Antitumor activity without on-target off-tumor toxicity of GD2–chimeric antigen receptor T cells in patients with neuroblastoma

著者: Karin Straathof, Barry Flutter, Rebecca Wallace, Neha Jain, Thalia Loka, Sarita Depani, Gary Wright, Simon Thomas, Gordon Weng-Kit Cheung, Talia Gileadi, Sian Stafford, Evangelia Kokalaki, Jack Barton, Clare Marriott, Dyanne Rampling, Olumide Ogunbiyi, Ayse U. Akarca, Teresa Marafioti, Sarah Inglott, Kimberly Gilmour, Muhammad Al‐Hajj, William Day, Kieran McHugh, Lorenzo Biassoni, Natalie Sizer, Claire Barton, David Edwards, I. Dragoni, Julie Silvester, Karen Dyer, Stephanie Traub, Lily Elson, Susan Brook, Nigel Westwood, L Robson, Ami Bedi, Karen Howe, Ailish Barry, Catriona Duncan, Giuseppe Barone, Martin Pulé, John Anderson - Science Translational Medicine 2020 被引用: 205

CAR-T cells after fludarabine/cyclophosphamide conditioning, two experienced grade 2 to 3 cytokine release syndrome, and three demonstrated regression of soft tissue and bone marrow disease. This clinical activity was achieved without on-target off-tumor toxicity. Targeting neuroblastoma with GD2 CAR-T cells appears to be a valid and safe strategy but requires further modification to promote CAR-T cell longevity.