SJALL23T: Adding Dasatinib Or Venetoclax To Improve Responses In Children With Newly Diagnosed T-Cell Acute Lymphoblastic Leukemia (ALL) Or Lymphoma (T-LLY) Or Mixed Phenotype Acute Leukemia (MPAL)

This is a clinical trial testing whether the addition of one of two chemotherapy agents, dasatinib or venetoclax, can improve outcomes for children and young adults with newly diagnosed T-cell acute lymphoblastic leukemia and lymphoma or mixed phenotype acute leukemia.
Primary Objective
* To evaluate if the end of induction MRD-negative rate is higher in patients with T-ALL treated with dasatinib compared to similar patients treated with 4-drug induction on AALL1231.
* To evaluate if the end of induction MRD-negative rate is higher in patients with ETP or near-ETP ALL treated with venetoclax compared to similar patients treated with 4-drug induction on AALL1231.
Secondary Objectives
* To assess the event free and overall survival of patients treated with this therapy.
* To compare grade 4 toxicities, event-free survival (EFS) and overall survival (OS) of patients treated with this therapy in induction and reinduction to toxicities of similar patients treated on TOT17.

开始日期2025-01-01

申办/合作机构

St. Jude Children's Research Hospital, Inc.

[+1]

NCT06210750 / Withdrawn临床2期IIT

A Randomized Phase 2 Trial to Evaluate the Efficacy of BCL-2 Inhibitor Therapy With Chemotherapy Compared to Chemotherapy Alone in Adult Patients With Newly Diagnosed T-Cell ALL and T-Cell Lymphoblastic Lymphoma

This phase II trial tests the safety and effectiveness of giving chemotherapy with or without venetoclax and/or navitoclax for the treatment of patients with newly diagnosed T-cell acute lymphoblastic leukemia (T-ALL) or T-cell lymphoblastic lymphoma (T-LBL). Chemotherapy drugs work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Venetoclax and navitoclax are in a class of medications called B-cell lymphoma-2 (BCL-2) inhibitors. They may stop the growth of cancer cells by blocking Bcl-2, a protein needed for cancer cell survival. Giving chemotherapy with or without venetoclax and/or navitoclax may be effective treatments for patients with newly diagnosed T-ALL or T-LBL.

开始日期2024-08-09

申办/合作机构

National Cancer Institute

CTR20240655 / Recruiting临床3期

奈拉滨注射液治疗儿童和成人难治或复发的T淋巴母细胞白血病（T-ALL）和T淋巴母细胞淋巴瘤（T-LBL）患者的有效性及安全性研究

评估儿童和成人难治或复发的T-ALL和T-LBL患者，接受奈拉滨注射液治疗后IRC评估的2周期内的复合完全缓解率（CCR）。

开始日期2024-05-07

申办/合作机构

正大天晴药业集团南京顺欣制药有限公司

100 项与奈拉滨相关的临床结果

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100 项与奈拉滨相关的转化医学

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100 项与奈拉滨相关的专利（医药）

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254

项与奈拉滨相关的文献（医药）

2025-01-01·PEDIATRIC BLOOD & CANCER

Cost–utility of nelarabine for the first‐line treatment of newly diagnosed pediatric T‐cell acute lymphoblastic leukemia in Canada

Article

作者: Gibson, Paul ; Shoukry, Roaa ; Sala, Mandy ; Bond, Elizabeth ; Moskalewicz, Alexandra ; Pechlivanoglou, Petros ; Bradley, Nicole ; Gupta, Sumit

Abstract:

Background:

The Children's Oncology Group (COG)‐AALL0434 trial investigated the addition of nelarabine to the augmented Berlin–Frankfurt–Münster (aBFM) protocol in patients (1.0–30.99 years) with newly diagnosed T‐cell acute lymphoblastic leukemia (T‐ALL). Despite demonstrating superior outcomes, nelarabine is not currently funded by many health systems, in part due to a lack of cost‐effectiveness data. We estimated the cost–utility of nelarabine for this indication from a Canadian public healthcare payer perspective.

Methods:

We developed a microsimulation model that followed hypothetical patients with newly diagnosed T‐ALL from post‐induction therapy to death. Three health states were modeled: relapse‐free, post‐relapse, and death. Efficacy was estimated using AALL0434 and retrospective data from Ontario, Canada. Costs were obtained from Canadian sources. Utility estimates and long‐term mortality risks were sourced from literature. Total healthcare costs, quality‐adjusted life‐years (QALYs), and incremental cost‐effectiveness ratio (ICER) were reported. Probabilistic and scenario analyses were conducted.

