Over the past decades, healthcare systems face significant challenges to meet the needs of an aging population due to progressive debility, functional decline and chronic diseases development. While there is a growing appreciation of the potential impact of mTOR inhibitors on slowing aging processes, preventing chronic disease and prolonging healthy lifespan, a major challenge in developing clinical trials to establish the clinical efficacy of mTOR inhibitors is the absence of pharmacokinetics (PK) and pharmacodynamics (PD) data in older adults. The proposed study will provide the foundation for future clinical trials assessing the role of mTOR inhibitors on aging related indications

开始日期2025-05-01

申办/合作机构

The University of Texas Southwestern Medical Center

[+2]

NCT06752694

/ Not yet recruiting临床2期

Ruxolitinib Based GVHD Prophylaxis Regimen for Older Adults Receiving Non-ATG Containing Non-Myeloablative Hematopoietic Cell Transplantation for Acquired Aplastic Anemia

This phase II trial tests how well a ruxolitinib-based graft versus host disease (GVHD) prevention (prophylaxis) regimen works before, during, and after bone marrow/stem cell transplantation (hematopoietic cell transplantation [HCT]) in patients with acquired aplastic anemia. Acquired aplastic anemia (AA) is a condition in which the bone marrow is unable to produce blood cells. Affected patients typically present with infections due to abnormally low number of neutrophils, bleeding due to low platelet count, and/or fatigue due to a lower-than-normal number of red blood cells (anemia). Its incidence varies with age, occurring most frequently in patients aged 2-5 years, 20-25 years, and 55 years and older. Treatment of AA includes either immunosuppressive therapy (IST) or bone marrow/stem cell transplantation (HCT) with first-line therapy in younger adults often being HCT, while adults over 40 still frequently trial IST first due to the morbidity and mortality concerns with HCT. GVHD is a common complication after donor stem cell transplantation, resulting from donor immune cells recognizing recipients' cells and attacking them. Ruxolitinib, a drug in a class of oral medications called JAK inhibitors has been approved for the treatment of acute and chronic GVHD. It has also been shown to decrease GVHD when used in the prevention setting in patients with myelofibrosis. The current study aims to assess whether adding ruxolitinib to a standard GVHD prevention regimen may reduce the risk of Grade II-IV acute and chronic GVHD after bone marrow/stem cell transplantation in older patients with acquired aplastic anemia.

开始日期2025-02-15

申办/合作机构

Fred Hutchinson Cancer Center

[+1]

NCT04370795

/ Enrolling by invitation临床1/2期IIT

Matched Related and Unrelated Donor Stem Cell Transplantation for Severe Combined Immune Deficiency (SCID): Busulfan-based Conditioning With h-ATG, Radiation, and Sirolimus

Background:
Severe combined immune deficiency (SCID) is a group of conditions where the immune system does not work properly. The only cure for most SCIDs is a stem cell transplant (getting cells from a donor). These transplants can have serious complications. Before the transplant, people often get high doses of drugs and radiation to prepare the body to accept the cells from the donor. Researchers want to see if low doses of drugs alone without radiation work just as well as low doses of drugs with radiation for SCID patients getting stem cell transplants.
Objective:
To test a set of drugs with or without radiation given before a stem cell transplant.
Eligibility:
People ages 3-40 who have SCID and who have a stem cell donor - either related or unrelated.
Design:
Participants will be admitted to the hospital 10 days before transplant. They will undergo:
medical history
medication review
physical exam
blood and urine tests (may include a 24-hour urine collection)
heart, lung, and breathing tests
imaging scans
bone marrow sample
nutrition assessment
dental exam
eye exam
meeting with a social worker.
Participants will get a plastic port called a central line. It is a hollow tube that is placed in the upper chest. It will be used to give medicines and take blood.
All participants will take chemotherapy drugs. Some will get radiation.
Participants will have a stem cell transplant. They will get the cells as an infusion through their central line. They will stay in the hospital for 30 days after transplant.
Participants must stay within 1 hour of NIH for 3 months after transplant. During this time, they will have follow-up visits at NIH at least once a week. Then they will have follow-up visits once or twice a year for 5-6 years.

开始日期2025-01-22

申办/合作机构

National Institute of Allergy & Infectious Diseases

100 项与西罗莫司相关的临床结果

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100 项与西罗莫司相关的转化医学

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100 项与西罗莫司相关的专利（医药）

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25,765

项与西罗莫司相关的文献（医药）

2025-12-01·Journal of Clinical Immunology

Report of the Italian Cohort with Activated Phosphoinositide 3-Kinase δ Syndrome in the Target Therapy Era

Article

作者: Montin, Davide ; De Rosa, Antonio ; Ricci, Silvia ; Marinoni, Maddalena ; Trizzino, Antonino ; Moratti, Mattia ; Rivalta, Beatrice ; Zecca, Marco ; Tommasini, Alberto ; Marzollo, Antonio ; Barzaghi, Federica ; Baselli, Lucia Augusta ; Badolato, Raffaele ; Cancrini, Caterina ; Conti, Francesca ; Lougaris, Vassilios ; Lodi, Lorenzo ; Giardino, Giuliana ; Pignata, Claudio ; Panza, Giuseppina ; Costagliola, Giorgio ; Martire, Baldassarre ; Chinello, Matteo ; Milito, Cinzia

