Open-label Clinical Phase 1/2 Study to Assess the Safety and Efficacy of the SpectraCure P18 System and Verteporfin for Injection for the Treatment of Primary Localized Prostate Cancer

The goal of this study is to obtain safety data, establish dose parameters, and effectiveness of treatment for the SpectraCure P18 System with IDOSE®, together with verteporfin for injection (VFI) as photosensitizer, for the treatment of primary localized prostate cancer.
The study will be divided into two parts, with Phase I, dose-escalation, to study safety and establish an effective light dose, followed by Phase II, cohort expansion, to evaluate clinical efficacy and confirm safety/tolerability. The subjects will be followed for a period of 18 months to determine the primary outcome. The long-term follow-up is an additional 18 months, i.e. a total of 36 months.
Interstitial Photodynamic Therapy (PDT) will be performed during general anesthesia. Optical fibers will be inserted into the prostate with a transperineal approach using transrectal ultrasound guidance. The intent is to deliver an adequate light dose throughout the prostate. Subjects will receive VFI intravenously, approximately 60-90 minutes prior to light delivery.

开始日期2025-01-06

申办/合作机构

SpectraCure AB

NCT06381154

/ Recruiting临床2期IIT

Photodynamic Priming to Facilitate Immunologic Activity of Anti-PD1 in Patients with Pancreatic Cancer

This phase II trial tests how well photoradiation with verteporfin and pembrolizumab plus standard of care chemotherapy works in treating patients with pancreatic cancer that cannot be removed by surgery (unresectable), that has spread to nearby tissue or lymph nodes (locally advanced) or to other places in the body (metastatic). Photoradiation uses light activated drugs, such as verteporfin, that become active when exposed to light. These activated drugs may kill tumor cells. Vertoporfin may also increase tumor response to immunotherapy. Immunotherapy with monoclonal antibodies, such as pembrolizumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Chemotherapy drugs, such as modified fluorouracil, leucovorin, irinotecan, and oxaliplatin (mFOLFIRINOX), work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Photoradiation with verteporfin and pembrolizumab plus standard of care chemotherapy may kill more tumor cells in patients with unresectable, locally advanced or metastatic pancreatic cancer.

开始日期2024-12-06

申办/合作机构

Mayo Clinic

[+1]

NCT06306638

/ Recruiting临床1/2期IIT

A Phase I/II Study of Interstitial Photodynamic Therapy Following Palliative Radiotherapy for Patients With Inoperable Malignant Central Airway Obstruction

This phase I/II trial studies the side effects of interstitial photodynamic therapy following palliative radiotherapy and how well it works in treating patients with inoperable malignant central airway obstruction. Patients who have advanced stage cancer tumors in the lung can often have the breathing passages to the lung partially or completely blocked. These tumors could be due to lung cancer or other cancers (e.g., renal, breast, kidney, etc.) that spread to the lung. This blockage puts the patient at a higher risk for respiratory failure, post-obstructive pneumonia, and prolonged hospitalizations. Treatment for these patients may include bronchoscopic intervention (such as mechanical removal, stenting, laser cauterization, or ballooning), radiation therapy with and without chemotherapy. While palliative x-ray radiotherapy may help in shrinking the tumor, high dose curative radiotherapy that can ablate (a localized, nonsurgical destruction) the tumor also has high risk to cause significant toxicity, including bleeding, abnormal connections or passageways between organs or vessels and abnormal scar tissue that can also produce airway obstruction. Photodynamic therapy (PDT) is another possible treatment that can provide local control of the tumor. PDT consists of injecting a light sensitive drug (photosensitizer, PS) into the vein, waiting for the PS to accumulate in the tumor, and then activating it with a red laser light. Radiation therapy uses high energy x-rays, particles, or radioactive seeds to kill cancer cells and shrink tumors. Giving interstitial photodynamic therapy following palliative radiotherapy may improve tumor response and survival without the serious side effects that are associated with the typical high dose curative x-ray radiotherapy alone in patients with malignant central airway obstruction.

开始日期2024-10-01

申办/合作机构

Roswell Park Comprehensive Cancer Center

[+1]

100 项与维替泊芬相关的临床结果

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100 项与维替泊芬相关的转化医学

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100 项与维替泊芬相关的专利（医药）

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1,907

项与维替泊芬相关的文献（医药）

2025-12-01·MOLECULAR BIOLOGY REPORTS

Targeting breast cancer stem cells in ER-positive breast cancer by repurposing the benzoporphyrin derivative verteporfin as a YAP/TAZ small molecule inhibitor

Article

作者: Roy, Himansu ; Chatterji, Urmi ; Talukdar, Priyanka Dey

BACKGROUND:

Current treatment strategies for hormone-dependent breast cancers, including adjuvant endocrine therapy, often fail due to persistence of breast cancer stem cells (brCSCs), which are significant contributors to tumor recurrence and treatment resistance. Therefore, gaining deeper insights into the molecular regulators driving breast cancer aggressiveness is important. Moreover, given the complexities and expenses involved in developing new pharmacological agents, the strategic repurposing of existing FDA-approved drugs to target these key molecular pathways presents a compelling approach for identifying novel therapeutic interventions aimed at mitigating tumor refractoriness.

METHODS:

The study employs survival analysis from TCGA database, protein expression analyses alongside aldefluor assays, sphere formation efficiency tests to evaluate cellular stemness, and DCFDA analysis combined with antioxidant enzyme assays to investigate redox imbalance in brCSCs. These analyses were conducted following the genetic deletion of YAP/TAZ and pharmacological treatment with verteporfin.

RESULTS:

The study demonstrated that transcriptional co-activators YAP/TAZ are significantly upregulated in chemotreated ER⁺ patient breast tumors and MCF-7 mammospheres, where it was found to interact with the transcription factor SOX2 within the nuclear compartment. Genetic ablation and pharmacological inhibition of YAP/TAZ markedly impaired stemness properties and disrupted redox homeostasis in the mammospheres. Additionally, treatment with verteporfin led to a substantial reduction in the frequency and viability of brCSCs, suggesting their effective eradication.