Results:

Incorporating nelarabine in the aBFM protocol increased costs by $51,670 Canadian dollars per patient, but resulted in 1.97 more QALYs and an ICER of $26,184/QALY. Most of the identified cost and benefit were accrued within the AALL0434 trial period (first 11 years post diagnosis) and while patients were in the relapse‐free health state. Across multiple scenarios, the ICER was stable under an assumed $50,000/QALY threshold.

Conclusion:

Incorporating nelarabine into aBFM was cost‐effective across different scenarios and assumptions. These results support its funding by public and private payers.

2024-12-01·LEUKEMIA

Longitudinal follow up of a phase 2 trial of venetoclax added to hyper-CVAD, nelarabine and pegylated asparaginase in patients with T-cell acute lymphoblastic leukemia and lymphoma

Article

作者: Wierda, William ; Issa, Ghayas ; Alvarado, Yesid ; Ferrajoli, Alessandra ; Maiti, Abhishek ; Jabbour, Elias ; Konopleva, Marina ; Garcia-Manero, Guillermo ; Kantarjian, Hagop M. ; Kadia, Tapan ; Short, Nicholas J ; Kebriaei, Partow ; Ravandi, Farhad ; Borthakur, Gautam ; Kantarjian, Hagop M ; Huang, Xuelin ; Balkin, Hayley ; Short, Nicholas J. ; Garris, Rebecca ; Jain, Nitin ; Senapati, Jayastu

Optimal frontline use of active agents in T-cell acute lymphoblastic leukemia/lymphoma (T-ALL/LBL) is prudent to improve outcomes. We report the long-term follow-up of the phase 2 trial of HyperCVAD with nelarabine and pegylated asparaginase (Original cohort). In the latest protocol iteration venetoclax was added to the induction/consolidation regimen (Venetoclax cohort). Eligible patients were adults with untreated T-ALL/LBL or after minimal therapy and with adequate organ function. Primary endpoint of this analysis was improvement in 2-year progression free survival (PFS) and overall survival (OS) with venetoclax. From Aug 2007 to Dec 2024, 145 patients, at a median age of 35.4 years, were treated; 46 (33.8%) were in the venetoclax cohort. At median follow-up (mFU) of 62.4 months, 5-year PFS, duration of response (DOR), and OS were 63.7%, 72.0% and 66.2% respectively. In the venetoclax cohort (mFU 24.4 months) 2-year PFS (87.9% versus 64.1%, p = 0.03) and 2-year DOR (93.6% versus 69.2%, p = 0.005) were superior to the original cohort (mFU 89.4 months) and 2-year OS appeared better (87.8% versus 73.9%, p = 0.16). Febrile neutropenia was the most common serious adverse event, seen in 60% patients. The addition of venetoclax to HyperCVAD-nelarabine-pegylated asparaginase was tolerable and led to improvement in DOR and PFS.

2024-11-18·Cureus Journal of Medical Science

Utility of Obtaining Follow-Up Blood Cultures in Gram-Negative Bacterial Bloodstream Infection Among Patients With Hematologic Malignancies

Article

作者: Dhanaraj, Nitya ; Foppiano Palacios, Carlo ; Rowe, Kelly

BACKGROUND:

There is an ongoing debate on the benefit of obtaining repeat blood cultures for Gram-negative bacterial bloodstream infections (GNBSI). However, there is a scarcity of data concerning patients with hematologic malignancies. We sought to assess the utility of obtaining follow-up blood cultures (FUBC) in GNBSI among patients with hematologic malignancies.

METHODS:

A retrospective chart review was conducted to identify all admitted patients with hematologic malignancies with GNBSI from 2018 to 2021 at a tertiary academic medical center. We collected demographics, cancer history and treatment, microbiology and antibiotic use, and clinical course. Descriptive statistics were used.

RESULTS:

A total of 46 episodes of GNBSI among 38 patients were included. The median age was 61.5 years, 63% were male, 50% were White, and 16% were Latinx. Most patients had acute myeloid leukemia (60%), and the most common chemotherapy regimen was cytarabine or nelarabine regimens (35%). Klebsiella pneumoniae was responsible for 37% of the GNBSI cases, in the setting of long-term central venous catheter use (65%) and gastrointestinal sources (50%). FUBCs were collected among almost all patients (98%). Only three cases (7%) had positive FUBC: one had a delay in appropriate therapy, another had a lack of source control, and the last case did not have a source identified. Most patients were treated with beta-lactams (52%) with duration 8-14 days (52%). 22% were admitted to ICU and 9% died during their hospitalization.

CONCLUSION:

We found a few cases of positive FUBC. Routine FUBC may not be appropriate for all GNBSI patients with hematological malignancies, particularly during the current blood culture bottle shortage.