BACKGROUNDActivated Phosphoinositide 3-Kinase (PI3K) δ Syndrome (APDS), an inborn error of immunity due to upregulation of the PI3K pathway, leads to recurrent infections and immune dysregulation (lymphoproliferation and autoimmunity).METHODSClinical and genetic data of 28 APDS patients from 25 unrelated families were collected from fifteen Italian centers.RESULTSPatients were genetically confirmed with APDS-1 (n = 20) or APDS-2 (n = 8), with pathogenic mutations in the PIK3CD or PIK3R1 genes. The median age at diagnosis was 15.5 years, with a median follow-up of 74 months (range 6-384). The main presenting symptoms were respiratory tract infections alone (57%) or associated with lymphoproliferation (17%). Later, non-clonal lymphoproliferation was the leading clinical sign (86%), followed by respiratory infections (79%) and gastrointestinal complications (43%). Malignant lymphoproliferative disorders, all EBV-encoding RNA (EBER)-positive at the histological analysis, occurred in 14% of patients aged 17-19 years, highlighting the role of EBV in lymphomagenesis in this disorder. Diffuse large B-cell lymphoma was the most frequent. Immunological work-up revealed combined T/B cell abnormalities in most patients. Treatment strategies included immunosuppression and PI3K/Akt/mTOR inhibitor therapy. Rapamycin, employed in 36% of patients, showed efficacy in controlling lymphoproliferation, while selective PI3Kδ inhibitor leniolisib, administered in 32% of patients, was beneficial on both infections and immune dysregulation. Additionally, three patients underwent successful HSCT due to recurrent infections despite ongoing prophylaxis or lymphoproliferation poorly responsive to Rapamycin.CONCLUSIONSThis study underscores the clinical heterogeneity and challenging diagnosis of APDS, highlighting the importance of multidisciplinary management tailored to individual needs and further supporting leniolisib efficacy.

2025-12-01·Journal of Clinical Immunology

Novel Compound Heterozygous Variants in the FAS Gene Lead to Fetal Onset of Autoimmune Lymphoproliferative Syndrome (ALPS)

Article

作者: Yu, Meiping ; Wu, Bingbing ; Zhu, Li ; Chen, Yao ; Li, Qifan ; Wang, Chenghao ; Qian, Feng ; Zhou, Weitao ; Liu, Luyao ; Wu, Qi ; Hui, Xiaoying ; Zhou, Qinhua ; Hou, Jia ; Wang, Xiaochuan ; Sun, Bijun ; Wang, Ying ; Sun, Jinqiao ; Ying, Wenjing ; Wang, Wenjie

OBJECTIVEFAS gene defects lead to autoimmune lymphoproliferative syndrome (ALPS), which is often inherited in an autosomal dominant and rarely in an autosomal recessive manner. We report a case of a newborn girl with novel compound heterozygous variants in FAS and reveal the underlying mechanism.METHODSWhole-exome sequencing (WES) was used to identify pathogenic variants. Multiparametric flow cytometry analysis, phosflow analysis, and FAS-induced apoptosis assays were used to explore the effects of the variants on FAS expression, apoptosis, and immunophenotype. The HEK293T cells were used to assess the impact of the variants on protein expression and FAS-induced apoptosis.RESULTSThe patient was born with hepatosplenomegaly, anemia, and thrombocytopenia. She also experienced COVID-19, rotavirus infection, herpes simplex virus infection, and severe pneumonia. The proportion of double-negative T cells (DNTs) was significantly elevated. Novel FAS compound heterozygous variants c.310T > A (p.C104S) and c.702_704del (p.T235del) were identified. The apoptotic ability of T cells was defective, and FAS expression on the surface of T cells was deficient. The T235del variant decreased FAS expression, and the C104S protein remained in the endoplasmic reticulum (ER) and could not translocate to the cell surface. Both mutations resulted in loss-of-function in terms of FAS-induced apoptosis in HEK293T cells. The DNTs were mainly terminally differentiated T (TEMRA) and CD45RA⁺HLA-DR⁺, with high expression of CD85j, PD-1, and CD57. The percentage of Th1, Tfh, and autoreactive B cells were significantly increased in the patient. The abnormal immunophenotyping was partially attenuated by sirolimus treatment.CONCLUSIONSWe identified two variants that significantly affect FAS expression or localization, leading to early disease onset of in the fetus. Abnormalities in the mTOR pathway are associated with a favorable response to sirolimus.

