","DOIUrl":"10.5582/ddt.2025.01128","url":null,"abstract":"<p><p>Glossodynia-related pain refers to persistent, chronic pain occurring on the oral mucosal surface. Various medications are prescribed depending on symptom profiles and have demonstrated therapeutic benefits; however, these agents are often associated with adverse effects such as drowsiness or dizziness. Goreisan, a traditional Japanese Kampo medicine, has long been used empirically for glossodynia-related pain, particularly in patients reporting symptom fluctuation associated with weather changes. Nevertheless, high-quality clinical evidence supporting its efficacy remains limited. This multicenter, randomized, open-label preliminary exploratory study enrolled patients receiving treatment for glossodynia-related pain. Participants were assigned to receive either Goreisan (7.5 g/day for 12 weeks) in addition to standard therapy or standard therapy alone. Pain intensity was assessed using the visual analog scale (VAS) at baseline and at 4, 8, and 12 weeks. Salivary amylase activity was measured as an exploratory stress-related biomarker, and atmospheric pressure was recorded at each outpatient visit. Owing to insufficient enrollment, all analyses were descriptive and exploratory. Pain intensity improved from baseline in both groups. However, the proportion of patients achieving ≥ 20% improvement in VAS at week 12 was not higher in the Goreisan group than in the control group. Weak negative correlations between VAS scores and barometric pressure were observed in both groups (r ≈ - 0.2). No clinically relevant adverse events or hepatic dysfunction related to Goreisan were identified. This preliminary exploratory study did not confirm a clear analgesic efficacy of Goreisan for glossodynia. However, the findings provide descriptive data on pain trajectories, safety, and potential meteorological associations, supporting the need for future large-scale, double-blind, placebo-controlled trials.</p>","PeriodicalId":520606,"journal":{"name":"Drug discoveries & therapeutics","volume":" ","pages":"80-84"},"PeriodicalIF":0.0,"publicationDate":"2026-03-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146204723","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
New diagnostic code "5B72 Undernutrition in Adults" approved for inclusion in the 11th Revision of the International Classification of Diseases (ICD-11). 批准将新的诊断代码“5B72成人营养不良”列入《国际疾病分类》(ICD-11)第11版。
Drug discoveries & therapeutics Pub Date : 2026-01-07 Epub Date: 2025-11-14 DOI: 10.5582/ddt.2025.01118
Machiko Uenishi, Peipei Song
{"title":"New diagnostic code \"5B72 Undernutrition in Adults\" approved for inclusion in the 11th Revision of the International Classification of Diseases (ICD-11).","authors":"Machiko Uenishi, Peipei Song","doi":"10.5582/ddt.2025.01118","DOIUrl":"10.5582/ddt.2025.01118","url":null,"abstract":"<p><p>The World Health Organization (WHO) has approved a new diagnostic code, \"5B72 Undernutrition in Adults\", in the 11th Revision of the International Classification of Diseases (ICD-11). Prior to this update, undernutrition in adults was represented only by the code \"5B54 Underweight in Adults\". However, the increasing diversity of causes of undernutrition in adults and the accompanying rise in associated diseases have been recognized as global health challenges in recent years. The approval of the \"5B72 Undernutrition in Adults\" category signifies its formal recognition as a distinct disease entity and is expected to improve data collection and research, enhance clinical diagnosis and interventions, and support policy development and nutritional education.</p>","PeriodicalId":520606,"journal":{"name":"Drug discoveries & therapeutics","volume":" ","pages":"437-439"},"PeriodicalIF":0.0,"publicationDate":"2026-01-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145535164","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Verification of the protective effect of transfecting brain-derived neurotrophic factor and B-cell lymphoma 2 genes on retina ganglion cells in a rat model of optic nerve injury. 转染脑源性神经营养因子和b细胞淋巴瘤2基因对视神经损伤大鼠视网膜神经节细胞保护作用的验证
Drug discoveries & therapeutics Pub Date : 2026-01-07 Epub Date: 2025-12-13 DOI: 10.5582/ddt.2025.01101
Zijia Zhao, Yuan Fang, Gezhi Xu, Jiawei Xiong, Xiaofen Mo, Jiawen Fan