项与奈拉滨相关的新闻（医药）

2024-12-06

·学术经纬

Cancer Discovery｜儿童急性T淋巴细胞白血病的临床特征与高危突变

撰文 | 尹彤责编 | 周叶斌急性T淋巴细胞白血病（T-ALL）是儿童最常见的血液系统肿瘤之一。其中γδ T-ALL是一个较少研究的亚型，其肿瘤细胞表面表达由γ链和δ链组成的T细胞受体（TCR），约占全部T-ALL病例的10%。既往研究提示，该亚型可能面临更差的治疗效果和更高的复发风险。由于缺乏大规模针对γδ T-ALL的研究，其临床特征和发病机制仍不清楚。虽然近年来通过基因组学研究取得了一些进展，但这些研究纳入的γδ T-ALL病例较少，且缺乏对非编码区等基因组特征的分析，因此亟需进行更全面、深入的研究。近日，以圣裘德儿童研究医院 Charles G. Mullighan 和 Hiroto Inaba 团队牵头的多国联合团队在 Cancer Discovery 发表了题为 Biologic and Clinical Analysis of Childhood Gamma Delta T-ALL Identifies LMO2/STAG2 Rearrangements as Extremely High Risk 的文章。该研究基于200例γδ T-ALL的临床和基因组数据，揭示了发病年龄<3岁、诱导治疗后MRD≥1%和STAG2/LMO2突变是儿童γδ T-ALL的高危因素，进而筛选了针对此类患者有效的治疗策略。本研究纳入了由13项临床研究组成的多国合作组的200例25岁以下的γδ T-ALL患者，以及1,067例根据治疗方案匹配的非γδ T-ALL对照病例。研究发现，γδ T-ALL患者总体上表现出较差的预后，诊断年龄更小，且治疗后的应答更差，表现为在诱导治疗和巩固治疗后都有更高的微小残留病变（measurable residual disease，MRD）水平，需要进行造血干细胞移植的比例更高，且原发病或治疗副作用造成的死亡率更高。诊断年龄小于3岁的γδ T-ALL患者，其5年无事件生存率（event-free survival，EFS）仅为33%，总生存率（overall survival，OS）仅为49%，显著低于诊断年龄≥3岁的γδ T-ALL患者或非γδ T-ALL患者。此外，诱导治疗结束时（end of induction，EOI），MRD≥1%的患者在所有年龄段的生存率均显著低于MRD<1%的患者。这些数据表明诊断年龄 <3岁和 EOI 时 MRD≥1% 是提示 γδ T-ALL 患儿不良预后的高危因素，因此这些高风险患者可能需要优化的治疗方案。此外，研究发现，γδ T-ALL患者的复发主要局限于骨髓（骨髓复发率高达91.3%），而非γδ T-ALL患者中枢神经系统复发更常见。研究设计和患者临床结局：（A）研究方案，（B）γδ T-ALL患者与非γδ T-ALL患者的生存率对比，（C/D）γδ T-ALL患者中诊断年龄和EOI时MRD水平对生存率的影响结合全基因组测序（whole genome sequencing，WGS）、RNA测序和HiChIP等技术，研究者对γδ T-ALL患者的基因变异进行了分型，并解析了突变导致的疾病机制。γδ T-ALL患者与非γδ T-ALL患者相比具有独特的基因型分布。STAG2/LMO2双突变是γδ T-ALL患者特征性变异之一，所有纳入研究的该亚型γδ T-ALL患儿的诊断年龄都小于3岁，且与其他类型患者相比生存期显著较短。进一步研究表明STAG2/LMO2双突变患者中最常见的变异类型是LMO2::STAG2重排，导致STAG2失活和LMO2过度激活。STAG2是黏着复合物（cohesin complex）的重要组分，参与染色质空间折叠以形成拓扑相关结构域（topologically associating domain，TAD），进而通过增强子-启动子相互作用调控基因正常表达。通过构建STAG2缺失和过表达的细胞模型，并进行RNA-seq和H3K27ac ChIP-seq分析，研究者证明了STAG2失活将导致TAD结构异常，严重破坏T细胞分化相关基因表达，导致白血病的发生。此外，研究还发现了其他γδ T-ALL特异性基因分型，如LMO2-γδ样、超二倍体T-ALL伴重现性染色体增加、TLX3重排和PICALM::MLLT10融合等，这些亚型γδ T-ALL患者具有各自不同的临床特征。 γδ T-ALL基因分型和临床特征：（A）参考所有T-ALL基因组数据对γδ T-ALL进行基因分型，（B）不同基因型在γδ T-ALL与非γδ T-ALL患者中的分布，（D）γδ T-ALL患者不同基因型与诊断年龄的关系，（E）不同基因型γδ T-ALL患者的生存曲线最后，研究者通过高通量药物筛选探索了潜在的治疗策略。PARP抑制剂talazoparib和组蛋白去乙酰化酶抑制剂vorinostat是对STAG2/LMO2 γδT-ALL细胞具有显著杀伤作用的药物，其中PARP抑制剂靶向STAG2失活造成的DNA复制缺陷，使双链DNA断裂积累并诱导细胞死亡。在患者来源的异种移植模型（PDX）中，talazoparib 和 vorinostat 都可以有效地抵抗肿瘤生长。