2025-12-01·Molecular Biology Reports

Rapamycin and Niacin combination induces apoptosis and cell cycle arrest through autophagy activation on acute myeloid leukemia cells

Article

作者: Akçok, İsmail ; Gencer Akçok, Emel Başak ; Subay, Lale Beril

BACKGROUNDAcute myeloid leukemia (AML) is a heterogeneous hematological malignancy caused by disorders in stem cell differentiation and excessive proliferation resulting in clonal expansion of dysfunctional cells called myeloid blasts. The combination of chemotherapeutic agents with natural product-based molecules is promising in the treatment of AML. In this study, we aim to investigate the anti-cancer effect of Rapamycin and Niacin combination on THP-1 and NB4 AML cell lines.METHODS AND RESULTSThe anti-proliferative effects of Rapamycin and Niacin were determined by MTT cell viability assay in a dose- and time-dependent manner. The combination indexes were calculated by isobologram analysis. Furthermore, apoptosis was investigated by Annexin-V/Propidium Iodide(PI) double staining and cell cycle distribution was measured by PI staining. The expression levels of autophagy-related proteins were detected by western blotting. The combination of Rapamycin and Niacin synergistically decreased cell viability of AML cell lines. The combination treatment induced the apoptotic cell population of THP-1 and NB4 by 4.9-fold and 7.3-fold, respectively. In THP-1 cells, the cell cycle was arrested at the G2/M phase by 10% whereas the NB4 cells were accumulated at the G0/G1 phase. The combination treatment decreased Akt and p-Akt expression. Besides, the ATG7 expression was reduced by combination treatment on THP-1 cells. Similarly, the ATG5 level was downregulated in NB4 cells. The level of LC3B-II/LC3B-I, which is an indicator of autophagy flux, was upregulated in THP-1 and NB4 cells.CONCLUSIONAlthough further studies are required, the combination of Rapamycin and Niacin combats cell proliferation by inducing cellular apoptosis, cell cycle arrest and autophagy activation.

592

项与西罗莫司相关的新闻（医药）

2025-01-20

·PRNewswire

SIRPAD - WORLD'S LARGEST RCT INVESTIGATING MAGICTOUCH PTA SIROLIMUS COATED BALLOON COMPLETES PATIENT ENROLMENT

TAMPA, Fla., Jan. 20, 2025 /PRNewswire/ -- Concept Medical Inc. (CMI), a global leader in innovative drug-delivery technology across vascular interventions, proudly announces the successful completion of patient enrolment in the SirPAD Trial, with over 1,250 patients now enrolled. Continue Reading Concept Medical proudly announces that over 1,250 patients have successfully enrolled in the SirPAD Trial. The SirPAD (Major adverse limb events in patients with femoro-popliteal and below-the-knee peripheral arterial disease treated with either sirolimus-coated or uncoated balloon) randomised controlled trial is the world's largest study evaluating the treatment of Peripheral Artery Disease (PAD) using the MagicTouch PTA Sirolimus Coated Balloon (Concept Medical Inc.) versus uncoated balloons and one of the largest device studies ever performed for PAD. More than 1,250 patients have been enrolled and will be followed up at 12 months to assess the primary outcome of major adverse limb events (MALE). Final results are expected by Q1-Q2 2026. SirPAD is an investigator-initiated, multi-centre, randomized, open-label trial that aims to determine whether the MagicTouch PTA sirolimus-coated balloon is non-inferior to plain old balloon angioplasty (POBA). The trial will also explore the potential for superiority. Principal Investigators Prof. Dr. med. Nils Kucher and Prof. Dr. med. Stefano Barco, from the University Hospital Zurich, who leads this ambitious study, said, "Sirolimus-coated balloons represent a promising technology in treating symptomatic PAD, supported by evidence primarily from studies with surrogate endpoints. While trials like SIRONA and ongoing studies such as SirPAD are expanding knowledge, robust data from randomized controlled trials with clinical outcomes are needed to guide treatment and shape future guidelines. This result, having been able to randomize so many patients in a PAD device study, is unprecedented and we are very thankful to all investigators and patients for having supported the trial" on achieving the significant milestone of enrolling 1,250+ patients and successfully completing this phase of the study. With sirolimus-coated balloons gathering global momentum and MagicTouch PTA at the forefront, SirPAD is a crucial milestone in expanding the evidence for PAD treatment options. While patients with PAD have limited therapeutic avenues, SirPAD aims to bolster data supporting sirolimus-coated balloon technology. This large RCT was critical to evaluating treatment alternatives, and its year-long follow-up will provide much-anticipated answers. "At Concept Medical, we are committed to generating robust clinical evidence to support our innovative technologies. Following the successful completion of SIRONA—the largest superficial femoral artery (SFA) trial comparing our device against paclitaxel drug-coated balloons (DCBs), which demonstrated non-inferiority—we are proud to announce the completion of enrollment for another historical milestone: the largest randomised clinical trial for peripheral all-comers patients, completed enrolment of 1250+ patients. With this achievement, Concept Medical continues to lead the way with the most extensive and advanced clinical trial program in the field, reinforcing our position as a global pioneer in patient-centric vascular innovations," said Dr. Manish Doshi, Founder & Managing Director of Concept Medical. About Concept Medical Concept Medical Inc., headquartered in Tampa, Florida, has a global presence and is dedicated to enhancing patient care through cutting-edge research and development of drug-delivery technologies. Its proprietary platforms are designed to deliver pharmaceutical agents across vascular luminal surfaces with unparalleled precision. Concept Medical is the developer of the MagicTouch family of Sirolimus Coated Balloons (SCBs)—the world's first and most utilized SCB technology—well recognized for its versatility and efficacy in treating coronary and peripheral artery disease. The revolutionary MagicTouch and Abluminus product lines have been used to treat over a million patients globally, setting a new standard for vascular therapy. For more information, please visit Photo: Logo: SOURCE Concept Medical WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

