Trial reportMolecular therapy : the journal of the American Society of Gene Therapy2024
Case study of CD19 CAR T therapy in a subject with immune-mediate necrotizing myopathy treated in the RESET-Myositis phase I/II trial.
Trial report in Molecular therapy : the journal of the American Society of Gene Therapy, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT06154252 (A Phase 1/2, Open-Label Study to Evaluate the Safety and Efficacy of Autologous CD19-specific Chimeric Antigen Receptor T Cells), which is not on this map. Cited by 31 papers, 3 of them syntheses that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
A Phase 1/2, Open-Label Study to Evaluate the Safety and Efficacy of Autologous CD19-specific Chimeric Antigen Receptor T Cells (CABA-201) in Subjects With Active Idiopathic Inflammatory Myopathy or Juvenile Idiopathic Inflammatory Myopathy
Who cites it
31 citing papers in PubMed, 3 syntheses or guidelines pooled it.
- Safety profiles of CAR-T cell therapy in systematic autoimmune diseases: a systematic review and analysis.Immunologic research · 2026Pooled it
- A new hope with CAR T-cell therapy for refractory idiopathic inflammatory myopathies: a systematic review.Journal of translational medicine · 2026Pooled it
- Safety and efficacy of CAR-T cell therapy in patients with autoimmune diseases: a systematic review.Rheumatology international · 2025Pooled it
- Trial
- Chimeric antigen receptor T (CAR-T) cell therapy in non-oncological diseases.Signal transduction and targeted therapy · 2026Review
- CAR-T therapy for immune-mediated necrotizing myopathy: a review of mechanisms and emerging clinical evidence.Clinical rheumatology · 2026Review
- Immunopathogenesis of Immune-Mediated Necrotizing Myopathy: Insights Drawn from Pathological Phenotypes and Mechanistic Elaboration.Biomedicines · 2026Review
- Emerging therapies in idiopathic inflammatory myopathies.Journal of neuromuscular diseases · 2026Review
- Clinical progress of engineered cellular immunotherapies for autoimmunity.Nature biotechnology · 2026Review
- Roads and detours for CAR T cell therapy in autoimmune diseases.Nature reviews. Drug discovery · 2026Review
- Biomarker-empowered precision navigation of CAR-T cell therapy.Molecular cancer · 2026Review
- Case study of resecabtagene autoleucel in a subject with diffuse cutaneous systemic sclerosis treated in the RESET-SSc trial.Molecular therapy. Advances · 2026Article
- CAR-T therapy for autoimmune rheumatic diseases: navigating clinical frontiers between breakthroughs and uncertainties.Clinical and experimental medicine · 2026Review
- Autoantibody internalization in myositis skeletal muscle: Emerging evidence, mechanistic insights, and therapeutic relevance.Autoimmunity reviews · 2026Review
- CAR-T and BiTE: new horizons in the treatment of rheumatic autoimmune diseases.Frontiers in immunology · 2026Review
- B cell targeted CAR-T therapy for autoimmune diseases.Frontiers of medicine · 2025Review
- Autoimmune Neuromuscular Disorders at a Molecular Crossroad: Linking Pathogenesis to Targeted Immunotherapy.International journal of molecular sciences · 2025Review
- CAR-T cells targeting CD19 for the treatment of ANCA vasculitis.Clinical kidney journal · 2025Article
- Targeted Therapy for Idiopathic Inflammatory Myopathy.Journal of cachexia, sarcopenia and muscle · 2025Review
- The Effect of Anti-3-Hydroxy-3-Methylglutaryl-CoA Reductase (HMGCR) Human Autoantibodies on Muscle Regeneration in Mice.Muscle & nerve · 2025Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
18 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Under compassionate use, chimeric antigen receptor (CAR) T cells have elicited durable remissions in patients with refractory idiopathic inflammatory myopathies (IIMs). Here, we report on the safety, efficacy, and correlative data of the first subject with the immune-mediated necrotizing myopathy (IMNM) subtype of IIM who received a fully human, 4-1BBz anti-CD19-CAR T cell therapy (CABA-201) in the RESET-Myositis phase I/II trial (NCT06154252). CABA-201 was well-tolerated following infusion. Notably, no evidence of cytokine release syndrome or immune effector cell-associated neurotoxicity syndrome was observed. Creatine kinase levels decreased, and muscular strength improved post-infusion. Peripheral B cells were depleted rapidly following infusion, and the subject achieved peripheral B cell aplasia by day 15 post-infusion. Peripheral B cells returned at 2 months post-infusion and were almost entirely transitional. Autoantibodies to SRP-9, SRP-72, SRP-54, and Ro-52, decreased relative to baseline, whereas antibodies associated with pathogens and vaccinations remained stable. The infusion product consisted of predominantly CD4
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.