其他有效的药物包括nelarabine和L-门冬酰胺酶等，这些药物都已经被用于T-ALL或其他肿瘤的临床研究或治疗。此发现为高风险的γδ T-ALL患儿提供了一种潜在的精准治疗策略。针对talazoparib（Tal）和vorinostat（Vor）的临床前试验表明两种药物均能够显著抑制PDX肿瘤细胞的生长总之，本研究首次全面探讨了儿童γδ T-ALL的临床特征和基因分型，并揭示了高危突变的致病机制及潜在的治疗策略。研究明确了诊断年龄和MRD水平对患者预后的关键作用，并揭示了高危患儿STAG2失活对染色质结构和基因表达具有深远影响。更重要的是，本研究提出了 PARP 抑制剂等可做为靶向 STAG2/LMO2 亚型的潜在治疗策略，这些成果加深了对儿童γδ T-ALL的认识，也为未来γδ T-ALL患儿的风险分层和个体化治疗开发提供了重要参考。撰文责编制作排版 | Sheila 校对 | uu 延伸阅读儿童T细胞急性淋巴细胞白血病的多组学分析竺晓凡/张英驰团队揭示儿童T-ALL癌细胞克隆演化模式及耐药新机制 *本文由深圳市拾玉儿童公益基金会“儿童肿瘤前沿”团队编译或约稿，文中图表均源引自文献原文。本文著作权归文章作者所有，欢迎个人转发分享，未经允许禁止转载，作者拥有所有法定权利，违者必究。如需转载，请留言或联系shiyu@curekids.cn。本文旨在分享儿童肿瘤科研前沿成果，不是治疗方案推荐。如需获得疾病治疗方案指导，请前往正规医院就诊。 ▼滑动查看更多▼ 原文摘要（Abstract） Acute lymphoblastic leukemia expressing the gamma delta T-cell receptor (γδ T-ALL) is a poorly understood disease. We studied 200 children with γδ T-ALL from 13 clinical study groups to understand the clinical and genetic features of this disease. We found age and genetic drivers were significantly associated with outcome. γδ T-ALL diagnosed in children under 3 years of age was extremely high-risk and enriched for genetic alterations that result in both LMO2 activation and STAG2 inactivation. Mechanistically, using patient samples and isogenic cell lines, we show that inactivation of STAG2 profoundly perturbs chromatin organization by altering enhancer-promoter looping, resulting in deregulation of gene expression associated with T-cell differentiation. High-throughput drug screening identified a vulnerability in DNA repair pathways arising from STAG2 inactivation, which can be targeted by poly(ADP-ribose) polymerase inhibition. These data provide a diagnostic framework for classification and risk stratification of pediatric γδ T-ALL. Significance: Patients with acute lymphoblastic leukemia expressing the gamma delta T-cell receptor under 3 years old or measurable residual disease ≥1% at end of induction showed dismal outcomes and should be classified as having high-risk disease. The STAG2/LMO2 subtype was enriched in this very young age group. STAG2 inactivation may perturb chromatin conformation and cell differentiation and confer vulnerability to poly(ADP-ribose) polymerase inhibition. DOI: 10.1158/2159-8290.CD-23-1452 👇点击此处，直达原文