临床研究

2025-01-20

·药物分子设计

返老还童，OpenAI做到了？首个逆龄AI将登场，人类寿命可延长10年

第一性原理计算解决50年悬而未决难题：半导体中铜为何扩散更快？ Ab initio及第一性原理入门参考书介绍《海贼王》告诉你，做科研为什么不能闭门造车…… 来自公众号：新智元本文以传播知识为目的，如有侵权请后台联系我们，我们将在第一时间删除。新智元报道编辑：KingHZ 桃子【新智元导读】逆龄AI真的被OpenAI实现了！新模型GPT-4b micro通过蛋白质重编程技术，有望将人类寿命延长十年。超级智能要来了？人类「长生不老」有希望了？外媒称，OpenAI开发出了首款用于「长寿」的AI模型 ——GPT-4b micro，预计将人类寿命延长10年。这一突破性成果，可能会彻底改变人类对衰老的认知。与其他预测蛋白质结构的AI不同，GPT-4b micro更像是一位「蛋白质社交专家」，能够精准预测不同蛋白质之间的互动方式。利用这个独特的优势，科学家们成功设计了两种关键的「山中因子」蛋白质。这些蛋白质在胎儿发育中起着关键作用，堪称生命的「时光机」——让成熟的细胞重返年轻态，具有无限可能的干细胞。不仅如此，这次AI辅助设计的新蛋白质，展现出惊人的效率，将干细胞生产效率提高了多达50倍！这意味着什么？简单来说，这些经过AI优化的蛋白质，在将普通细胞转化为干细胞的能力上，远超自然界的版本。值得一提的是，新模型是OpenAI与Retro Biosciences合作的成果，奥特曼曾向这家公司投入了1.8亿美元。目前，该模型不能公开使用，也没有明确时间表什么时候可以商用。更令人兴奋的是，它将为治疗多种疾病开辟了新的可能性。那些曾被认为无法治愈的疾病，比如糖尿病、心脏病等，可能找到治愈的希望。研究预印本随后发布 2024年，谷歌DeepMind的哈萨比斯，因AlphaFold获得了诺贝尔奖。如此看来，如果继续突破下去，OpenAI也有可能获得诺贝尔奖。逆龄AI GPT-4b micro，或延长10年寿命 GPT-4b micro代表了AI在生物技术领域应用的一个重大进步，专门为蛋白质重编程，而非单纯的结构预测而设计。与AlphaFold等同类模型不同，AlphaFold主要集中在蛋白质空间结构的预测，而GPT-4b micro则通过积极重编程蛋白质，特别关注Yamanaka因子（山中因子）的作用。 GPT-4b micro可以主动修改蛋白质结构，达到预期的效果。那么，为什么要对蛋白质重编程？这里有两个原因：首先，能够更快获得更多的iPSCs（诱导多能干细胞），便意味着减少了iPSC扩增所需的天数，缩短获得细胞的时间。其次，这能够降低细胞培养基的使用量和总时间，这些都是细胞治疗成本的关键因素。 Yamanaka因子至关重要，因为能够将成年细胞重置为多能状态，并分化为几乎所有类型的细胞，因而在再生医学和延长人类寿命的治疗领域中，具有巨大的潜力。而通过靶向这些蛋白质，GPT-4b micro为创建更有效的再生治疗方案提供了可能，有望将人类寿命延长十年。实际应用：器官创造、细胞替代疗法 GPT-4b micro在器官创造和细胞替代疗法中的实际应用标志着再生医学的重大进展。 GPT-4b micro大大提高了各种细胞的生产效率，有望解决器官移植中关键的短缺问题，并为多种退行性疾病提供创新治疗方案。从实际角度来看，这种AI驱动的方法可能为器官移植领域带来突破性变化。科学家有可能为每位患者量身定制器官，大大减少排斥反应的风险，并提高移植的成功率。此外，还可能开发细胞替代疗法，用于治疗如帕金森病、糖尿病和心脏病等目前治疗效果有限的疾病。除了单一应用外，这项技术可能会提供大规模组织工程的新策略，彻底改变再生医学领域。这将有助于再生整个器官，或是修复器官中的特定关键组织，这些组织可能因衰老、疾病或损伤而受损。因此，AI驱动的蛋白质工程的进展有望加速再生医学的研究步伐，促进医学、生物学与AI领域的跨界合作。随着技术的不断成熟，它有可能从根本上改变治疗方式，提升生活质量，并可能引领在寿命延长和衰老相关疾病治疗上的范式转变。押注三种主流抗衰老技术此前，早有报道称，Sam Altman一直在押注一项雄心勃勃的秘密计划——延长人类寿命。为此，他找到了哈佛、MIT、加州理工学院联合培养的科学家Joe Betts-LaCroix。这位曾经开发出世界上最小个人电脑的天才，多年来一直致力于推动长寿领域的深度研究。表面上看，进军生物科技似乎与奥特曼的AI事业相去甚远，但深入了解就会发现，这其实他未来主义世界观的自然延伸。奥特曼早已为可能发生的末日灾难，准备了充足的武器、黄金、和地产。而从AI到核聚变能源，再到延长生命，奥特曼相信，自己有能力去解决这些关乎人类未来的重大课题。虽然让人类寿命延长10年，听起来像是科幻小说的情节。但在如今AI飞速发展的今天，或许这个目标已经不再遥不可及。在距离OpenAI旧金山总部南部约30英里处，Retro Biosciences的总部悄然坐落于此。自2021年7月，启动了集装箱实验以来，Retro将研究方向分为三个领域。细胞自噬（Autophagy）细胞自噬是细胞内的一种自我消化过程，可以清除受损的细胞器和蛋白质。目前，最接近于能够提高细胞效率的药物是雷帕霉素（rapamycin）或二甲双胍（metformin）。这两种现有药物（分别用于肾移植和糖尿病治疗）在延长寿命方面显示出了希望，一些生物黑客已经在进行非常规使用，将其作为自行干预衰老的手段。但到目前为止，还没有任何药物能够直接、明确且正式地针对这个细胞「房屋清理」问题。细胞重编程这是当今长寿科学领域最热门的想法之一。