CONCLUSION:

This study highlights the potential of repurposing verteporfin, an FDA-approved drug originally formulated for age-related macular degeneration, as a therapeutic agent for targeting YAP/TAZ-mediated stemness and redox balance in brCSCs, thereby reducing their viability in ER-positive breast cancers.

2025-05-01·SURVEY OF OPHTHALMOLOGY

Enhancing anti-vascular endothelial growth factor with photodynamic therapy for polypoidal choroidal vasculopathy: A meta-analysis

Review

作者: Lin, Ting-Han ; Lin, Hung-Yi ; Tseng, Po-Chen

Anti-vascular endothelial growth factor (anti-VEGF) agents administered as either monotherapy or combination with verteporfin photodynamic therapy (PDT) are the 2 dominant treatment for polypoidal choroidal vasculopathy (PCV); however, controversies remain due to small sample sizes and inconsistency in prognosis from randomized controlled trials (RCTs). In accordance with the PRISMA statement, we investigated the efficacy of PDT plus anti-VEGF combination with anti-VEGF monotherapy. This study was accepted by the International Prospective Register of Systematic Reviews (CRD42023471362). Studies published up to July, 2024, were retrieved from PubMed, Embase, and Cochrane databases. A total of 7 RCTs with 926 eyes were reviewed. In 6 trials, combination therapy showed significantly higher rate of complete polyp regression (risk ratio [RR]: 1.56, 95 % CI: 1.15-2.13, p = 0.005). In 5 trials, combination therapy also significantly reduced the number of anti-VEGF injections (SMD: -0.65, 95 % CI: -0.95 to -0.35, p < 0.0001). For best corrected visual acuity improvement, central retinal thickness reduction, and rate of ocular adverse events, the performance of the 2 modalities were comparable. We conclude that PDT plus anti-VEGF combination therapy constitutes a safe and effective modality and should be considered the first-line treatment for PCV.

2025-04-01·JOURNAL OF CONTROLLED RELEASE

Repurposing verteporfin and hyaluronic acid gel for ocular surface treatment to prevent corneal scarring

Article

作者: Kang, Nae-Won ; Myung, David ; Jiang, Li ; Liu, Wendy W ; Lin, Danting ; Jang, Kyeongwoo ; Seo, Youngyoon Amy ; Wungcharoen, Thitima ; Garcia-Sanchez, Julian ; Chen, Fang ; Basco, Chris A

There is an unmet need for point-of-care therapies to prevent scarring and promote corneal clarity after injury, which is essential for maintaining vision. Verteporfin, an inhibitor of Yes-associated protein (YAP), has been shown to prevent fibrosis in several organs. Visudyne (VP) is an FDA-approved liposomal formulation of verteporfin used to treat abnormal blood vessels in the eye. Here, we showed that VP reduces myofibroblast formation in corneal stromal fibroblasts. To prolong the residence time of verteporfin on the ocular surface, the cohesive viscoelastic ProVisc® hyaluronic acid (HA) gel was hybridized to VP. This formulation is readily translatable because both VP and ProVisc® HA gel are FDA-approved agents. The ProVisc® HA gel increased the residence of subconjunctivally injected verteporfin 12-fold at 24 h after injection compared with pure VP. A single subconjunctival administration of VP hybridized within ProVisc® HA gel (VP/HA hydrogel) significantly reduced YAP activation, corneal fibrosis, neovascularization, and inflammation, leading to reduced opacity without compromising epithelial wound healing in mechanically injured rat corneas. This work demonstrated that VP hybridized with a viscoelastic HA gel can be readily repurposed to promote scar-less healing in the cornea.