细胞疗法临床结果免疫疗法临床研究信使RNA

2024-06-28

·BioSpace

Shorla Oncology Announces FDA Approval for TEPYLUTE, A Novel Formulation to Treat Breast and Ovarian Cancer

TEPYLUTE is a ready-to-dilute formulation of thiotepa that reduces preparation time and enables accurate dosing CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Shorla Oncology (‘Shorla’), a U.S.-Ireland specialty pharmaceutical company, announced today that the U.S. Food and Drug Administration (FDA) has approved the company’s New Drug Application (NDA) for TEPYLUTE, a ready-to-dilute formulation to treat breast and ovarian cancer in an easier to prepare, injectable product that enables dosing accuracy.1 ‘’This approval fulfills an unmet need by addressing the shortcomings and handling complexities of the current lyophilized powder formulation,” said Sharon Cunningham, Chief Executive Officer and Co-Founder of Shorla Oncology. “We have taken a vital oncology drug and made it easier for oncology clinics and hospitals to use, while also reducing medical personnel exposure to a hazardous drug.” TEPYLUTE, formerly SH-105, is the third FDA-approved drug for Shorla, and a significant milestone for the company as it seeks approval for several cancer-fighting drugs for the U.S. market. “The approval of TEPYLUTE represents an important milestone for Shorla as our first in-house developed NDA,” said Orlaith Ryan, Chief Technical Officer and Co-Founder of Shorla Oncology. TEPYLUTE is a liquid form of a well-established, standard of care oncology drug, thiotepa. The new formulation eliminates the need for complex and time-consuming reconstitution. It provides consistent dosing accuracy and allows for “just in time” preparation.2 “Among TEPYLUTE’s many benefits, it removes the necessity to reconstitute which can introduce additional risks of drug preparation errors,” emphasized Rayna Herman, Chief Commercial Officer. “We look forward to providing an update on our launch plans for TEPYLUTE in the near future.” The American Cancer Society estimates that more than 300,000 women will be diagnosed with breast cancer in the U.S in 2024.3 About 19,680 women will be diagnosed with ovarian cancer in the United States.4 Shorla Oncology is currently marketing two products with a robust pipeline including SH-201, the first palatable oral liquid treatment for certain forms of leukemia and other cancers. In April, the company announced the FDA had accepted SH-201 for an NDA review with an expected action date of November 30, 2024. About Shorla Oncology Shorla Oncology is a privately- held, U.S. and Ireland- based commercial stage specialty pharmaceutical company established by Sharon Cunningham and Orlaith Ryan. The company has an advanced pipeline of innovative oncology drugs for orphan and pediatric cancers. Shorla is focused on indications where existing treatments are limited, in shortage or the drug applications are inadequate for the target population. The company’s growing portfolio brings accessible, affordable and life-saving treatments to patients, delivering a major contribution to patient care. Shorla currently markets two products, Nelarabine for the treatment of T-cell Leukemia and JYLAMVO for the treatment of acute lymphoblastic leukemia and other indications. For further information, please visit ________________________ 1 Carausu M, Carton M, Darlix A, Pasquier D, Leheurteur M, Debled M, Mouret-Reynier MA, Goncalves A, Dalenc F, Verret B, Campone M. Breast cancer patients treated with intrathecal therapy for leptomeningeal metastases in a large real-life database. ESMO Open 6: 100150. 1 Reinhardt H, Otte P, Eggleton AG, Ruch M, Wöhrl S, Ajayi S, Duyster J, Jung M, Hug MJ, Engelhardt M. Avoiding chemotherapy prescribing errors: analysis and innovative strategies. Cancer. 2019 May 1;125(9):1547-57. 2 TEPYLUTE Prescribing information and data on Shorla Oncology. 3 Breast Cancer Statistics | How Common Is Breast Cancer? | American Cancer Society, Accessed June 27, 2024 4 Ovarian Cancer Statistics | How Common is Ovarian Cancer | American Cancer Society, Accessed June 27, 2024

上市批准申请上市

2024-06-14

·GlobeNewswire-Health Care

Phase 2 Findings Show Wugen’s Investigational Allogeneic CAR-T, WU-CART-007, Was Highly Effective and Surpassed Standard of Care in Treating Hard-to-Treat T-ALL/LBL; Data Presented Today at European Hematology Association (EHA) 2024 Hybrid Congress

