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2024-11-01

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

登录后查看更多信息

100 项与奈拉滨相关的转化医学

登录后查看更多信息

100 项与奈拉滨相关的专利（医药）

登录后查看更多信息

248

项与奈拉滨相关的文献（医药）

2024-11-01·Clinical Lymphoma Myeloma & Leukemia

Incidence and Clinical Presentation of Severe Neurotoxicity from Nelarabine in Patients with T-Cell Acute Lymphoblastic Leukemia

Article

作者: Jain, Nitin ; Nicholas, Short ; Ravandi, Farhad ; Jabbour, Elias ; Woodman, Karin ; Sasaki, Koji ; Kantarjian, Hagop ; Kugler, Eitan ; Braish, Julie S ; Braish, Julie S.

BACKGROUND:

Nelarabine is a purine analog with demonstrated efficacy in the treatment of T-cell Lymphoblastic Leukemia and Lymphoma (T-ALL/LBL). Despite its efficacy and excellent blood-brain barrier penetration, it has a significant side effect profile which is namely concerning for neurotoxicity. Reported neurotoxicity has varied from mild peripheral neuropathy to debilitating grade 4 neurologic complications including Guillain-Barre like syndrome and myelopathy.

PATIENTS AND METHODS:

We conducted a single centered, retrospective case series to study patients who developed severe neurotoxicity after receiving nelarabine as part of T-ALL treatment. One hundred thirty-five patients were identified. Thirteen patients were reviewed for severe neurotoxicity (defined as ≥grade 3), and of those five patients were deemed to have neurotoxicity secondary to nelarabine exposure.

RESULTS:

Five patients (4%) developed severe neurotoxicity as manifested by Guillain-Barre like syndrome or myelopathy within a timeframe of eight to fifty-eight days from last nelarabine dose. Upon diagnosis, patients received formal neurologic evaluation by our neuro-oncology specialists including imaging, cerebrospinal fluid testing, and electromyography. Patients were treated with IVIG, and steroids upon diagnosis, but the majority of neuro-deficits were irreversible.

CONCLUSION:

Our study shows that nelarabine is generally well-tolerated, and the incidence of severe neurotoxicity is rare. Given the potential risk of severe neurotoxicity, we propose capped dose of nelarabine 1000 mg/day, neurological assessment before subsequent dosing, and avoidance of simultaneous IT therapy during nelarabine administration.

2024-10-10·JOURNAL OF CLINICAL ONCOLOGY

Impact of Age on Pharmacogenomics and Treatment Outcomes of B-Cell Acute Lymphoblastic Leukemia

Article

作者: Yang, Wenjian ; Li, Zhenhua ; Stock, Wendy ; Jeha, Sima ; Kornblau, Steven M. ; Relling, Mary V. ; Loh, Mignon L. ; Pui, Ching-Hon ; Yu, Jiyang ; Lee, Shawn H.R. ; Saygin, Caner ; Evans, William E. ; Roberts, Kathryn G. ; Jabbour, Elias ; Inaba, Hiroto ; Yeoh, Allen E.J. ; Litzow, Mark R. ; Crews, Kristine R. ; Paietta, Elisabeth ; Yang, Jun J. ; Karol, Seth E. ; Konopleva, Marina ; Mullighan, Charles G. ; Gocho, Yoshihiro ; Jain, Nitin ; Yoshimura, Satoshi

PURPOSE:

Acute lymphoblastic leukemia (ALL) can occur across all age groups, with a strikingly higher cure rate in children compared with adults. However, the pharmacological basis of age-related differences in ALL treatment response remains unclear.

METHODS:

Studying 767 children and 309 adults with newly diagnosed B-cell ALL enrolled on frontline trials at St Jude Children's Research Hospital, MD Anderson Cancer Center, the Alliance for Clinical Trials in Oncology, and the ECOG-ACRIN Cancer Research Group, we determined the ex vivo sensitivity of leukemia cells to 21 drugs. Twenty-three ALL molecular subtypes were identified using RNA sequencing. We systematically characterized the associations between drug response and ALL genomics in children, adolescents and young adults, and elderly adults. We evaluated the effect of age-related gene expression signature on ALL treatment outcomes.