项与维替泊芬相关的新闻（医药）

2025-01-21

·药事纵横

脂质体药物及其研究进展

许多游离药物具有溶解度低、稳定相差和安全性低在内的诸多特点而限制了其临床应用，纳米技术的进步为使用纳米颗粒进行药物递送创造了无限可能。FDA已经批准了约100种纳米药物上市，通常用作药物输送系统的纳米颗粒有包括脂质体在内的多种体系，它们的粒径范围从几纳米到几百纳米不等。它们通过装载药物使之提高稳定性，克服组织细胞的摄取阻碍，增加药物在体内靶位的富集，提高了药物的安全性与治疗效果。图1：通常用作药物输送系统的纳米颗粒。a 脂质体b 病毒载体c 自组装蛋白d 聚合物纳米颗粒e 金属纳米颗粒f 单壁碳纳米。g树枝状聚合物h 具有核/壳结构的聚苯乙烯涂层磁性颗粒i胶束制剂在Pubmed上以“纳米”、“脂质体”、“药物”为相关关键词来搜索相关研究，纳米相关药物的发文量在2016年后超过脂质体且较为迅速增长，脂质体相关研究中的药物研究占了很大比重，相关发文量都呈现增长模式，预计在不久后会有更多的实验室产品走到工厂或是市场，为各种疾病带来新的治疗机会。图2：以纳米、脂质体和药物相关关键词搜索近10年出现的文章数脂质体纳米平台应用包括生物医学诊断和生物化学中的信号载体、疫苗佐剂和纳米医学中的递送载体等一系列领域。1995年FDA批准Johnson的包载有盐酸阿霉素脂质体抗肿瘤药物Doxil上市，目前美国食品药品监督管理局（FDA）批准了多款脂质体药物上市，适应症也从单一的肿瘤领域拓展到了感染、炎症和镇痛一系列领域。表1：FDA批准上市的脂质体药物表2：新型脂质体（LNP）上市核酸药物/疫苗图3：脂质体/脂质纳米颗粒（LNP，一种新型脂质体）发展时间轴脂质体最早由Bengham在1960年代发现，后来成为最广泛的药物输送系统之一。脂质体是由磷脂和胆固醇组成的球形结构，脂类有天然与人工合成两类，磷脂结构可以带有电荷以便控制其吸收排布。脂质体表面可以带有电荷，由于电荷排斥可以起到稳定脂质体的作用，负电荷的存在（如DMPG）使得脂质体容易被巨噬细胞识别并且能够通过内吞作用进入细胞（速率比中性脂质更快），从而缩短循环时间，而带正电荷的脂质体可能与血液中的阴离子成分（如血浆蛋白）相互作用，被MPS摄取清除。事实上，带正电荷脂质体与带负电的核酸作用而有利于核酸的递送，目前两款新型脂质体技术包载的核酸疫苗（两种阳离子脂质为：SM-102/ALC-0315）便是利用了这个原理而使脂质体在抗击新冠疫情中大放异彩。胆固醇控制磷脂堆积、膜流动性和脂质体表面电荷的能力，脂质体中胆固醇的添加使脂质体在血流中的稳定时间从几分钟延长到了几小时。脂质体也可由单个或多个脂质双分子层组装而成，目前FDA批准上市产品大都为100nm左右大小的单层小囊泡。添加聚合物如聚乙二醇则有延长的其循环的特性（增加脂质体与血液中其他成分的空间位阻），保护负载药物免受灭活或代谢降解，进一步增强稳定性并改善细胞内摄入。脂质体成分中特定靶向成分的添加或进行功能化（使用多糖、糖蛋白或特定受体的配体）来达到靶向的作用（被动靶向主要应用于肿瘤的治疗，依靠于肿瘤血管特性来实现，而主动靶向主要是通过脂质表面的功能化来达到目的）。脂质体产品结构比较复杂，环境或结构的变化可能对脂质体而言是引起其安全性和有效性的挑战，脂质体药物的辅料中一般会添加一些其他成分，如pH缓冲用的柠檬酸/柠檬酸盐体系、渗透压调节用的氯化钠体系、稳定用的糖类。图4：载药脂质体的常见结构以及组分化合物结构示意图目前对于脂质体的制备已经有了许多的技术方法：薄膜水合法、乙醇/乙醚注射法、反相蒸发法、洗涤剂去除法、微流控法、膜挤出法、均质化、超声法、冷冻干燥方法、超临界流体方法等。对于制备完成的脂质体特征（粒径、电位、形态、相位和层状性）的评价则有许多种技术。表3：脂质体参数评估的方法特征技术粒径显微镜、纳米颗粒跟踪分析（NTA）、动态光散射（DLS）、扫描/透射电镜（SEM/TEM）、原子力显微镜（AFM）、低温透射电镜（Cryo-TEM）电位电泳迁移率、DLS 形态 Cryo-TEM、TEM、AFM 相位特性差示扫描量热法（DSC）、X射线衍射（XRD）、热重分析（TGA）层状性 Cryo-TEM 、核磁脂质体的粒径大小影响着药物吸收与分布（体循环、清除、外渗、转运摄取等），理想的粒径在50-200nm范围之内。脂质体所带的净电荷为Zeta电位，电位的大小的绝对值影响着脂质体的稳定性。脂质体电位收到外部环境和离子强度的影响，绝对值较小的脂质体之间排斥力较弱，随时间的推移聚集的可能性较大。显微镜检测是对于脂质体形态检测最直接的方式，其中Cryo-TEM技术检测可以较好的保留脂质体的原始结构，最大限度减小脂质体形态的扭曲变化；AFM技术是一种快速、强大和非侵入性的技术，在脂质体原生条件下提供到高分辨率三维图像。脂质体相行为影响着脂质体的稳定性，DSC是脂质体相行为检测最常见方法。脂质体的层状性影响着药物的释放，Cryo-TEM是最常用的方法，它提供了关于脂质体层状双层厚度和双层的距离等信息。当然，每种技术有自己的优缺点，多种方法联合使用才能从多角度将脂质体的性质进行更加全面的表征。脂质体的疏水外壳包裹着亲水内核，亲水性药物一般包裹在亲水内核中，而疏水药物则包裹在疏水外壳中，目前已经开发出了脂质体的主动载药法（一般发生在脂质体制备完成后阶段）（如Doxil、Marqibo、DaunoXome和Onivyde）和被动载药法（一般发生在脂质体制备过程阶段）（如AmBisome、Visudyne、Arikayce、DepoCyte、DepoDur和Expel）。Vyxeos则采用了组合方式载药（阿糖胞苷和柔红霉素）。目前还有第三种脂质体载药技术---化学偶联技术，具有合适的化学官能团将药物与脂质体连接来控制药物的负载与释放。图5：脂质体的药物装载我们在临床试验资料库（https://clinicaltrials.gov/）上以“脂质体”（liposome）为关键词进行所搜来汇总目前脂质体相关研究的临床情况，2019年后的疫情几年完成的临床数呈下降趋势。我们发现2020年后（近3年）有35项实验已经完成，其中有4项附有研究结果。图6：以“脂质体”（liposome）关键词搜索近10年临床情况表4：临床研究部分信息脂质体的临床研究主要方向是装载药物来对各种疾病进行治疗，研究1的试验调查了伊立替康脂质体和贝伐珠单抗在治疗对铂类治疗（铂类耐药）、复发（复发）或对治疗无反应（难治性）的卵巢癌、输卵管癌或原发性腹膜癌患者中的作用。研究2的目的是确定脂质体布比卡因与盐水在腰椎手术中减少阿片类药物使用的功效，并确定患者报告的结果和住院时间是否存在差异。研究3评估 Exparel、Exparel与盐酸布比卡因混合与盐酸布比卡因联合治疗下肢手术受试者术后镇痛的疗效、安全性和药代动力学。研究4探索Exparel（脂质体布比卡因）在内收肌管阻滞中的有效性（用于全膝关节置换术手术疼痛控制）。通过脂质体改善靶向部位的递送、提高疗效和降低的全身毒性，这是大多数进入临床试验脂质体新药的典型目标。脂质体的出现，以其独特的性质而是许多药物为治疗疾病成为了可能，但是脂质体的组成（成分/纯度）、理化性质、稳定性、表征技术和一系列的临床药学研究（药代/药动/利用度等）仍有许多问题待解决，相信不久的将来在新方法的研究以及新技术的引入会刺激脂质体研究的进一步发展。 REFERENCE 1. Beltrán-Gracia E et al., Cancer Nano. 2019 2. Almeida B et al., Molecules. 2020 3. Gote V et al., Int J Mol Sci. 2023 4. Tenchov R et al., ACS Nano. 2021 药事纵横征稿启事