这项技术在操作上也很棘手。其基本理念是使用四种著名的「山中因子」将老化细胞「重编程」到稍微年轻的状态，使其重新表现出年轻细胞的特征。但在实验中，要以一种不会潜在导致癌症或其他健康问题的方式完成这种重塑过程是很困难的。血浆疗法这听起来最像吸血鬼中情节的概念。 Betts-LaCroix指出，通过用生理盐水稀释小鼠的血浆进行的「返老还童」研究显示出了很有希望的结果。这似乎比单纯给老年小鼠输入年轻血液更有效。在接受血浆稀释治疗的衰老小鼠中，多种与年龄相关的问题都得到了改善。合作介绍 OpenAI的研究员John Hallman、Aaron Jaech，以及来自Retro的Rico Meinl，主导了新模型GPT-4b micro的开发。从左至右：John Hallman、Aaron Jaech、Rico Meinl 合作的具体目标是推动器官创造和细胞替代疗法的技术进步。 OpenAI与Retro Biosciences将研究集中在山中因子，希望彻底革新再生医学，提供更高效的方式，培育定制器官并替换细胞。 GPT-4b micro与其他AI模型的关键特点之一，是它不仅能够预测蛋白质结构，还能重新编程蛋白质。这一点至关重要，因为它为理解和操控生物过程开辟了新的方向。此外，这项技术的应用领域极为广泛，能够在器官创造和细胞替代疗法等多个领域提供潜在的解决方案。目前，该项目没有设定完成的具体时间表。然而，双方承诺将与科学界分享研究成果，确保从这一项目中获得的知识为科学界提供更广泛的理解与进展。未来与影响专家意见 Retro Biosciences顾问、哈佛大学教授Vadim Gladyshev，对GPT-4b micro的潜力表示乐观。他指出，在不同的细胞类型和物种中，原有技术往往出现不一致性，而新AI模型能提供高效的重编程过程。他认为，AI在干细胞生产中的潜力巨大。 Retro Biosciences的首席执行官Joe Betts-Lacroix，表示新的技术是生物界的AlphaGo，指出即使在对模型原理没有完全理解之前，也能取得其令人瞩目的成果。Betts-Lacroix强调了GPT-4b micro在蛋白质重编程中的变革性能力，进一步巩固了在推动长寿研究中的重要作用。尽管GPT-4b micro的前景非常广阔，生物伦理学家们却提出了严重的担忧。根据《The Conversation》中的专家观点，延长人类寿命可能导致代际更替的减少，从而减缓社会进步。随着生命延长技术的出现，可能会造成「生物鸿沟」，即富人可以负担这些技术，从中受益，而穷人则不然。关于GPT-4b micro的讨论不仅关于科学潜力，也是社会责任的问题。AI与「长生不老」 AI与长寿科学，正以前所未有的方式交织在一起，推动着技术和生物学的发展。与OpenAI的开创性努力相呼应，其他领域也取得了显著进展。 Alphabet的Isomorphic Labs因在AI驱动的药物发现方面的突破而备受瞩目，成功预测了与衰老相关的蛋白质相互作用。 Calico Life Sciences也在AI设计的细胞清除化合物方面取得了持续进展，为抗击细胞衰老做出了贡献。这些里程碑不仅标志着AI在医疗保健领域的重要作用，也为未来生物研究和治疗发展奠定了基础。重塑人类长寿的旅程充满了希望与挑战。专家认为，AI在生物学研究中的整合提供了前所未有的效率和效果，但其社会影响，尤其是公平性和伦理问题，仍然是需要严肃讨论的领域。随着AI与长寿科学的对话不断推进，围绕监管框架和伦理标准的讨论日益重要。 FDA最近为AI辅助的生物研究制定了指导方针，体现了对新技术挑战的应对。这一监管措施对规范AI驱动的蛋白质和细胞重编程至关重要，预示着未来AI将在医学科学中持续塑造并不断重新定义其边界。公众反应与认知 OpenAI与Retro Biosciences合作延长人类寿命的宣布，引发了多种反应，既有的希望也有怀疑。一方面，支持者将这一倡议视为生物技术的一大突破，热切期待可能带来更健康、更长寿生活的进步。许多网友，对AI解决人类最紧迫健康挑战的潜力表示兴奋。另一方面，这种技术也令人担忧。一些人担心大幅延长人类寿命的伦理影响，例如可能对全球资源和社会结构造成的压力。社交媒体平台上的批评者质疑这些长寿治疗手段的可及性，担心它们最初可能只是富人的特权，可能会加剧现有的社会不平等。以后富人不光在经济上享有特权，还能活得更久。越来越多的人呼吁进行透明的讨论和监管，以确保这些治疗手段的开发和分配都能以合乎伦理和平等的方式进行。此外，AI在延长寿命方面的作用可能会导致社会结构的重大转变，例如退休制度和代际动态。至关重要的是，要平衡这些进步的巨大潜力与积极措施来降低风险，为未来做好准备。参考资料： https://x.com/ArtirKel/status/1880275877927481606 https://www.forbes.com/sites/alexknapp/2025/01/17/the-prototype-openai-and-retro-biosciences-made-an-ai-model-for-bioengineering/ https://www.businessinsider.com/retro-biosciences-sam-altman-longer-lifespan-2024-3 https://opentools.ai/news/openai-and-retro-biosciences-a-bold-leap-in-longevity-with-ai-powered-protein-re-engineering

