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MHRA Grants Conditional Approval for CAR-T Therapy Aucatzyl to Treat Relapsed B-Cell ALL

CAR-TDrug Approval
  • The MHRA granted conditional marketing authorization for obecabtagene autoleucel (Aucatzyl), a CAR-T cell therapy for adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia on April 25, 2025.
  • The European Medicines Agency's CHMP adopted a positive opinion for Aucatzyl on May 22, 2025, recommending conditional approval for adults aged 26 years and older with the same indication.
  • B-cell precursor ALL is a blood cancer affecting white blood cells where bone marrow produces abnormal, immature B-lymphocytes that grow and divide rapidly.
  • This approval represents a significant advancement in CAR-T cell therapy for treating this aggressive form of blood cancer in patients who have failed previous treatments.

Real-World Data Confirms Low-Severity Safety Profile for Lisocabtagene Maraleucel Across Blood Cancer Settings

CAR-TReal World Evidence
  • Real-world data from 877 patients and clinical trial data from 702 patients demonstrate that most cytokine release syndrome and neurological events with lisocabtagene maraleucel occur within 15 days of infusion and are not severe.
  • Among patients experiencing late-onset adverse events after day 15, the vast majority were grade 1/2 events that resolved without requiring intensive care unit-level management.
  • The findings support optimized monitoring strategies that could improve patient access to CAR-T therapy while maintaining safety standards across multiple blood cancer indications.

Lisocabtagene Maraleucel (JCAR017) as Second-Line Therapy (TRANSCEND-PILOT-017006)

NCT03483103CompletedPhase 2
Juno Therapeutics, a Subsidiary of Celgene
Posted 7/27/2018

Study Evaluating the Safety and Pharmacokinetics of JCAR017 in B-cell Non-Hodgkin Lymphoma (TRANSCEND-NHL-001)

NCT02631044CompletedPhase 1
Juno Therapeutics, a Subsidiary of Celgene
Posted 1/6/2016

A Study to Evaluate the Efficacy and Safety of JCAR017 in Adult Subjects With Relapsed or Refractory Indolent B-cell Non-Hodgkin Lymphoma (NHL)

NCT04245839RecruitingPhase 2
Celgene
Posted 7/14/2020

A Study to Compare the Efficacy and Safety of JCAR017 to Standard of Care in Adult Subjects With High-risk, Transplant-eligible Relapsed or Refractory Aggressive B-cell Non-Hodgkin Lymphomas

NCT03575351CompletedPhase 3
Celgene
Posted 10/23/2018

Study Evaluating Safety and Efficacy of JCAR017 in Subjects With Relapsed or Refractory Chronic Lymphocytic Leukemia (CLL) or Small Lymphocytic Lymphoma (SLL)

NCT03331198RecruitingPhase 1
Juno Therapeutics, a Subsidiary of Celgene
Posted 11/27/2017

Real-World Data Shows Yescarta CAR-T Therapy Safe and Effective in Outpatient Setting

CAR-TReal World Evidence
  • Kite presented real-world data from 238 patients showing comparable safety and effectiveness outcomes for Yescarta CAR-T therapy administered in outpatient versus inpatient settings for relapsed/refractory large B-cell lymphoma.
  • The analysis found no significant differences in rates of cytokine release syndrome, neurologic events, or immune effector cell-associated neurotoxicity syndrome grade ≥3 between treatment settings.
  • Nearly 25% of outpatient-assigned patients avoided hospital admission within 30 days, and 50% avoided admission within 3 days, demonstrating potential cost-effective advantages.
  • The study supports expanding access to this potentially curative one-time treatment while reducing healthcare system burden and improving patient convenience.

Study of Effectiveness of Axicabtagene Ciloleucel Compared to Standard of Care Therapy in Patients With Relapsed/Refractory Diffuse Large B Cell Lymphoma

NCT03391466CompletedPhase 3
Kite, A Gilead Company
Posted 1/25/2018

Gilead's Dual-Target CAR-T Therapy Achieves 62% Response Rate in Recurrent Glioblastoma

CAR-TOncology
  • Gilead Sciences' dual-target CAR-T therapy shrank tumors in 62% of patients with recurrent glioblastoma, a rare achievement for this fatal brain cancer.
  • The experimental treatment targets both EGFR and interleukin-13 receptor alpha 2, injected directly into spinal fluid to overcome glioblastoma's multiple tumor cell populations.
  • While responses were encouraging, benefits proved largely temporary with many patients relapsing within two to three months after treatment.
  • Kite is developing a triple-target version to improve persistence and plans to test the therapy in newly diagnosed patients next year.

Dual-Target CAR T Cell Therapy Achieves Tumor Shrinkage in 62% of Recurrent Glioblastoma Patients

CAR-T
  • A novel dual-target CAR T cell therapy targeting EGFR and IL13Rα2 proteins achieved tumor shrinkage in 62% of patients with recurrent glioblastoma, the most aggressive form of brain cancer.
  • The experimental treatment delivered directly into cerebrospinal fluid showed encouraging survival signals, with 43% of patients surviving beyond 12 months compared to typical 6-10 month survival rates.
  • Penn researchers established the therapy's safety profile and maximum tolerated dose, paving the way for upcoming clinical trials in newly diagnosed glioblastoma patients.