上市批准核酸药物疫苗信使RNA

2024-02-03

·今日头条

Nature新闻：人类肠菌中古怪的微小RNA

02月03日的《热心肠日报》，我们解读了 15 篇文献，关注：RNA，GLP-1RA，植物真菌，微流控，早产儿，脂肪肝，肥胖，纳米医学，噬菌体，菌群多样性，益生菌，菲仕兰，和黄医药，Daehwa，百吉福，Earthful。该篇日报由R·AI辅助创作生成，人工审核校对。 Nature：人类肠道中发现RNA新实体——“方尖碑”（新闻） Nature——[64.8] ① 根据近期发表的一项预印本研究，人类微生物群刚刚收获一个新成员——比病毒还小的微小RNA片段，在人类肠道和口腔细菌内定殖；② 虽然遗传物质片段太小，不足以被视为标准的生命形式，但其是向细胞传递指令的最小已知元素之一，其编码序列是全新的；③ 作者将该新元素称为“方尖碑”，尽管与许多类病毒具有相同的形状，但其基因序列截然不同，表明它们是一个独立但相关的群体；④ 各大洲人类粪便样本中都有大量“方尖碑”，来自身体不同部位微生物中的“方尖碑”具有不同的序列；⑤ 由于“方尖碑”所含基因不同于迄今为止在其他生物中发现的基因，它们包含了一类不同的RNA，这些RNA已经在人类和全球微生物群中定殖，并且未被注意到。【原文信息】 ‘Wildly weird’ RNA bits discovered infesting the microbes in our guts 2024-01-29 , doi: 10.1038/d41586-024-00266-7 国内团队BMJ：15种GLP-1RA在2型糖尿病中的疗效及安全性对比 British Medical Journal——[105.7] ① 北京中医药大学的万瑾毅团队发表的一项荟萃分析结果，总结了15种GLP-1受体激动剂（GLP-1RA）在成人2型糖尿病患者中的疗效和安全性，发现Tirzepatide在血糖控制方面效果最显著，CagriSema在体重管理方面表现最佳；② 纳入76项研究进行荟萃分析，共涉及15种GLP-1RA及39,246名2型糖尿病患者；③ 15种GLP-1RA均可有效降低糖化血红蛋白和空腹血糖，其中Tirzepatide的降糖效果最好；④ CagriSema（司美格鲁肽+卡格雷肽）的减重效果最好；⑤ 司美格鲁肽可有效降低LDL胆固醇和总胆固醇；⑥ 高剂量GLP-1RA可能导致胃肠道不良事件。【原文信息】 Comparative effectiveness of GLP-1 receptor agonists on glycaemic control, body weight, and lipid profile for type 2 diabetes: systematic review and network meta-analysis 2024-01-29 , doi: 10.1136/bmj-2023-076410 Science：受体相关激酶控制植物-真菌共生过程中的脂质供应 Science——[56.9] ① 本研究揭示了植物与丛枝菌根真菌（AM真菌）共生过程中，膜蛋白CKL1和CKL2的关键作用，它们通过调控转录因子影响脂质供应程序，并在内共生体适应中发挥重要作用；② CKL1和CKL2与LysM受体样激酶和DMI2相互作用，并被它们磷酸化，对AM共生至关重要；③ 脂质供应的启动与丛枝菌根真菌分支和RAM1调控元件的启动同时发生，对于内共生体的完全适应至关重要；④ CKL1和CKL2是AM共生所必需的，它们控制着转录因子的表达，而转录因子则调控着部分脂质供应程序；⑤ 这些发现为理解植物与真菌共生关系的分子机制提供了重要线索，有望为农业生产和生物技术领域提供新的思路和方法。【原文信息】 Receptor-associated kinases control the lipid provisioning program in plant-fungal symbiosis 2024-01-25 , doi: 10.1126/science.ade1124 微流控芯片生态进化获得氧化应激适应性益生菌 Small——[13.3] ① 实验室生态进化可克服益生菌工业应用面临氧化应激的缺陷，但目前微流控生态进化技术存在空间定义和细胞传代缺陷；② 本研究利用微流控芯片平台EVoc成功驱动益生菌鼠李糖乳杆菌GG对氧化应激的自适应进化，使其在高H2O2浓度下表现出显著的生长和抵抗能力；③ 经过两次适应性进化，菌株在H2O2浓度为1mM时的生长时间从31小时缩短至21小时，2mM时为28小时，3mM时为42小时；④ 适应性菌株在形态、基因表达模式和基因组序列上与野生型有所不同，蛋白质omega-amidase中存在潜在的有义单核苷酸突变。【原文信息】 Adaptive Laboratory Evolution of Probiotics toward Oxidative Stress Using a Microfluidic-Based Platform 2024-01-21 , doi: 10.1002/smll.202306974 益生菌在术后管理早产儿肠道手术的作用（荟萃分析） Cochrane Database of Systematic Reviews——[8.4] ① 评估在35周孕龄后出生的新生儿进行胃肠手术后给予益生菌的疗效和安全性，用于术后管理；② 与安慰剂相比，补充益生菌对于接受胃肠手术的新生儿在降低获得性败血症的风险（阳性细菌培养，局部或全身性）或达到全肠道喂养的时间上几乎没有影响；③ 两个研究组间都没有报道出院前死亡的情况；④ 相比安慰剂组，益生菌组婴儿肠道双歧杆菌科相对丰度明显更高，但其临床意义仍需探究。