细胞疗法

2025-01-16

·PRNewswire

Concept Medical Announces First Patient Enrollment in the MAGICAL BTK IDE Trial, Launching its PAD Clinical Program in the United States

TAMPA, Fla., Jan. 16, 2025 /PRNewswire/ -- Concept Medical Inc., a global pioneer in innovative drug delivery technology, is proud to announce the successful enrolment of the first patient in the "MAGICAL BTK" (Randomized Controlled Trial of MagicTouch PTA – Sirolimus Coated Balloon Versus Standard Balloon Angioplasty in the Treatment of Below The Knee arterial disease) U.S. Investigational Device Exemption (IDE) pivotal trial. The first patient was enrolled by Dr. Prakash Krishnan, MD, and his team at the Icahn School of Medicine at Mount Sinai, marking a crucial milestone in the advancement of treatment options for patients suffering from peripheral artery disease (PAD) below the knee. Continue Reading First patient enrollment in MAGICAL-BTK IDE Study in the United States. Following the recent IDE Approval by the U.S. Food and Drug Administration (FDA) for MagicTouch PTA, this enrolment heralds the beginning of Concept Medical's peripheral clinical trial program in the United States. With the ongoing IDE study for MagicTouch SCB in coronary artery disease, Concept Medical is now actively enrolling in two U.S. clinical trials in both coronary and peripheral interventions—an unprecedented achievement aimed at improving patient outcomes and expanding therapeutic choices. "The enrolment of the first patient in the MAGICAL BTK trial represents an important stride toward improving the standard of care for below-the-knee disease in patients with peripheral artery disease," said Dr. Prakash Krishnan, MD, Icahn School of Medicine at Mount Sinai. "Sirolimus-coated balloon technology has the potential to significantly enhance limb salvage and patient quality of life. I am honored to be part of this landmark study and look forward to the meaningful clinical evidence it will generate." The MAGICAL BTK pivotal trial is designed to evaluate the safety and effectiveness of MagicTouch PTA Sirolimus Coated Balloon compared to standard percutaneous transluminal angioplasty (PTA) for the treatment of below-the-knee arterial disease. The primary endpoint, measured at 12 months, is primary patency, ensuring that the trial's findings focus on meaningful clinical outcomes. This global, multi-center study is led by Prof. Sahil Parikh (Chairperson), with a distinguished panel of Principal Investigators including Dr. Eric Secemsky (Beth Israel Deaconess Hospital and Harvard Medical School), Prof. Brian DeRubertis (NY Presbyterian Weill Cornell Medical Center and Cornell University Medical College), Prof. Edward Choke (Northern Heart Hospital), and Prof. Osamu Iida (Osaka Keisatsu Hospital). "The initiation of the MAGICAL BTK Trial heralds a new era in the treatment of patients with chronic limb-threatening ischemia and offers hope to the millions of patients with this condition who are at risk for limb loss because of insufficient arterial circulation in their legs," stated Prof. Sahil Parikh. Sirolimus-coated balloon technology has already reshaped coronary and peripheral artery disease management, with MagicTouch achieving positive results in large-scale trials across Asia and Europe.. Now, with the MAGICAL BTK trial, U.S. patients have the opportunity to benefit from this breakthrough approach. Concurrently, patient enrolment from Asia will broaden the global patient cohort, offering a more comprehensive understanding of the therapy's benefits across diverse populations. "At Concept Medical, our unwavering commitment to innovation and patient care drives us to push the boundaries of what is possible in vascular interventions," said Dr. Manish Doshi (Founder & Managing Director). "The start of MAGICAL BTK further validates our dedication to bringing next-generation, evidence-based solutions to clinicians and patients worldwide. Our goal is to redefine current standards, ensuring better outcomes and improved quality of life for those affected by PAD." With the MAGICAL BTK trial and the MAGICAL SFA IDE trial for superficial femoral artery (SFA) treatment set to begin, Concept Medical is poised to revolutionise endovascular care. The company's distinctive technology platform has already elevated the management of coronary artery disease, and now, by expanding its reach into peripheral interventions, Concept Medical reinforces its leading position in advancing patient-focused, drug-delivery solutions. About Concept Medical Concept Medical Inc., headquartered in Tampa, Florida, has a global presence and is dedicated to enhancing patient care through cutting-edge research and development of drug-delivery technologies. Its proprietary platforms are designed to deliver pharmaceutical agents across vascular luminal surfaces with unparalleled precision. Concept Medical is the developer of the MagicTouch family of Sirolimus Coated Balloons (SCBs)—the world's first and most utilized SCB technology—well recognized for its versatility and efficacy in treating coronary and peripheral artery disease. The revolutionary MagicTouch and Abluminus product lines have been used to treat over a million patients globally, setting a new standard for vascular therapy. For more information, please visit . Photo - Logo - WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