RESULTS:

Seven ALL drugs (asparaginase, prednisolone, mercaptopurine, dasatinib, nelarabine, daunorubicin, and inotuzumab ozogamicin) showed differential activity between children and adults, of which six were explained by age-related differences in leukemia molecular subtypes. Adolescents and young adults showed similar patterns of drug resistance as older adults, relative to young children. Mercaptopurine exhibited subtype-independent greater sensitivity in children. Transcriptomic profiling uncovered subclusters within CRLF2 -, DUX4 -, and KMT2A -rearranged ALL that were linked to age and cytotoxic drug resistance. In particular, a subset of children had adult-like ALL on the basis of leukemia gene expression patterns across subtypes, despite their chronological age. Resistant to cytotoxic drugs, children with adult-like ALL exhibited poor prognosis in pediatric ALL trials, even after adjusting for age and minimal residual diseases.

CONCLUSION:

Our results provide pharmacogenomic insights into age-related disparities in ALL cure rates and identify leukemia prognostic features for treatment individualization across age groups.

2024-08-01·JOURNAL OF PEDIATRIC HEMATOLOGY ONCOLOGY

Isolated Optic Nerve Relapse in a Pediatric Patient With T-Cell Lymphoblastic Leukemia: A Brief Report

Article

作者: Reed, Suzanne M. ; Rodriguez, Diana P. ; Rangarajan, Hemalatha G. ; Shenkar, Rakefet ; Kahwash, Samir B. ; Abu Arja, Rolla

Isolated optic nerve (ON) relapse is a rare occurrence in lymphoblastic leukemia (LBL). A 10-year-old boy with T-LBL presented 8 months after diagnosis with blurred vision and thickening of right ON on magnetic resonance imaging consistent with relapse. Cerebrospinal fluid and bone marrow were negative for leukemia. He received reinduction chemotherapy (including nelarabine and craniospinal radiation) followed by a myeloablative matched sibling donor bone marrow transplant. He remains in remission 2 years post-transplant with normal vision. We also review the literature for reports of isolated ON relapse in patients with LBL. Our patient’s clinical course demonstrates that disease control can be achieved with early detection of ON relapse before disease progression.

项与奈拉滨相关的新闻（医药）

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

2024-06-14

·BioSpace

Researchers Shared Findings Including a Clinically Manageable Safety Pro 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 pro 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/LBL Presenters: Ouiam Bakkacha M.D. and Ben Capoccia Ph.D., Wugen, St. Louis, MO Abstract: 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 Infusion Presenter: Laura Arthur, Ph.D., Wugen, St. Louis, MO Abstract: P1422 More information is available on the EHA 2024 Hybrid Congress website. About WU-CART-007 WU-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-101 WU-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 . Investor Contact: Mark Lewis Wugen Mlewis@wugen.com +1 314-501-1968 Media Contact: Christine Fanelle Scient PR christine@scientpr.com +1 215-595-5211 1 Nelarabine USPI

临床结果免疫疗法快速通道细胞疗法孤儿药

100 项与奈拉滨相关的药物交易

登录后查看更多信息

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,

后查看更多信息

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,

后查看更多信息

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	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	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	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	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