【原文信息】 Probiotics for the postoperative management of term neonates after gastrointestinal surgery 2024-01-23 , doi: 10.1002/14651858.CD012265.pub2 国内团队：多形拟杆菌或可治疗脂肪肝 Gut Microbes——[12.2] ① 中国医学科学院彭宗根团队研究了多形拟杆菌（Bt）对非酒精性脂肪肝病（NAFLD）代谢紊乱的作用并阐明其潜在机制；② 给予Bt治疗可显著改善高脂饮食小鼠的代谢综合征，包括减轻体重和脂肪堆积、降低高脂血症和胰岛素抵抗、预防脂肪肝和肝损伤；③ Bt通过调节肠道菌群组成，降低厚壁菌门/拟杆菌门比例，增加肠-肝叶酸水平和肝代谢物，从而缓解代谢功能障碍；④ Bt治疗还可增加小鼠肝脏中多不饱和脂肪酸的比例，改善非酒精性脂肪肝病；⑤ 本研究突出Bt作为潜在NAFLD益生菌的治疗作用，为开发新的治疗策略提供了重要的理论基础。【原文信息】 Bacteroides thetaiotaomicron ameliorates mouse hepatic steatosis through regulating gut microbial composition, gut-liver folate and unsaturated fatty acids metabolism 2024-01-26 , doi: 10.1080/19490976.2024.2304159 肥胖相关的肠菌特征（荟萃分析） Gut Microbes——[12.2] ① 使用来自17个不同国家的3329个样本（肥胖，n=1494；对照，n=1835），揭示肥胖与肠菌间的关联，鉴定出肥胖相关的物种和通路，为其研究和干预提供了重要线索；② 在肥胖者肠道微生物组中观察到多样性显著减少，推测这种减少导致了健康微生物功能的紊乱；③ 鉴定出25种高度预测的物种和37种通路与肥胖的标志相关联，并在独立数据集中得以验证；④ 肥胖者的肠道中SCFAs产生者和促进肠屏障完整性的物种减少，氨基酸、酶辅因子和肽聚糖合成通路显著富集；⑤ 绘制出重要的与肥胖相关功能变化的贡献者，并观察到这些贡献者既是数据集特定的，也是跨数据集共享的。【原文信息】 Meta-analysis reveals obesity associated gut microbial alteration patterns and reproducible contributors of functional shift 2024-01-24 , doi: 10.1080/19490976.2024.2304900 国内团队：纳米粒子声动力疗法治疗幽门螺杆菌感染 Nature Communications——[16.6] ① 中国科学技术大学阳丽华团队开发了一种新的声动力疗法，使用预先加载了Verteporfin的乳磷脂双层包被的聚（乳酸-聚乙二醇）纳米颗粒，Ver-PLGA@Lecithin，利用纳米颗粒和超声波成功治疗了幽门螺杆菌感染，且不影响肠道菌群平衡；② Ver-PLGA@Lecithin纳米颗粒能中和幽门螺杆菌的毒力因子，并通过产生活性氧杀灭细菌；③ 在小鼠模型中，声动力疗法与标准三联抗生素疗法效果相当，且不引起肠道菌群失调，仅引起乳酸菌丰度升高；④ 这一疗法为克服抗生素耐药提供了潜在解决方案，减少了抗生素耐药的风险，且对肠道菌群的干扰最小化。【原文信息】 A nanoparticle-based sonodynamic therapy reduces Helicobacter pylori infection in mouse without disrupting gut microbiota 2024-01-29 , doi: 10.1038/s41467-024-45156-8 Nature子刊：在1300年前人类粪便中发现的古代细菌噬菌体基因组 Nature Communications——[16.6] ① 通过分析先前从古代粪便和人类肠内容物样本中生成的DNA序列数据集，鉴定出一个与现代Mushuvirus mushu完全相同的古代噬菌体基因组（一种感染肠道共生细菌的病毒）；② 基因组的DNA损伤模式与其古老起源一致，尽管经过1300年的进化，古代Mushuvirus基因组与其现代对应物共享97.7%的核苷酸同一性，表明噬菌体和其宿主间存在长期关系；③ 重建并验证了过去5300年中的297个其他噬菌体基因组，包括属于未知家族的噬菌体；④ 这些发现拓展了噬菌体领域，为揭示跨数千年的噬菌体-细菌相互作用提供了重要见解。【原文信息】 Ultraconserved bacteriophage genome sequence identified in 1300-year-old human palaeofaeces 2024-01-23 , doi: 10.1038/s41467-023-44370-0 Cell子刊：纳米比亚居伊桑西人群肠道微生物多样性调查 Cell Reports——[8.8] ① 纳米比亚Ju|'hoansi族群是一个以狩猎和采集为主的族群，很少收到现代工业化社会的影响，本研究对该族群的肠道微生物组进行了调查研究，该族群与其他人群具有不同的肠道微生物核心组成；② 该族群肠道细菌组成与典型的狩猎采集族群类似，富含普雷沃氏菌、布劳蒂亚菌、普拉梭菌亚、琥铂酸弧菌和密螺旋菌等；③ 其肠道真菌主要包括动物病原体和植物腐生菌，如镰刀菌、东方伊萨酵母和扇菇菌等；④ 相比较其他因素，该族群的饮食和文化是其肠道微生物组组成的主要影响因素，。