{"title":"Verification of the protective effect of transfecting brain-derived neurotrophic factor and B-cell lymphoma 2 genes on retina ganglion cells in a rat model of optic nerve injury.","authors":"Zijia Zhao, Yuan Fang, Gezhi Xu, Jiawei Xiong, Xiaofen Mo, Jiawen Fan","doi":"10.5582/ddt.2025.01101","DOIUrl":"10.5582/ddt.2025.01101","url":null,"abstract":"<p><p>This pilot study investigated the protective effect of transfecting brain-derived neurotrophic factor (BDNF) and B-cell lymphoma 2 (bcl-2) genes in retinal ganglion cells (RGCs) using in vivo electroporation in an adult rat optic nerve transection model. Sprague-Dawley rats were randomly divided into five groups: BDNF(+)/bcl-2(+), BDNF(+), bcl-2(+), empty plasmid (EP), and no surgery (NS). The plasmids were intravitreally injected and electroporated into the left eye. Seven days later, optic nerve transection was performed in all groups except the NS group. Protein expression was examined using Western blotting, RGC survival was quantified using 1,1'-dioctadecyl-3,3,3',3'-tetramethyl-indocarbocyanine perchlorate (DiI) retrograde labeling, and apoptosis was assessed using terminal deoxynucleotidyl transferase dUTP nick end labeling (TUNEL) at multiple time points (7, 14, and 28 d after transfection). A significantly higher number of DiI (+) RGCs and lower number of apoptotic cells were observed in the BDNF(+)/bcl-2(+), BDNF(+), and bcl-2(+) groups compared to those in the EP group at all time points. The number of DiI (+) RGCs in the three treatment groups was significantly lower than that in the NS group. However, there were no significant differences among the three treatment groups. The protective effects of gene transfection tended to be strongest in the BDNF(+)/bcl-2(+) group, followed by the BDNF(+) group and then the bcl-2(+) group. Thus, all gene transfection treatments had a protective effect against the loss of DiI(+) RGCs induced by optic nerve transection but did not result in full recovery. This study also confirmed the value of in vivo electroporation. The findings of this pilot study provide a working base for the development of gene therapy for blinding optic nerve disorders.</p>","PeriodicalId":520606,"journal":{"name":"Drug discoveries & therapeutics","volume":" ","pages":"395-403"},"PeriodicalIF":0.0,"publicationDate":"2026-01-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145759290","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Comparative study of contrast-enhanced ultrasound and contrast-enhanced computed tomography in the diagnosis of benign and malignant lesions of the gallbladder. 超声造影与计算机断层造影诊断胆囊良恶性病变的比较研究。
Drug discoveries & therapeutics Pub Date : 2026-01-07 Epub Date: 2025-12-16 DOI: 10.5582/ddt.2025.01105
Liang Fang, Zhiying Qiu, Lingwen Xu, Fei Hu, Yuchen Tao, Shengnan Ding, Jing Zhou, Ling Wang, Lin Chen
{"title":"Comparative study of contrast-enhanced ultrasound and contrast-enhanced computed tomography in the diagnosis of benign and malignant lesions of the gallbladder.","authors":"Liang Fang, Zhiying Qiu, Lingwen Xu, Fei Hu, Yuchen Tao, Shengnan Ding, Jing Zhou, Ling Wang, Lin Chen","doi":"10.5582/ddt.2025.01105","DOIUrl":"10.5582/ddt.2025.01105","url":null,"abstract":"<p><p>This study compared the diagnostic value of contrast-enhanced ultrasound (CEUS) and contrast-enhanced computed tomography (CECT) for differentiating benign and malignant gallbladder (GB) lesions. A retrospective analysis was performed on 151 hospitalized patients with GB lesions: 74 underwent CEUS alone, 25 CECT alone, and 52 both examinations preoperatively. Histopathology served as the reference standard. Imaging features of benign/malignant lesions were compared using t-test or chi-square (χ²) test; CEUS quantitative parameters were analyzed via t-test. Diagnostic efficacy of the two modalities was compared by χ² test. Of 151 patients, 57 (37.7%) had malignant and 94 (62.3%) benign lesions. Statistically significant differences between benign and malignant groups were observed in CEUS/CECT features (vascular enhancement morphology, contrast distribution, GB wall integrity, ill-defined borders with adjacent liver) and CEUS washout time (WT) (all P < 0.05). In the entire cohort, no significant differences existed between CEUS and CECT in sensitivity (89.2% vs. 85.7%), specificity (78.8% vs. 74.3%), accuracy (84.9% vs. 80.5%), positive predictive value (PPV) (85.7% vs. 80.0%) and negative predictive value (NPV) (83.7% vs. 81.2%) (all P > 0.05). Similar non-significant differences were noted in the 52-patient subgroup (sensitivity: 65.0% vs. 80.0%; specificity: 62.5% vs. 80.0%; accuracy: 63.5% vs. 76.9%; PPV: 40.6% vs. 50.0%; NPV: 51.3% vs. 66.7%; all P > 0.05). CEUS is effective for differentiating benign and malignant GB lesions, with diagnostic efficacy comparable to CECT.</p>","PeriodicalId":520606,"journal":{"name":"Drug discoveries & therapeutics","volume":" ","pages":"414-424"},"PeriodicalIF":0.0,"publicationDate":"2026-01-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145777121","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Elamipretide: The first cardiolipin-directed mitochondrial therapeutic for Barth syndrome approved under accelerated approval. Elamipretide:首个加速批准的Barth综合征的心磷脂导向线粒体治疗药物。