-- Researchers Shared Findings Including a Clinically Manageable Safety Profile and Anti-leukemic Activity with WU-CART-007 in Patients with R/R T-ALL/LBL and Previously Treated with Existing Therapies -- U.S. Biotech Wugen to Initiate Follow-Up Study Exploring Its Anti-CD7, Off-the-Shelf, Allogeneic CAR-T Cell Therapy in Pediatric and Minimal Residual Disease (MRD) Patients in Q424 -- Company Presented Additional New Data at EHA on WU-CART-007 and WU-NK-101, Its Off-the-Shelf Investigational Memory Natural Killer Cell Therapy ST. LOUIS and SAN DIEGO, June 14, 2024 (GLOBE NEWSWIRE) -- Wugen, Inc., a clinical-stage U.S. biotechnology company developing allogeneic, off-the-shelf cell therapies for the treatment of hematological and solid tumor malignancies, announces positive Phase 2 results for the company’s investigational anti-CD7 CAR-T therapy, WU-CART-007 (“W-T7”), in patients with relapsed/refractory (R/R) t-cell acute lymphoblastic leukemia/lymphoma (T-ALL/LBL). In his presentation today at the European Hematology Association (EHA) 2024 Hybrid Congress in Madrid, lead investigator Ibrahim Aldoss, M.D., City of Hope in Duarte, CA, shared data from this Phase 2 cohort expansion study showing a clinically manageable safety profile as well as evidence of anti-leukemic activity with WU-CART-007 (“W-T7”) in patients with R/R T-ALL/LBL previously treated with existing therapies for their cancers. “Relapsed and refractory T-ALL/LBL is a highly aggressive disease with high rates of relapse and mortality in both children and adults,” Dr. Aldoss said. “These results are exciting and support the potential of this allogeneic CAR-T as a new treatment for patients who still face a refractory illness despite having received the best medicines we have for T-ALL/LBL right now.” “The need for effective, allogeneic treatments that are available at the point of care without delays is urgent for patients with hard-to-treat blood cancers,” said Wugen Chief Medical Officer Jan Davidson-Moncada, M.D., Ph.D. “Wugen is confident we can overcome specific challenges that have existed for CAR-T therapies in targeting T-cell diseases. We are eager to conduct further studies of WU-CART-007, which was invented by Wugen’s Co-founder and Chief Scientific Officer Matt Cooper, Ph.D., to target CD7+ malignancies.” Key Phase 2 Findings The presentation at EHA featured results with the recommended dose (RP2D) of WU-CART-007 administered in 13 patients at a median age 30 years who were heavily pre-treated and had a high burden of disease at the start of the study. A substantial proportion of patients (38%) had progressed or relapsed after allogeneic stem cell transplant. Outcomes following WU-CART-007 treatment in the study surpassed current standard of care1 for R/R T-ALL/LBL and included: Composite complete remission rate (CRc) of 73% (8/11) (primary endpoint).Overall response rate of 91% (10/11; 2 partial response (PR) in patients with extramedullary disease, R/R T-LBL).Duration of response (DOR) (secondary endpoint): Median duration (mDOR) was 6.2 months (95% CI: 1.8, NE);Median follow up was 8.5 months (95% CI: 2.7, NE); 46% (5/11) patients remained in continuous remission at 4.3 to 8.6 months. Seven patients (5 at RP2D) successfully transplanted. Pharmacokinetic analysis exhibited rapid expansion and persistence of an allogeneic cell therapy: At RP2D, WU-CART-007 expansion peaked at Day 10 (223,799 copies/g of DNA) and can be detected out to Day 90. No patient developed drug product-specific anti-HLA antibodies.No anti-drug antibody detected on any patients to date. Phase 1 dose escalation data from this first-in-human global Phase 1/2 clinical trial of WU-CART-007 for the treatment of relapsed or refractory (R/R) T-cell acute lymphoblastic leukemia (T-ALL)/lymphoblastic lymphoma (LBL) were presented last December at the American Society of Hematology meeting. See those findings here. Additional Wugen Data at EHA Researchers presented other new findings with WU-CART-007 and WU-NK 101 in these posters at EHA: Title: Preliminary Effect of Enhanced Lymphodepletion (ELD) on WU-CART-007 in T-ALL/LBLPresenters: Ouiam Bakkacha M.D. and Ben Capoccia Ph.D., Wugen, St. Louis, MOAbstract: P408 Title: WU-NK-101 is an Off-The-Shelf Memory NK Cell with a Time-Resilient, Anti-Tumor Phenotype that can be Detected in R/R AML Patient PBMC After InfusionPresenter: Laura Arthur, Ph.D., Wugen, St. Louis, MOAbstract: P1422 More information is available on the EHA 2024 Hybrid Congress website. About WU-CART-007WU-CART-007 is an allogeneic, off-the-shelf, fratricide-resistant CD7-targeted CAR-T cell therapy engineered to overcome the technological challenges of harnessing CAR-T cells to treat CD7+ hematological malignancies. Wugen is deploying CRISPR/Cas9 gene editing technology to delete CD7 and the T-cell receptor alpha constant (TRAC), preventing CAR-T cell fratricide and mitigating the risk of graft-versus-host-disease (GvHD). WU-CART-007 is manufactured using healthy donor-derived T-cells to eliminate the risk of malignant cell contamination historically observed in the autologous CAR-T setting. WU-CART-007 is currently being evaluated in a global Phase 1/2 clinical trial for the treatment of relapsed or refractory (R/R) T-cell acute lymphoblastic leukemia (T-ALL)/lymphoblastic lymphoma (LBL). Additional information is available on clinicaltrials.gov, identifier NCT# 04984356. WU-CART-007 has received Orphan Drug, Fast Track, Rare Pediatric Disease and Regenerative Medicine Advanced Therapy (RMAT) designations from the U.S. Food and Drug Administration and received PRIME (“Priority Medicines”) designation in the European Union for the treatment of relapsed/refractory (R/R) T-cell acute lymphoblastic leukemia (T-ALL) and T-cell lymphoblastic lymphoma (T-LBL). RMAT and PRIME designations provide increased agency support to expedite the development and review of promising therapies for patients in need. About WU-NK-101WU-NK-101 is a novel immunotherapy harnessing the power of memory natural killer (NK) cells to treat liquid and solid tumors. Memory NK cells are hyper-functional, long-lasting immune cells that exhibit enhanced anti-tumor activity and a cytokine-induced memory-like (CIML) phenotype. This cell population has a superior phenotype, proliferation capacity, and metabolic fitness, making it better suited for cancer therapy than other NK cell therapies. Wugen is applying its proprietary MonetaTM platform to advance WU-NK-101 as a commercially scalable, off-the-shelf cell therapy for cancer. WU-NK-101 is currently in development for acute myelogenous leukemia (AML). Wugen is planning to initiate solid tumor studies of WU-NK-101 in combination with cetuximab. Studies of WU-NK-101 to date have shown promising robust in vivo activity in various tumor indications, retention of anti-cancer activity in TME, resistance to immune suppression, and enhanced activity with checkpoint inhibitors. WU-NK-101 has received Orphan Drug Designation from the U.S. Food and Drug Administration for the treatment of AML. About Wugen A spin-out of Washington University in St. Louis, Wugen, Inc. is a clinical-stage U.S. biotechnology company developing the next generation of off-the-shelf CAR-T and memory natural killer (NK) cell therapies for cancer. For more information, please visit www.wugen.com. Investor Contact:Mark LewisWugenMlewis@wugen.com+1 314-501-1968 Media Contact:Christine FanelleScient PRchristine@scientpr.com+1 215-595-5211 1 Nelarabine USPI