⑤ 这些结果为理解不同生活方式对肠道微生物组的影响提供了新视角，有助于拓展对人类肠道微生物组多样性的认识，为定制个性化的医疗和营养方案提供参考。【原文信息】 Documenting the diversity of the Namibian Ju|'hoansi intestinal microbiome 2024-01-19 , doi: 10.1016/j.celrep.2024.113690 皇家菲仕兰母乳低聚糖原料2'-FL泰国获批 ① 近日，荷兰皇家菲仕兰公司宣布，旗下2’-盐藻基乳糖（2’-FL）原料获得泰国监管部门批准，可用于婴幼儿配方奶粉产品；② 菲仕兰成为泰国唯一一家可同时提供母乳低聚糖和低聚半乳糖的原料供应商；③ 2’-FL已被证实对支持肠道健康和婴儿发育具有重要作用，菲仕兰公司计划将该原料与Vivinal低聚半乳糖等益生元成分复配，为消费者提供更具健康支持功效的婴配产品。【原文信息】 FrieslandCampina Ingredients secures Aequival 2’-FL approval in Thailand 2024-01-30 , Nutraceutical Business Review 和黄医药呋喹替尼中国香港地区获批 ① 1月30日，和黄医药宣布呋喹替尼在中国香港地区获批，用于治疗转移性结直肠癌患者；② 呋喹替尼是一种口服血管内皮生长因子受体（VEGFR）-1、-2及-3抑制剂，在大陆地区、中国澳门以及美国已获批用于结直肠癌治疗；③ 呋喹替尼的独特设计使其激酶选择性更高，具有更低的脱靶毒性、更高的耐受性，以及对靶点更稳定的覆盖；④ 基于中国国家药监局的批准，以及在中国香港当地的临床数据，和黄医药提交了呋喹替尼在中国香港的注册申请。【原文信息】和黄医药「呋喹替尼」在中国香港地区获批 2024-01-31 , 医药观澜 Daehwa二线胃癌药Liporaxel孤儿药资格认定获EMA推荐 ① 1月29日，韩国制药公司Daehwa Pharm宣布，旗下管线药物Liporaxel用于二线治疗胃癌孤儿药资格认定（ODD），获得了欧洲药品管理局积极意见；② Liporaxel是世界上首个口服紫杉醇制剂，Daehwa Pharm公司采用脂质自乳化递送技术开发而来；③ 在中韩两国开展的2个3期临床试验中，Liporaxel表现出非劣效性，不良反应降低；④ Daehwa Pharm国内合作公司海和药物正积极寻求Liporaxel在中国的批准上市。【原文信息】 Daehwa Pharm's Liporaxel receives positive EMA opinion for ODD as 2nd-line gastric cancer drug 2024-01-29 , Korea BIomedical Review 奶酪品牌「百吉福」获得凯辉基金独家战略投资 ① 1月26日，凯辉基金宣布与Savencia建立战略合作伙伴关系，并对Savencia旗下奶酪品牌百吉福进行独家战略投资；② 早在1996年，Savencia便正式进入中国市场，在天津设立了第一家工厂，成为中国首家外商独资的奶酪生产企业；③ Savencia将利用自身在产品创新、市场适应性和技术先进性方面的优势，进一步拓展中国市场；④ 凯辉基金的产业资源和行业洞察也将为百吉福在产品开发、品牌塑造、渠道拓展和供应链管理等方面带来新的发展。【原文信息】凯辉基金对Savencia旗下品牌百吉福进行独家战略投资 2024-01-30 , Foodaily 印度营养品牌「Earthful」获得3.3亿卢比种子轮融资 ① 近日，印度消费者营养品牌Earthful宣布完成3300万卢比（约合人民币285万元）的天使轮融资；② 投资方包括风投机构Green Ivy Venture和几位天使投资人；③ Earthful公司计划用融资进行研发投入、构建多渠道销售体系以及扩大团队规模；④ Earthful公司专注于开发植物基健康营养补充品，包括四种口味的素食蛋白粉、多种维生素、骨骼和皮肤健康保健品。【原文信息】 D2C nutrition brand Earthful raises ₹3.3 cr in pre-seed funding 2024-01-26 , The Hindu Businessline 感谢本期日报的审核者：RZN，aluba，NL，Johnson，九卿臣，圆圈儿，Zhonghua，Richard 点击阅读过去10天的日报： 0202 | 流感肆虐，高分Cell子刊揭示肠菌如何影响呼吸道感染 0201 | 1月，最值得看的40篇肠道健康文献！ 0131 | 今日Cell：于君/沈祖尧团队发现新的促胃癌菌 0130 | 炎症性肠病如何进行营养治疗？胃肠病学顶刊发出最新专家建议 0129 | 国内团队24页综述详解瘤内菌群——肿瘤研究新前沿 0128 | 32分综述详解：饮食如何通过菌群代谢物影响免疫？ 0127 | 今日Cell双发聚焦肠道！揭示肠道免疫和肠肝轴新机制 0126 | 改善心衰，肠菌代谢物提供治疗新思路 0125 | 高糖高油食品如何"绑架"大脑？Cell子刊揭示肠脑机制 0124 | 郝海平等Cell子刊：精神压力如何伤肠道？揭晓肠菌介导的脑-肠轴机制