Drug discoveries & therapeutics Pub Date : 2026-01-07 Epub Date: 2025-11-20 DOI: 10.5582/ddt.2025.01111
Chenru Zhao, Xuemei Zhuang, Jianjun Gao
{"title":"Elamipretide: The first cardiolipin-directed mitochondrial therapeutic for Barth syndrome approved under accelerated approval.","authors":"Chenru Zhao, Xuemei Zhuang, Jianjun Gao","doi":"10.5582/ddt.2025.01111","DOIUrl":"10.5582/ddt.2025.01111","url":null,"abstract":"<p><p>Barth syndrome (BTHS) is a rare X-linked mitochondrial disorder caused by tafazzin mutations that impair cardiolipin remodeling, leading to mitochondrial dysfunction and symptoms such as cardiomyopathy, myopathy, and neutropenia. On September 19, 2025, the U.S. Food and Drug Administration (FDA) granted accelerated approval to elamipretide, the first therapy directly targeting the mitochondrial etiology of BTHS. Elamipretide binds to cardiolipin on the inner mitochondrial membrane, stabilizing respiratory chain supercomplexes, enhancing electron transport efficiency, and reducing reactive oxygen species production. In a randomized, double-blind, placebo-controlled, crossover trial, elamipretide resulted in no significant improvement in the 6-minute walk test or fatigue scores; however, sustained benefits were observed during a 168-week open-label extension. The most common adverse events were mild injection-site reactions. As a condition of accelerated approval, a confirmatory trial is required. Elamipretide represents a promising therapy addressing an unmet medical need in BTHS and provides a foundation for future mitochondria-targeted treatments.</p>","PeriodicalId":520606,"journal":{"name":"Drug discoveries & therapeutics","volume":" ","pages":"435-436"},"PeriodicalIF":0.0,"publicationDate":"2026-01-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145558917","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Real-world use of non-oral administration of oral anticancer agents via the simple suspension method in Japanese patients with cancer: A nationwide claims-based analysis. 在日本癌症患者中,通过简单的悬浮液方法使用非口服给药的口服抗癌药物:一项全国性的基于索赔的分析。
Drug discoveries & therapeutics Pub Date : 2026-01-07 Epub Date: 2025-12-11 DOI: 10.5582/ddt.2025.01095
Hiroaki Ohta, Takeo Yasu
{"title":"Real-world use of non-oral administration of oral anticancer agents via the simple suspension method in Japanese patients with cancer: A nationwide claims-based analysis.","authors":"Hiroaki Ohta, Takeo Yasu","doi":"10.5582/ddt.2025.01095","DOIUrl":"10.5582/ddt.2025.01095","url":null,"abstract":"<p><p>The enteral administration of drugs as suspensions is a potential alternative route for patients with dysphagia. The simple suspension method is commonly used in Japan; however, nationwide data are lacking. This study aimed to investigate the real-world use and potential impact of oral anticancer agent (OAA) suspensions in patients with dysphagia in Japan. A large Japanese administrative claims database was used in this retrospective study (April 2014 to August 2023). Patients with cancer who had nasogastric, gastrostomy, or jejunostomy tubes and received OAAs during non-oral intake periods were included. These periods were defined using procedural codes, and it was assumed that the prescribed drugs were suspended. Three frequently used drugs (temozolomide, lenvatinib, and osimertinib) were analyzed. Patients were divided into suspension and non-suspension groups using propensity score matching, and overall survival (OS) was compared using the Kaplan-Meier method. Among 25,252 patients with feeding tubes, 126 received OAAs during the non-oral period. The median age was 76 years, and 62% had gastrostomy tubes. Temozolomide was the most common drug suspension, followed by lenvatinib and osimertinib. No significant difference in OS was observed between the temozolomide and lenvatinib groups. However, OS of the osimertinib suspension group was shorter than that of the non-suspension group (p = 0.047). This large-scale study of OAA suspensions in Japan suggests that drug suspensions may support continued anticancer treatment in patients with dysphagia. However, drug stability and exposure risks should be considered. Further evidence and more specific guidelines are required to ensure safe and effective use.</p>","PeriodicalId":520606,"journal":{"name":"Drug discoveries & therapeutics","volume":" ","pages":"378-388"},"PeriodicalIF":0.0,"publicationDate":"2026-01-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145727900","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
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