临床研究

100 项与西罗莫司相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D00753	西罗莫司	L04AA10 S01XA23	DB00877

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
血管畸形	日本	Nobelpharma Co., Ltd.	2024-01-18
血管肿瘤	日本	Nobelpharma Co., Ltd.	2024-01-18
血管纤维瘤	欧盟	Pfizer Inc.	2023-05-15
血管纤维瘤	冰岛	Pfizer Inc.	2023-05-15
血管纤维瘤	列支敦士登	Pfizer Inc.	2023-05-15
血管纤维瘤	挪威	Pfizer Inc.	2023-05-15
结节性硬化症	中国	Nobelpharma Co., Ltd.	2023-03-28
结节性硬化相关血管纤维瘤	美国	Nobelpharma Co., Ltd.	2022-03-22
淋巴管扩张	日本	Nobelpharma Co., Ltd.	2021-09-27
淋巴管瘤	日本	Nobelpharma Co., Ltd.	2021-09-27
原发性骨质溶解	日本	Nobelpharma Co., Ltd.	2021-09-27
淋巴管肌瘤病	冰岛	Pfizer Europe MA EEIG	2001-03-13
淋巴管肌瘤病	欧盟	Pfizer Europe MA EEIG	2001-03-13
淋巴管肌瘤病	挪威	Pfizer Europe MA EEIG	2001-03-13
淋巴管肌瘤病	列支敦士登	Pfizer Europe MA EEIG	2001-03-13
肾移植排斥反应	美国	PF Prism CV	1999-09-15

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
Taylor型局灶性皮质发育不良	临床3期	日本	Nobelpharma Co., Ltd.	-
肝移植排斥反应	临床前	法国	Wyeth AB	2002-12-01
肝移植排斥反应	临床前	比利时	Wyeth AB	2002-12-01
肝移植排斥反应	临床前	葡萄牙	Wyeth AB	2002-12-01
肝移植排斥反应	临床前	荷兰	Wyeth AB	2002-12-01
肝移植排斥反应	临床前	瑞士	Wyeth AB	2002-12-01
同种异体移植排斥反应	药物发现	-	Wyeth AB	2002-08-01
移植物抗宿主病	药物发现	美国	Wyeth AB	2002-05-20
移植物抗宿主病	药物发现	加拿大	Wyeth AB	2002-05-20
肾脏疾病	药物发现	-	Wyeth AB	2000-07-01

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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