临床结果

2023-11-01

·GlobeNewswire-Health Care

Research Funded by Bay Area Lyme Foundation Identifies New Investigational Therapy Regimen Capable of Irreversibly Damaging Lyme Bacteria in Laboratory Tests

Inspired by research that targets cancer tumors, these new findings in Lyme could also lead to novel R&D strategies for other diseasesPORTOLA VALLEY, Calif., Nov. 01, 2023 (GLOBE NEWSWIRE) -- Bay Area Lyme Foundation, a leading sponsor of Lyme disease research in the US, today announced the development of a potential new drug, HS-291, that targets and destroys Borrelia burgdorferi, the bacterium that causes Lyme disease. Published in the peer-reviewed journal Cell Chemical Biology, this laboratory study represents a novel paradigm shift for anti-microbial treatment research as it is the first to target and inhibit Borrelia burgdorferi HtpG (high temperature protein G), a specific type of enzyme within the bacteria that causes Lyme disease— a condition affecting nearly 500,000 new patients annually. The research was conducted at Duke University School of Medicine, with collaboration from University of North Carolina, Tulane University, and Stanford University, and HS-291 is currently in preclinical stage testing at UC Davis. “As Lyme disease is currently treated with broad spectrum antibiotics and there are no targeted treatments, we are particularly excited about this discovery, and hopeful that our novel Lyme disease therapeutic HS-291 will specifically destroy the Lyme bacterium without off-target effects or antibiotic resistance,” said Timothy Haystead, PhD, professor of Pharmacology and Cancer Biology, Duke University School of Medicine, Bay Area Lyme Foundation grantee, and co-lead author. “This research has been an incredible opportunity to leverage knowledge from oncology to Lyme disease in order to design an investigational therapeutic that could one day benefit hundreds of thousands of patients with Lyme.” This new discovery has implications beyond Lyme disease as it demonstrates that using the drug HS-291 to deliver cellular toxins to HtpG, a type of non-essential enzyme that assists the folding or unfolding of large and complex proteins, greatly expands what can be considered druggable within any pathogen and opens a whole new area of infectious disease research. When activated, HS-291, an inhibitor of HtpG tethered to the photoactive cellular toxin verteporfin, causes discrete protein modifications, which wreaks havoc on the Lyme disease bacterium’s DNA. This impacts multiple processes including nucleoid collapse and cell wall disruptions. A single dose of HS-291, when activated by light, irreversibly damages Borrelia proteins in close proximity of Bb HtpG in vitro. “Antibiotics used to treat Lyme disease do not always work for all patients, which causes many to suffer for years with extreme symptoms including neurocognitive issues, disabling fatigue and sleep disruption,” said Linda Giampa, executive director of the Bay Area Lyme Foundation. “Bay Area Lyme concentrates on funding innovative research, including projects where knowledge can be deployed from other areas of medicine. We hope that this discovery will inspire others to join us in investing in impactful, translational research to bring relief to patients.” This research was made possible by a 2020 Bay Area Lyme Foundation grant of more than $2 million to Duke University School of Medicine in honor of Neil Spector, MD, a renowned oncologist who passed away from complications of Lyme disease that had been misdiagnosed for years. He encouraged scientists to take cancer staging techniques and immunotherapy learnings from oncology and apply them to Lyme research. Dr. Spector was the Sandra Coates associate professor in the Duke University Department of Medicine and also served on Bay Area Lyme Foundation’s Scientific Advisory Board. Haystead and Spector joined forces as Spector sought to leverage his knowledge of oncology to help better understand Lyme disease, and Haystead’s research is focused on the use of chemical biology approaches to define novel drug targets focused on the treatment of hypertension, obesity, cancer, inflammatory and infectious disease. Bay Area Lyme Foundation also funded University of North Carolina collaborator, Matt Redinbo, PhD’s research on HS-291; his lab’s crystallography work was instrumental in the discovery process. Bay Area Lyme Foundation’s research grant program was made possible by the support from the Fairbairn Family, the Younger Family Fund, and Project Lyme. About Lyme disease The most common vector-borne infectious disease in the US, Lyme disease is a potentially disabling infection caused by bacteria transmitted through the bite of an infected tick to people and pets, and may also be passed from a pregnant mother to her unborn baby. If caught early, most cases of Lyme disease can be effectively treated, but it is commonly misdiagnosed due to lack of awareness and inaccurate diagnostic tests. There are approximately 500,000 new cases of Lyme disease each year, according to statistics released in 2018 by the CDC. As a result of the difficulty in diagnosing and treating Lyme disease, up to two million Americans may be suffering from the impact of its debilitating long-term symptoms and complications, according to Bay Area Lyme Foundation estimates. About Bay Area Lyme Foundation Bay Area Lyme Foundation, a national organization committed to making Lyme disease easy to diagnose and simple to cure, is the leading public not-for-profit sponsor of innovative Lyme disease research in the US. A 501c3 organization based in Silicon Valley, Bay Area Lyme Foundation collaborates with world-class scientists and institutions to accelerate medical breakthroughs for Lyme disease. It is also dedicated to providing reliable, fact-based information so that prevention and the importance of early treatment are common knowledge. A pivotal donation from The LaureL STEM FUND covers overhead costs and allows for 100% of all donor contributions to the Bay Area Lyme Foundation to go directly to research and prevention programs. For more information about Lyme disease or to get involved, visit www.bayarealyme.org or call us at 650-530-2439. Media contact:Tara DiMiliaPhone: 908-369-7168Tara.DiMilia@tmstrat.com

100 项与维替泊芬相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D01162	维替泊芬	S01LA01	DB00460

研发状态

批准上市

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
组织胞浆菌病	美国	Bausch + Lomb Ireland Ltd.	2008-06-30
变性近视	欧盟	CHEPLAPHARM Arzneimittel GmbH	2000-07-27
变性近视	冰岛	CHEPLAPHARM Arzneimittel GmbH	2000-07-27
变性近视	列支敦士登	CHEPLAPHARM Arzneimittel GmbH	2000-07-27
变性近视	挪威	CHEPLAPHARM Arzneimittel GmbH	2000-07-27
湿性年龄相关性黄斑变性	欧盟	CHEPLAPHARM Arzneimittel GmbH	2000-07-27
湿性年龄相关性黄斑变性	冰岛	CHEPLAPHARM Arzneimittel GmbH	2000-07-27
湿性年龄相关性黄斑变性	列支敦士登	CHEPLAPHARM Arzneimittel GmbH	2000-07-27
湿性年龄相关性黄斑变性	挪威	CHEPLAPHARM Arzneimittel GmbH	2000-07-27
年龄相关性黄斑变性	美国	Bausch + Lomb Ireland Ltd.	2000-04-12
脉络膜新生血管	美国	Bausch + Lomb Ireland Ltd.	2000-04-12

未上市

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
多发性基底细胞癌	临床3期	-	QLT, Inc.	2002-11-19
多发性基底细胞癌	临床3期	-	Novartis AG	2002-11-19
基底细胞痣综合征	临床3期	-	QLT, Inc.	2002-11-19
基底细胞痣综合征	临床3期	-	Novartis AG	2002-11-19
局限性前列腺癌	临床2期	美国	SpectraCure AB	2025-01-06
中心性浆液性脉络膜视网膜病变	临床2期	韩国	Novartis Korea Ltd.	2009-07-01
皮肤黑色素瘤	临床2期	美国	QLT, Inc.	2000-10-01

