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  1. No Access

    Article

    Enhancing pediatric access to cell and gene therapies

    Increasing numbers of cell and gene therapies (CGTs) are emerging to treat and cure pediatric diseases. However, small market sizes limit the potential return on investment within the traditional biopharmaceut...

    Crystal L. Mackall, Catherine M. Bollard, Nancy Goodman, Casey Carr in Nature Medicine (2024)

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    Article

    Unanswered questions following reports of secondary malignancies after CAR-T cell therapy

    Reports of T cell malignancies after CAR-T cell therapy should be investigated, but existing data from follow-up studies suggest a low risk compared with other cancer treatments.

    Bruce L. Levine, Marcelo C. Pasquini, John E. Connolly, David L. Porter in Nature Medicine (2024)

  3. No Access

    Article

    Tumor inflammation-associated neurotoxicity

    Cancer immunotherapies have unique toxicities. Establishment of grading scales and standardized grade-based treatment algorithms for toxicity syndromes can improve the safety of these treatments, as observed f...

    Jasia Mahdi, Jorg Dietrich, Karin Straathof, Claire Roddie in Nature Medicine (2023)

  4. Article

    Post-infusion CAR TReg cells identify patients resistant to CD19-CAR therapy

    Approximately 60% of patients with large B cell lymphoma treated with chimeric antigen receptor (CAR) T cell therapies targeting CD19 experience disease progression, and neurotoxicity remains a challenge. Biom...

    Zinaida Good, Jay Y. Spiegel, Bita Sahaf, Meena B. Malipatlolla in Nature Medicine (2022)

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    Article

    Anti-GD2 synergizes with CD47 blockade to mediate tumor eradication

    The disialoganglioside GD2 is overexpressed on several solid tumors, and monoclonal antibodies targeting GD2 have substantially improved outcomes for children with high-risk neuroblastoma. However, approximate...

    Johanna Theruvath, Marie Menard, Benjamin A. H. Smith, Miles H. Linde in Nature Medicine (2022)

  6. Article

    Open Access

    CAR T cells with dual targeting of CD19 and CD22 in adult patients with recurrent or refractory B cell malignancies: a phase 1 trial

    Despite impressive progress, more than 50% of patients treated with CD19-targeting chimeric antigen receptor T cells (CAR19) experience progressive disease. Ten of 16 patients with large B cell lymphoma (LBCL)...

    Jay Y. Spiegel, Shabnum Patel, Lori Muffly, Nasheed M. Hossain, Jean Oak in Nature Medicine (2021)

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    Article

    Locoregionally administered B7-H3-targeted CAR T cells for treatment of atypical teratoid/rhabdoid tumors

    Atypical teratoid/rhabdoid tumors (ATRTs) typically arise in the central nervous system (CNS) of children under 3 years of age. Despite intensive multimodal therapy (surgery, chemotherapy and, if age permits, ...

    Johanna Theruvath, Elena Sotillo, Christopher W. Mount in Nature Medicine (2020)

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    Article

    Clinical lessons learned from the first leg of the CAR T cell journey

    Chimeric antigen receptor (CAR) T cell therapy for B cell malignancies has surpassed expectations, driving an ever-expanding number of clinical trials and the first US Food and Drug Administration approvals of...

    Robbie G. Majzner, Crystal L. Mackall in Nature Medicine (2019)

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    Article

    Potent antitumor efficacy of anti-GD2 CAR T cells in H3-K27M+ diffuse midline gliomas

    Diffuse intrinsic pontine glioma (DIPG) and other diffuse midline gliomas (DMGs) with mutated histone H3 K27M (H3-K27M)15 are aggressive and universally fatal pediatric brain cancers6. Chimeric antigen receptor ...

    Christopher W. Mount, Robbie G. Majzner, Shree Sundaresh, Evan P. Arnold in Nature Medicine (2018)

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    Article

    CD22-targeted CAR T cells induce remission in B-ALL that is naive or resistant to CD19-targeted CAR immunotherapy

    Fry et al. report the first results from a human trial of a CD22-directed chimeric antigen receptor (CAR) T cell therapy providing evidence of efficacy in the treatment of pre–B cell acute lymphoblastic leukemia ...

    Terry J Fry, Nirali N Shah, Rimas J Orentas, Maryalice Stetler-Stevenson in Nature Medicine (2018)

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    Article

    4-1BB costimulation ameliorates T cell exhaustion induced by tonic signaling of chimeric antigen receptors

    Crystal Mackall and colleagues report that antigen-independent signaling of chimeric antigen receptors (CARs) causes T cell exhaustion and reduced therapeutic efficacy of CAR T cells that can be overcome by in...

    Adrienne H Long, Waleed M Haso, Jack F Shern, Kelsey M Wanhainen in Nature Medicine (2015)

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    Article

    Lymphopenia and interleukin-2 therapy alter homeostasis of CD4+CD25+ regulatory T cells

    CD4+CD25+ regulatory T (Treg) cells have a crucial role in maintaining immune tolerance. Mice and humans born lacking Treg cells develop severe autoimmune disease1,2, and depletion of Treg cells in lymphopenic mi...

    Hua Zhang, Kevin S Chua, Martin Guimond, Veena Kapoor, Margaret V Brown in Nature Medicine (2005)