临床结果细胞疗法免疫疗法孤儿药ASH会议

100 项与奈拉滨相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D05134	奈拉滨	L01BB07	DB01280

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
T细胞急性淋巴细胞白血病/淋巴瘤	日本	Sandoz Pharmaceuticals AG	2007-10-19
前体T淋巴细胞白血病淋巴瘤	美国	Sandoz, Inc.	2005-10-28

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
复发性T淋巴母细胞淋巴瘤	临床3期	中国	正大天晴药业集团股份有限公司	2024-05-01
难治性 T 急性淋巴细胞白血病	临床3期	中国	正大天晴药业集团股份有限公司	2024-05-01
T细胞急性淋巴细胞白血病复发	临床2期	美国	Novartis AG	2010-06-01
T细胞急性淋巴细胞白血病复发	临床2期	美国	GSK Plc	2010-06-01
T细胞急性淋巴细胞白血病复发	临床2期	澳大利亚	Novartis AG	2010-06-01
T细胞急性淋巴细胞白血病复发	临床2期	澳大利亚	GSK Plc	2010-06-01
T细胞急性淋巴细胞白血病复发	临床2期	奥地利	GSK Plc	2010-06-01
T细胞急性淋巴细胞白血病复发	临床2期	奥地利	Novartis AG	2010-06-01
T细胞急性淋巴细胞白血病复发	临床2期	加拿大	GSK Plc	2010-06-01
T细胞急性淋巴细胞白血病复发	临床2期	加拿大	Novartis AG	2010-06-01

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临床结果

适应症

分期

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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


