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#细胞治疗

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Sep 2Wed
  1. Nat Med59

    Intramyocardial injection of allogeneic human induced pluripotent stem cell-derived cardiomyocytes in advanced ischemic heart failure: an early-stage randomized trial.

    Allogeneic human induced pluripotent stem cell-derived cardiomyocytes injected into the myocardium during CABG met the safety primary endpoint in the HEAL-CHF trial (n = 20): no sustained ventricular tachycardia in the 1- to 6-month window and no tumorigenicity at 12 months. All 10 cell-treated patients developed accelerated idioventricular rhythm within 4 weeks, and 2 had ventricular tachycardia over 140 bpm that resolved after cardioversion. Cell therapy improved 6-min walk distance, myocardial perfusion and relative wall thickening beyond CABG alone, but not ejection fraction, ventricular volumes, scar size, NYHA class or quality-of-life scores.

    Why it matters: First randomized evidence on safety and early efficacy of allogeneic hiPSC-derived cardiomyocyte injection in ischemic heart failure.

Aug 27Thu
  1. Nat Med50

    CD19 CAR T cell therapy for treatment-refractory seropositive rheumatoid arthritis: a phase 1 trial.

    A single infusion of mivocabtagene autoleucel, a fully human CD19 CAR T cell therapy, depleted CD19+ B cells in blood and tissue of six patients with severe, treatment-refractory, ACPA-positive rheumatoid arthritis after lymphodepletion. CRS occurred in all six but was limited to grade 1 and 2, with no ICANS or serious adverse events. Disease activity improved in all patients off immunosuppression, with a median 34% DAS28-CRP reduction, DAS28-CRP remission and ACR70 response in 3 of 6, and ACPA seroconversion in 4 of 6. The primary safety endpoint was met, allowing advancement to phase 2.

Aug 25Tue
  1. Lancet62

    Deramiocel heart-derived cellular therapy in advanced Duchenne muscular dystrophy (HOPE-3): a phase 3, randomised, double-blind, placebo-controlled trial.

    Intravenous deramiocel, a heart-derived cellular therapy, slowed disease progression in advanced Duchenne muscular dystrophy in the phase 3 HOPE-3 trial. Among 106 participants aged 10 years or older, total Performance of the Upper Limb 2.0 percentage change at 12 months significantly favoured deramiocel over placebo, with a safety profile similar to placebo. Quarterly outpatient infusions preserved skeletal muscle function regardless of the underlying genetic lesion.

    Why it matters: Phase 3 evidence that a quarterly outpatient cell infusion slows upper limb function loss in advanced DMD.

Aug 19Wed
  1. nature.com56

    Intramyocardial injection of allogeneic human induced pluripotent stem cell-derived cardiomyocytes in advanced ischemic heart failure: an early-stage randomized trial

    Intramyocardial injection of allogeneic human induced pluripotent stem cell-derived cardiomyocytes was tested in patients with advanced ischemic heart failure in an early-stage randomized trial. The trial evaluated safety and efficacy of the cell therapy delivered directly into the heart muscle. No results are reported here.

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