{"entity":{"id":"paper-nxc-201-hbi0101-asherie-haematologica-2023","kind":"paper","name":"Development and manufacture of novel locally produced anti-BCMA CAR T cells for the treatment of relapsed/refractory multiple myeloma: results from a phase I clinical trial (HBI0101)","aka":[],"tldr":"A hospital in Jerusalem built and made its own CAR-T cells against the myeloma protein BCMA; in the first 20 heavily treated patients, 75 percent responded and half went into complete remission, with only mild immune reactions.","summary":"Report of the Hadassah phase 1 dose-escalation study (NCT04720313) of HBI0101, a second-generation optimised anti-BCMA CAR-T therapy developed in an academic setting and given fresh without cryopreservation. Twenty heavily pretreated patients with relapsed or refractory multiple myeloma were treated in three cohorts: 150 million (n = 6), 450 million (n = 7) and 800 million (n = 7) CAR-T cells.\n\nGrade 1 or 2 cytokine release syndrome occurred in 18 patients (90 percent); no grade 3 or 4 cytokine release syndrome, no neurotoxicity of any grade and no dose-limiting toxicity were observed. The overall response rate was 75 percent, (stringent) complete response 50 percent and very good partial response 25 percent; responses were dose dependent, with 85 percent overall response, 71 percent complete response and 57 percent minimal residual disease negativity in the high-dose cohort. Median overall survival was 308 days (range 25 to more than 466), with estimated survival of 55 percent at the data cutoff; median progression-free survival was 160 days, with six patients progression free at the cutoff. The authors argue that the results support decentralised CAR-T production in academic centres to meet local demand.","asOf":"2026-09-24","links":[{"label":"Haematologica 2023","url":"https://doi.org/10.3324/haematol.2022.281628"},{"label":"PubMed","url":"https://pubmed.ncbi.nlm.nih.gov/36200421/"},{"label":"ClinicalTrials.gov NCT04720313","url":"https://clinicaltrials.gov/study/NCT04720313"}],"tags":[],"related":[],"cancers":["multiple-myeloma"],"sections":[],"technologies":["car-t"],"targets":["bcma"],"drugs":["nxc-201-car-t"],"companies":[],"institutions":["hadassah"],"pathways":[],"terms":[],"trials":["nxc-201-mm"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":["haematologica"],"dependsOn":[],"notes":[],"journal":"Haematologica","year":2023,"doi":"10.3324/haematol.2022.281628","pmid":"36200421","authors":"Asherie N, Kfir-Erenfeld S, Avni B, et al.","paperType":"observational","findings":["Overall response rate 75 percent, (stringent) complete response 50 percent and very good partial response 25 percent in 20 patients.","Grade 1 to 2 cytokine release syndrome in 90 percent; no grade 3 to 4 cytokine release syndrome, neurotoxicity or dose-limiting toxicity.","Dose-dependent responses: 85 percent response, 71 percent complete response and 57 percent MRD negativity at 800 million cells; median progression-free survival 160 days."],"whatItMeans":"The clinical result is in line with commercial BCMA CAR-T products, but the point of the paper is the manufacturing: an academic hospital produced its own CAR-T cells, infused them fresh and treated patients who would otherwise wait for a commercial slot. It is the evidence base for what Immix Biopharma now develops as NXC-201.","caveats":["Phase 1 in 20 patients at a single centre; follow-up was short and the median progression-free survival of about five months is modest.","No comparison group; the historical comparators are the pivotal trials of idecabtagene vicleucel and ciltacabtagene autoleucel.","The registry lists a much larger continuing enrolment, so later cohorts are not described here."],"changedPractice":false,"participants":20},"route":"/key-papers/paper-nxc-201-hbi0101-asherie-haematologica-2023/","neighbours":{"cancer":[{"id":"multiple-myeloma","kind":"cancer","name":"Multiple myeloma","route":"/cancers/multiple-myeloma/"}],"technology":[{"id":"car-t","kind":"technology","name":"CAR-T cell therapy","route":"/technologies/car-t/"}],"target":[{"id":"bcma","kind":"target","name":"BCMA","route":"/targets/bcma/"}],"drug":[{"id":"nxc-201-car-t","kind":"drug","name":"NXC-201 CAR-T","route":"/drugs/nxc-201-car-t/"}],"institution":[{"id":"hadassah","kind":"institution","name":"Hadassah Medical Center","route":"/institutions/hadassah/"}],"trial":[{"id":"nxc-201-mm","kind":"trial","name":"HBI0101 (NXC-201) multiple myeloma phase 1","route":"/trials/nxc-201-mm/"}],"journal":[{"id":"haematologica","kind":"journal","name":"Haematologica","route":"/journals/haematologica/"}]}}