ASH2023	N/A	前体T淋巴细胞白血病淋巴瘤	133	Nelarabine (NEL)	選鹹艱鏇鹹夢壓鬱鹽襯(顧鏇醖壓襯蓋齋顧壓鑰) = 齋遞積醖顧鬱壓築鏇繭觸遞艱糧鹽鹹齋製願願 (壓鑰糧鹹廠鹹鹹鏇餘齋 )	-	2023-12-11
				Pegylated Asparginase (PEG)	選鹹艱鏇鹹夢壓鬱鹽襯(顧鏇醖壓襯蓋齋顧壓鑰) = 憲獵製構憲鏇醖蓋鹹窪觸遞艱糧鹽鹹齋製願願 (壓鑰糧鹹廠鹹鹹鏇餘齋 )
GRAALL-2014/T (ASH2023)	临床2期	前体T淋巴细胞白血病淋巴瘤巩固	325	Nelarabine	觸簾夢鹽構範願廠積齋(選艱襯淵觸壓膚壓淵餘) = 範鹽壓衊夢鹽獵選觸鹹範繭廠觸蓋積襯膚網廠 (願積願衊艱餘蓋鬱壓廠, 20 ~ 37) 更多	-	2023-12-11
NCT02763384 (CTgov)	临床2期	难治性 T 急性淋巴细胞白血病 \| 淋巴母细胞淋巴瘤	12	BL-8040+Nelarabine	糧齋觸鬱淵膚鬱廠壓遞(獵顧鹽窪觸積鹹製襯糧) = 淵鑰鏇範鑰鹽壓築蓋獵醖鏇獵膚醖網願繭繭選 (鑰鹹顧壓餘醖構淵願製, 選鏇膚築鏇鹽淵鏇淵獵 ~ 網憲醖積齋願獵遞網淵) 更多	-	2023-10-31
Pubmed	临床1期	肿瘤	-	Nelarabine	夢獵醖膚簾鹽願獵醖範(製顧鑰顧觸艱窪鹽顧壓) = 觸壓繭糧膚積廠窪顧獵網襯網膚醖醖憲醖衊膚 (鑰觸築遞鑰齋壓鹹築繭 ) 更多	-	2022-11-29
EHA2022	N/A	T细胞急性淋巴细胞白血病/淋巴瘤	120	HyperCVAD + Nelarabine	齋觸齋壓膚鹹鏇壓顧衊(鏇艱觸襯繭鹽鑰繭糧齋) = elevated liver enzymes in 25 pts (21%) 築觸簾選鹹醖簾窪醖鏇 (鹽憲選襯窪構淵選鏇範 ) 更多	-	2022-05-12
				HyperCVAD + Nelarabine + PEGylated Asparaginase
NCT00501826 (ASH 12020 \| ASH 22020) 人工标引	临床2期	前体T淋巴细胞白血病淋巴瘤 \| 复发性T淋巴母细胞淋巴瘤一线	96	Peg-Asparaginase+Nelarabine	鏇鏇獵鏇繭齋齋鏇鏇範(蓋觸糧願築範鹹遞壓構) = 廠憲願醖繭顧顧窪蓋網蓋壓壓膚構醖壓簾壓構 (餘構膚遞廠遞夢糧襯獵 ) 更多	积极	2020-11-05
				Peg-Asparaginase+Nelarabine (T-ALL)	構醖廠憲範觸範製鑰蓋(鑰構鹽醖觸鹽鏇淵範範) = 網糧顧獵觸憲餘築膚構鹹遞淵積窪範窪糧襯繭 (製窪簾築艱願蓋積鑰觸 )
NCT00408005 (AALL0434, Pubmed \| J Urol \| J Clin Oncol) 人工标引	临床3期	前体T淋巴细胞白血病淋巴瘤	659	Berlin-Frankfurt-Muenster +Nelarabine	廠觸餘鹽選膚積鏇蓋淵(鹹鏇選壓鹹壓膚鹹築壓) = 鑰鑰蓋鏇網淵齋夢製範餘糧餘夢遞餘鏇願壓簾 (艱蓋築窪艱襯鏇觸範膚, 2.4)	优效	2020-10-01
				Berlin-Frankfurt-Muenster	廠觸餘鹽選膚積鏇蓋淵(鹹鏇選壓鹹壓膚鹹築壓) = 網製觸遞鹹選憲糧餘選餘糧餘夢遞餘鏇願壓簾 (艱蓋築窪艱襯鏇觸範膚, 2.7)
NCT00981799 (CTgov)	临床1/2期	复发性T淋巴母细胞淋巴瘤 \| T细胞急性淋巴细胞白血病复发	23	Etoposide+cyclophosphamide+Filgrastim+Nelarabine+Methotrexate (Nelarabine Dose Level 1)	鬱齋憲鑰鏇夢鬱遞壓鏇(膚願廠鹽憲築壓顧遞顧) = 鹽糧衊網鹽遞夢壓積廠範夢糧範選窪淵醖齋構 (襯齋膚鏇獵遞鹽顧鹹構, 襯醖鏇鏇餘鏇獵鏇鹽艱 ~ 獵憲繭糧膚製鬱鬱構選) 更多	-	2020-10-01
				Etoposide+cyclophosphamide+Filgrastim+Nelarabine+Methotrexate (Nelarabine Dose Level 2)	鬱齋憲鑰鏇夢鬱遞壓鏇(膚願廠鹽憲築壓顧遞顧) = 襯簾艱淵網淵淵鏇鑰襯範夢糧範選窪淵醖齋構 (襯齋膚鏇獵遞鹽顧鹹構, 顧獵選範觸鏇鹽膚壓製 ~ 襯膚顧鏇網壓顧獵淵製) 更多
EHA2019	临床4期	T细胞急性淋巴细胞白血病/淋巴瘤	118	Nelarabine	繭製鹹襯製網選膚遞選(壓鏇壓鬱膚淵廠簾糧構) = 糧範鑰鹹鏇築醖簾鏇鹹顧遞壓願艱蓋醖糧窪窪 (範鑰廠獵艱鹹顧築鬱構 ) 更多	-	2019-06-16
EHA2019	N/A	前体T淋巴细胞白血病淋巴瘤	9	Nelarabine	衊廠壓淵廠鏇觸憲選糧(繭餘膚艱廠構鑰糧齋積) = 網繭遞製顧膚壓選願選醖醖遞製築鬱顧糧膚顧 (顧願鹹醖獵窪窪顧夢鑰 ) 更多	-	2019-05-16