NCT05050149 (CTgov)	临床2期	淋巴管畸形	12	PTX-022	積齋製選淵壓網構廠窪(鑰鹹鹽積廠鹹憲鏇憲積) = 壓構簾鬱網鹽鏇窪顧鏇範獵築餘獵蓋願構選廠 (觸膚餘網淵範艱蓋鬱憲, 窪憲選壓範憲選艱膚艱 ~ 廠鏇鹽窪廠繭蓋憲窪積) 更多	-	2025-01-17
SIRONA (PRNewswire) 人工标引	N/A	外周动脉疾病	482	MagicTouch PTA	廠觸壓製築鏇網憲積範(膚憲醖壓蓋壓範鬱鹹膚) = 製鹽鬱獵鹹艱遞齋壓憲簾醖襯鹹壓襯顧壓鏇網 (鹽範壓夢鹹積襯醖繭醖 ) 达到更多	相似	2025-01-08
				紫杉醇涂层球囊	廠觸壓製築鏇網憲積範(膚憲醖壓蓋壓範鬱鹹膚) = 築餘鬱餘願衊鬱積壓鹽簾醖襯鹹壓襯顧壓鏇網 (鹽範壓夢鹹積襯醖繭醖 ) 达到更多
NCT04339101 (1043, ASH2024)	临床2期	骨髓纤维化 \| 急性白血病 \| 骨髓增生异常综合征 HGF \| IL17 \| TNFaR ... 更多	59	Itacitinib + Tacrolimus/Sirolimus	糧製糧夢選構衊餘餘築(繭繭醖淵顧醖襯糧顧醖) = 鹹獵窪蓋鹽憲鏇齋憲淵繭獵壓鬱蓋衊製簾鹹築 (窪構範醖鑰糧觸壓製衊, 41 ~ 66) 更多	积极	2024-12-09
				Tacrolimus/Sirolimus	糧製糧夢選構衊餘餘築(繭繭醖淵顧醖襯糧顧醖) = 繭鏇艱鑰窪壓醖築鹹衊繭獵壓鬱蓋衊製簾鹹築 (窪構範醖鑰糧觸壓製衊 )
ChiCTR1900020657 (1187Sirolimus, ASH2024)	临床3期	免疫性血小板减少症一线	-	HD-DXM plus sirolimus	艱窪襯鏇憲鑰艱築蓋壓(積繭願積廠齋膚遞醖觸) = 廠憲遞糧淵鏇築淵築範窪窪齋範齋壓餘衊夢鬱 (鹽鬱獵簾醖淵鬱鹹淵鹹 ) 更多	积极	2024-12-07
				HD-DXM monotherapy	艱窪襯鏇憲鑰艱築蓋壓(積繭願積廠齋膚遞醖觸) = 簾壓鑰簾遞網願憲餘顧窪窪齋範齋壓餘衊夢鬱 (鹽鬱獵簾醖淵鬱鹹淵鹹 ) 更多
NCT02105766 (CTgov)	临床2期	β地中海贫血 \| 造血干细胞移植 \| 镰状细胞血症 \| 移植物抗宿主病	56	Radiotherapy+cyclophosphamide+Sirolimus+Pentostatin+Alemtuzumab (Female Participants With SCD or Beta-thalassemia Receiving Stem Cell Transplant With Male Donor)	齋淵鬱夢夢蓋憲廠顧選(構夢鏇範製窪夢衊餘築) = 鬱願製襯簾艱製網醖鬱糧願獵膚製餘醖積構憲 (願範淵範鹽選獵膚淵網, 獵膚齋膚憲淵獵醖築淵 ~ 鹽網鑰艱膚選襯觸顧願) 更多	-	2024-11-29
				Radiotherapy+cyclophosphamide+Sirolimus+Pentostatin+Alemtuzumab (Participants With Pre-existing Antibodies and SCD or Beta-thalassemia Receiving Stem Cell Transplant)	蓋壓築壓選顧鏇蓋獵蓋(鏇願鹹窪壓餘繭願積築) = 膚鹽構膚選醖獵簾醖蓋壓夢遞廠範艱製簾膚網 (膚膚製範衊積淵築艱構, 鏇製網淵鹹遞願築窪製 ~ 網鏇餘獵積鏇簾鹽鏇襯) 更多
NCT03095703 (CTgov)	临床2期	腺瘤性结肠息肉病	4	Sirolimus	襯鬱構鑰獵餘襯餘鏇獵(廠膚襯齋鬱願鏇顧衊鬱) = 積鏇簾築齋蓋觸餘廠願餘鏇衊積構蓋鏇鑰製願 (壓壓獵壓網衊淵淵選壓, 襯襯鏇選選餘鑰願鹹鹹 ~ 夢鹹顧顧淵積鹹齋醖艱) 更多	-	2024-10-24
NCT04348292 (CTgov)	临床1期	可切除非小细胞肺癌 \| 非小细胞肺癌IIIA期	3	Sirolimus+Durvalumab	築網衊獵願繭衊糧遞醖(壓積願選廠壓餘選廠鬱) = 窪廠壓糧壓築鹽衊簾廠窪鏇積選衊廠憲廠淵鑰 (願齋廠艱鑰獵網積構繭, 憲衊廠範齋夢鬱遞齋鬱 ~ 製積築繭憲簾壓築衊窪) 更多	-	2024-08-22
NCT03077542 (CTgov)	临床1/2期	镰状细胞血症	57	Filgrastim	鏇願窪鬱醖範膚糧襯構(簾積網網鬱齋觸繭鬱壓) = 廠鏇衊鏇鏇鑰餘繭廠壓顧鹽鬱願艱衊顧願範餘 (襯齋襯鹹顧壓鏇網鹽鹹, 構餘網構壓衊範鏇糧襯 ~ 艱築遞醖憲積蓋鬱廠憲) 更多	-	2024-08-07
NCT04393454 (CTgov)	临床2期	转移性实体瘤 \| MMRD实体瘤 \| MSI-H 癌症	6	Sirolimus 2mg Tablet	(糧遞觸憲獵壓艱鑰鹽壓) = 積遞憲顧夢範齋窪遞獵壓鏇遞願淵簾構餘製糧 (鹹願淵膚製觸齋鹽艱艱, 製窪鏇觸衊繭簾壓築選 ~ 鬱網鹹築獵糧鹽夢窪憲) 更多	-	2024-07-25
NCT01467986 (RIST-rNB-2011, Pubmed) 人工标引	临床2期	神经母细胞瘤	129	dasatinib-rapamycin	(膚齋壓繭築窪鹽夢蓋願) = 襯積鹹鑰餘廠顧鏇鏇艱膚餘選觸餘遞窪構製網 (餘餘鑰鬱醖餘鏇鹽繭齋, 7 ~ 17) 更多	积极	2024-07-01
				irinotecan-temozolomide	(膚齋壓繭築窪鹽夢蓋願) = 憲壓選遞衊鹽簾膚製願膚餘選觸餘遞窪構製網 (餘餘鑰鬱醖餘鏇鹽繭齋, 2 ~ 8) 更多