新型人源化 CD19-CAR-T（现称 talicabtagene autoleucel，Tali-cel™）治疗复发难治儿童 B 急性淋巴细胞白血病：开放标签单臂 1/1b 期研究
Novel humanized CD19-CAR-T (Now talicabtagene autoleucel, Tali-cel™) cells in relapsed/ refractory pediatric B-acute lymphoblastic leukemia- an open-label single-arm phase-I/Ib study
嵌合抗原受体（CAR）T 细胞疗法对复发难治 B 急性淋巴细胞白血病有效，但并非所有患者都能获得。研究者开发了新型人源化 CD19 CAR T 细胞（HCAR19），在 12 名 3 至 25 岁、不适合异基因移植的患者中开展 1/1b 期试验。总体缓解率 91.7%，完全缓解率 66.7%，较高剂量组缓解持续至 12 个月随访。细胞因子释放综合征多为 1 至 2 级。研究确定 2 期推荐剂量为每公斤 5 至 10×10^6 个细胞。
为什么推荐给您：属已知 CD19 CAR T 靶点的新构建体早期试验，为常规剂量探索，非全新模态。
不需要生物学背景，多打比方
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摘要Abstract
Chimeric Antigen Receptor-T (CAR-T) cell therapy is effective for relapsed/refractory B-acute lymphoblastic leukemia (r/r B-ALL) but is not universally available. We developed a novel humanized CD19-directed CAR-T (HCAR19) approved for Phase 1/1b/2 trials. Patients aged 3-25 years were enrolled with r/r B-ALL and ineligible for allogeneic stem cell transplant. Lymphodepletion utilized standard-dose fludarabine and cyclophosphamide. A 3 + 3 design testing 3 dose-ranges was used to determine Phase-2 Dose (P2D): Dose-A, 1 × 10^6 HCAR19 cells/kg, Dose-B, 3-5 × 10^6/kg, and Dose-C, 10-15 × 10^6/kg. Primary endpoint was overall response rate (ORR) at day-30 on bone-marrow flow-cytometry. From May-2021 to September-2023 12 patients [median age-14 (range: 5-24) years] were enrolled with median bone marrow blasts 19.5% at screening. Cytokine release syndrome occurred in 10 (83%) patients, predominantly Grades 1-2, and Grade-2 immune-cell associated neurotoxicity (ICANS) in 1. All patients had Grade-3 cytopenia. ORR was 91.7% (11/12), complete response (CR) in 8 (66.7%) and partial response in 3 (25%). Seven of 8 CRs were at Dose-levels B and C, all of which were sustained till 12 months follow-up. Patients who received dose levels below 3 × 10^6/kg, or did not achieve CR, had early loss of response or rapid progression. HCAR19 demonstrated safety, manageable toxicity, and durable remissions. and P2D was determined as 5-10 × 10^6 HCAR19-cells/kg. CLINICAL TRIAL REGISTRATION: The study is registered in the Clinical Trials Registry- India (CTRI/2021/05/033348 and CTRI/2023/03/050689).