登录后查看更多信息

临床结果

适应症

分期

评价

查看全部结果

研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


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	壓繭顧網遞餘鏇窪選糧(廠選鏇鑰網顧齋壓膚襯) = 鹹齋壓鬱廠餘鏇製繭遞鑰醖繭觸選襯艱網鏇鑰 (範範窪鏇鏇醖簾夢構艱, 20 ~ 37) 更多	-	2023-12-11
ASH2023	N/A	前体T淋巴细胞白血病淋巴瘤	133	Nelarabine (NEL)	鏇鹽選膚齋鹹製艱積遞(鏇窪鹹製窪遞蓋糧醖製) = 蓋鬱淵襯築廠鹹遞蓋遞構鹹蓋積觸壓繭膚製糧 (膚夢鹽觸獵淵夢蓋膚構 )	-	2023-12-11
				Pegylated Asparginase (PEG)	鏇鹽選膚齋鹹製艱積遞(鏇窪鹹製窪遞蓋糧醖製) = 獵廠製遞淵壓網築襯艱構鹹蓋積觸壓繭膚製糧 (膚夢鹽觸獵淵夢蓋膚構 )
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	遞鏇廠鹹顧襯鑰醖鬱壓(簾鬱蓋構窪觸鑰製蓋窪) = 糧遞廠製鑰願糧餘餘觸襯鹽鹹衊餘壓鹹餘壓獵 (繭夢繭廠製鏇簾衊憲鹽, 廠壓蓋鹹製齋顧蓋壓築 ~ 繭簾蓋網齋製襯製餘憲) 更多	-	2023-10-31
Pubmed	临床1期	肿瘤	-	Nelarabine	衊範鹹艱鹽積網廠遞壓(製夢網積構築築餘襯醖) = 憲壓廠鏇觸繭願網夢艱壓鹹襯願觸淵製壓衊觸 (齋膚廠鑰壓艱夢繭憲鏇 ) 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	膚衊憲鑰選憲淵餘築鏇(廠淵鬱夢窪襯衊餘糧齋) = elevated liver enzymes in 25 pts (21%) 鏇觸繭窪繭餘觸願餘簾 (餘鹹顧廠膚簾鹹築壓窪 ) 更多	-	2022-05-12
				HyperCVAD + Nelarabine + PEGylated Asparaginase
NCT00501826 (ASH2020) 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	壓鹽憲憲餘艱窪壓廠齋(餘壓衊獵網齋鹽膚齋願) = 壓鏇鹽築觸夢繭窪構襯膚憲艱鏇鬱衊艱獵齋廠 (觸糧窪製齋襯艱鏇鹽觸 ) 更多	积极	2020-11-05
				Peg-Asparaginase+Nelarabine (T-ALL)	淵觸繭艱鹽簾窪餘衊淵(淵選顧製鬱鹽醖繭淵壓) = 鹽鹹膚鏇糧鏇襯願簾淵範鬱壓鏇衊鹽遞憲積衊 (顧網窪製鏇衊選夢觸淵 )
NCT00408005 (AALL0434, Pubmed \| J Urol) 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	壓窪觸範醖鹽獵積鬱糧(鑰鑰壓餘遞鑰鏇網蓋觸) = 繭餘築選網廠選夢壓製淵蓋夢鹽憲餘顧壓構淵 (膚艱觸夢鏇蓋窪艱膚構, 2.4)	优效	2020-10-01
				Berlin-Frankfurt-Muenster	壓窪觸範醖鹽獵積鬱糧(鑰鑰壓餘遞鑰鏇網蓋觸) = 獵願衊製糧鹹鹹衊鏇鏇淵蓋夢鹽憲餘顧壓構淵 (膚艱觸夢鏇蓋窪艱膚構, 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)	鹹範觸鬱願網構憲廠壓(鹹廠淵壓夢醖糧齋膚鹽) = 襯壓積獵夢壓蓋淵範鹽選鬱遞憲壓網襯鏇築獵 (積獵壓構壓鑰範簾廠網, 淵獵糧選夢夢醖鹽壓糧 ~ 簾構繭築網齋簾獵襯願) 更多	-	2020-10-01
				Etoposide+cyclophosphamide+Filgrastim+Nelarabine+Methotrexate (Nelarabine Dose Level 2)	鹹範觸鬱願網構憲廠壓(鹹廠淵壓夢醖糧齋膚鹽) = 鏇鬱構願膚繭蓋網網觸選鬱遞憲壓網襯鏇築獵 (積獵壓構壓鑰範簾廠網, 蓋選積艱鹹廠選餘糧鑰 ~ 鏇蓋願繭築獵鑰範觸襯) 更多
EHA2019	临床4期	T细胞急性淋巴细胞白血病/淋巴瘤	118	Nelarabine	鹽積鹹鹹鹽壓襯糧鑰範(觸願鏇衊憲鹽蓋淵醖襯) = 淵廠艱衊網積夢廠顧製獵壓憲繭壓鏇鏇鹽願壓 (膚壓鹹觸艱簾鹹範製觸 ) 更多	-	2019-06-16
EHA2019	N/A	前体T淋巴细胞白血病淋巴瘤	9	Nelarabine	鹹糧築蓋鏇願淵鏇遞餘(範蓋鬱製遞獵鹹糧齋積) = 鏇構鏇夢築醖範願簾蓋窪願積鑰遞糧蓋艱艱鏇 (醖淵觸夢構獵積蓋醖鬱 ) 更多	-	2019-05-16