A Study Evaluating Temferon in Patients with Glioblastoma & Unmethylated MGMT

NCT03866109RecruitingPhase 1
Genenta Science
Posted 3/5/2019

CART-EGFR-IL13Ra2 in Newly Diagnosed GBM Following Initial Radiotherapy

NCT06973096RecruitingPhase 1
University of Pennsylvania
Posted 7/18/2025

Dual-Targeted CAR-T Therapy KITE-363 Shows 87% Response Rate in Relapsed B-Cell Lymphoma

CAR-TOncology
  • KITE-363, a dual CD19/CD20-targeted CAR-T therapy, achieved an 87% objective response rate with 78% complete responses in CAR-T naive patients with relapsed/refractory large B-cell lymphoma.
  • The therapy demonstrated superior CAR-T cell expansion compared to existing treatments, with peak levels 3-5 fold higher than axicabtagene ciloleucel.
  • Safety profile showed manageable toxicity with no grade 3 or 4 cytokine release syndrome or neurotoxicity in primary refractory patients at the recommended dose.
  • Primary refractory patients achieved 80% response rates with 67% complete responses, addressing a critical unmet need in this difficult-to-treat population.

Study of KITE-363 or KITE-753 in Participants With Relapsed and/or Refractory B-cell Lymphoma

NCT04989803RecruitingPhase 1
Kite, A Gilead Company
Posted 10/27/2021

CAR-T Cell Therapy Shows Promise for Severe Autoimmune Neurological Diseases in Pioneering European Studies

CAR-T
  • German physicians at Ruhr-University Bochum successfully treated two patients with chronic inflammatory demyelinating polyneuropathy (CIDP) using CAR-T cell therapy, achieving over 200% improvement in clinical scores and neurophysiological tests.
  • The treatment specifically targets pathogenic B-cells responsible for autoimmune attacks on the peripheral nervous system, with patients regaining mobility within days and requiring no further immune therapy after a single treatment.
  • Dutch researchers at LUMC became the first in the Netherlands to treat an autoimmune disease patient with CAR-T therapy, successfully eliminating autoantibodies in a neurological lupus case and enabling discontinuation of immunosuppressive treatment.
  • Both studies represent groundbreaking applications of cancer immunotherapy to severe autoimmune neurological conditions, offering new hope for treatment-resistant patients who have exhausted conventional therapeutic options.

Novel CAR T-Cell Therapy ALLO-316 Shows 33% Response Rate in Advanced Renal Cell Carcinoma

CAR-T
  • ALLO-316, a novel CAR T-cell therapy targeting CD70, demonstrated a 33% confirmed objective response rate in patients with advanced clear cell renal cell carcinoma whose tumors expressed CD70 in at least 50% of cells.
  • The TRAVERSE first-in-human trial enrolled 44 patients who underwent lymphodepletion, with 39 receiving ALLO-316 infusion, targeting patients who had failed checkpoint inhibitors and tyrosine kinase inhibitors.
  • Safety profile showed manageable toxicity with 25 patients developing cytokine-release syndrome and only 2 patients experiencing dose-limiting toxicities, with no cases of graft-versus-host disease observed.
  • The therapy addresses a significant unmet medical need for metastatic clear cell renal cell carcinoma patients with limited alternative treatment options after standard therapies fail.

A Safety and Efficacy Study Evaluating CTX130 in Subjects With Relapsed or Refractory Renal Cell Carcinoma (COBALT-RCC)

NCT04438083TerminatedPhase 1
CRISPR Therapeutics AG
Posted 6/16/2020

Safety and Efficacy of ALLO-316 in Subjects With Advanced or Metastatic Clear Cell Renal Cell Carcinoma

NCT04696731RecruitingPhase 1
Allogene Therapeutics
Posted 2/24/2021

Universal CAR-T Cell Therapy Achieves 91% Response Rate in Aggressive T Cell Cancers

CAR-T
  • An international Phase I/II trial of WU-CART-007, a universal CAR-T cell therapy, demonstrated a 91% overall response rate in patients with relapsed or refractory T cell acute lymphoblastic leukemia and T cell lymphoblastic lymphoma.
  • The therapy achieved complete remission in 72.7% of evaluable patients who received the full dose, significantly outperforming standard-of-care treatments that typically achieve only 20%-40% remission rates.
  • Using CRISPR gene editing technology, this "off-the-shelf" therapy can be manufactured from healthy donor cells and made readily available, eliminating the 3-6 week manufacturing delay of traditional CAR-T therapies.
  • Most patients experienced manageable cytokine release syndrome, with 88.5% having mild to moderate cases and only 19% experiencing more severe reactions.

Myeloid Therapeutics Reports First-in-Human Data for In Vivo mRNA CAR Therapies at ASCO 2025

CAR-T
  • Myeloid Therapeutics presented first-in-human data for MT-302 and MT-303, marking the first clinical applications of systemically administered in vivo mRNA CAR therapies.
  • Single-cell RNA sequencing demonstrated selective CAR expression in myeloid cells with increased pro-inflammatory gene signatures across tumor types for MT-302.
  • The therapies eliminate the need for ex vivo cell manipulation while delivering tumor-specific immune activation and demonstrating successful tumor penetration.
  • Both MT-302 targeting TROP2 and MT-303 targeting GPC3 showed favorable safety profiles and confirmed target engagement through pharmacodynamic markers.

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