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临床结果

适应症

分期

评价

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


EURETINA2024	N/A	脉络膜出血	-	Photodynamic therapy (PDT)	齋網糧齋築壓選夢膚艱(蓋壓艱襯選鏇顧繭獵壓) = No irreducible retinal detachment or globe loss were described, while four patients developed chronic neurosensory detachment 鹹夢願鹹觸範襯襯夢艱 (廠遞夢襯顧範鑰膚淵觸 ) 更多	-	2024-09-19
EURETINA2022	N/A	中心性浆液性脉络膜视网膜病变	-	Patients with verteporfin shortage	鹽鏇襯壓範糧築鬱築餘(壓網夢願構築選窪範憲) = There was a significant impact with visual loss in patients with CSCR due to the shortage of verteporfin with the inability to undergo PDT 顧艱憲繭廠壓壓鹽鏇夢 (淵艱構遞構憲鏇鏇鹽繭 )	-	2022-09-01
				Patients without verteporfin shortage
ITVT-02 (SNO2021)	临床1/2期	胶质母细胞瘤 EGFR \| PI3K	-	Verteporfin	築夢鏇醖鹹壓鏇鑰衊遞(製窪膚淵窪鹹襯壓衊壓) = 廠範淵選鬱獵餘憲積鏇壓齋鬱壓夢範積淵壓鑰 (選憲鑰夢鹹糧願襯簾壓 )	-	2021-11-12
				Verteporfin Photodynamic Therapy	築夢鏇醖鹹壓鏇鑰衊遞(製窪膚淵窪鹹襯壓衊壓) = 餘蓋糧鏇憲憲製鑰鏇窪壓齋鬱壓夢範積淵壓鑰 (選憲鑰夢鹹糧願襯簾壓 )
NCT03033225 (Pubmed \| Gastrointest Endosc)	临床2期	胰腺癌	8	EUS-guided verteporfin PDT	窪憲顧憲築壓選衊網醖(蓋淵淵淵艱壓築製糧夢) = 衊觸遞鏇窪廠鏇顧壓繭簾顧膚鏇淵衊鏇膚襯製 (鑰選積鹽選淵艱蓋襯網 ) 更多	积极	2021-02-26
AAO2020	N/A	葡萄膜黑色素瘤	26	Primary PDT with Verteporfin	繭網鹹繭艱觸淵網築廠(鹽淵餘鏇齋觸夢糧鏇淵) = 鹽艱築夢壓艱憲餘鹹鹹積夢觸願醖醖淵糧遞齋 (淵製網構鬱鏇觸鏇壓鹽 )	不佳	2020-11-13
NCT01846273 (EVEREST II, CTgov)	临床4期	年龄相关性黄斑变性 \| 息肉状脉络膜血管病	321	Ranibizumab (Ranibizumab 0.5 mg + vPDT)	醖糧觸憲壓網憲獵齋繭(繭構築積壓膚鹹襯遞觸) = 鏇鹽範簾膚鹽衊齋蓋繭製膚觸遞鏇壓壓糧醖餘 (廠襯遞鹹構鏇鏇衊廠網, 1.00) 更多	-	2019-06-07
				Ranibizumab (Ranibizumab 0.5 mg)	醖糧觸憲壓網憲獵齋繭(繭構築積壓膚鹹襯遞觸) = 鹽顧網鹹餘廠觸襯窪膚製膚觸遞鏇壓壓糧醖餘 (廠襯遞鹹構鏇鏇衊廠網, 1.06) 更多
OR02-4 (ENDO2019)	N/A	肾上腺皮质肿瘤 \| 肾上腺肿瘤辅助 YAP1	-	Verteporfin	簾積艱鬱壓齋艱餘餘鏇(築積餘膚襯鏇蓋選製淵) = 遞餘選鹹廠膚顧餘網齋鹽鑰鏇遞選醖衊餘簾憲 (淵憲糧齋鹹衊顧獵遞膚 ) 更多	积极	2019-04-30
EVEREST II (APAO2018)	N/A	息肉	322	ranibizumab 0.5 mg plus verteporfin photodynamic therapy (vPDT)	獵繭觸餘願鹹廠遞築鬱(膚夢鬱憲選膚構憲夢觸) = 願憲艱餘鏇餘網齋範網鏇憲廠夢鹽觸繭製積選 (積齋襯遞簾範製廠膚壓 )	积极	2018-02-08
				ranibizumab 0.5 mg monotherapy	獵繭觸餘願鹹廠遞築鬱(膚夢鬱憲選膚構憲夢觸) = 鹽鬱醖鹹鏇範遞憲齋壓鏇憲廠夢鹽觸繭製積選 (積齋襯遞簾範製廠膚壓 )
NCT01846273 (Pubmed \| JAMA Ophthalmol) 人工标引	临床4期	息肉状脉络膜血管病	322	verteporfin photodynamic therapy +Ranibizumab	觸餘製選遞衊築網遞鏇(艱夢製構艱艱繭蓋製糧) = 願積觸夢鹽夢窪鹹顧築鬱艱繭願醖簾鹹積獵簾 (鑰醖膚鬱繭鏇觸鬱膚製 ) 更多	积极	2017-11-01
				Ranibizumab	觸餘製選遞衊築網遞鏇(艱夢製構艱艱繭蓋製糧) = 網顧齋築鹹簾憲觸顧網鬱艱繭願醖簾鹹積獵簾 (鑰醖膚鬱繭鏇觸鬱膚製 ) 更多
ARVO2017 人工标引	N/A	湿性年龄相关性黄斑变性	16	Adjunctive Indocyanine Green Angiography-Directed Verteporfin Photodynamic Therapy	觸觸糧鏇顧衊膚衊衊醖(繭鑰顧齋鹹醖製範艱膚) = 糧醖遞築齋餘繭餘鏇繭艱糧淵淵願顧衊夢簾壓 (窪蓋憲積鑰鑰窪鹽壓糧 )	积极	2017-06-01