CRISPR-engineered T cells in patients with refractory cancer

Edward A. Stadtmauer(University of Pennsylvania), Joseph A. Fraietta(Parker Institute for Cancer Immunotherapy), Megan M. Davis(University of Pennsylvania), Adam D. Cohen(University of Pennsylvania), Kristy Weber(University of Pennsylvania), Eric Lancaster(University of Pennsylvania), Patricia Mangan(University of Pennsylvania), Irina Kulikovskaya(University of Pennsylvania), Minnal Gupta(University of Pennsylvania), Fang Chen(University of Pennsylvania), Lifeng Tian(University of Pennsylvania), Vanessa Gonzalez(University of Pennsylvania), Jun Xu(University of Pennsylvania), In-Young Jung(University of Pennsylvania), J. Joseph Melenhorst(Parker Institute for Cancer Immunotherapy), Gabriela Plesa(University of Pennsylvania), Joanne Shea(University of Pennsylvania), Tina Matlawski(University of Pennsylvania), Amanda Cervini(University of Pennsylvania), Avery L. Gaymon(University of Pennsylvania), Stephanie Desjardins(University of Pennsylvania), Anne Lamontagne(University of Pennsylvania), January Salas-McKee(University of Pennsylvania), Andrew D. Fesnak(University of Pennsylvania), Donald L. Siegel(University of Pennsylvania), Bruce L. Levine(University of Pennsylvania), Julie K. Jadlowsky(University of Pennsylvania), Regina M. Young(University of Pennsylvania), Anne Chew(University of Pennsylvania), Wei‐Ting Hwang(University of Pennsylvania), Elizabeth O. Hexner(University of Pennsylvania), Beatriz M. Carreno(Parker Institute for Cancer Immunotherapy), Christopher L. Nobles(University of Pennsylvania), Frederic D. Bushman(University of Pennsylvania), Kevin R. Parker(Stanford University), Yanyan Qi(Stanford University), Ansuman T. Satpathy(Stanford University), Howard Y. Chang(Howard Hughes Medical Institute), Yangbing Zhao(University of Pennsylvania), Simon F. Lacey(University of Pennsylvania), Carl H. June(Parker Institute for Cancer Immunotherapy)
Science
February 6, 2020
10.1126/science.aba7365
Cited by 1,438Open Access
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Abstract

CRISPR takes first steps in humans CRISPR-Cas9 is a revolutionary gene-editing technology that offers the potential to treat diseases such as cancer, but the effects of CRISPR in patients are currently unknown. Stadtmauer et al. report a phase 1 clinical trial to assess the safety and feasibility of CRISPR-Cas9 gene editing in three patients with advanced cancer (see the Perspective by Hamilton and Doudna). They removed immune cells called T lymphocytes from patients and used CRISPR-Cas9 to disrupt three genes ( TRAC, TRBC , and PDCD1 ) with the goal of improving antitumor immunity. A cancer-targeting transgene, NY-ESO-1, was also introduced to recognize tumors. The engineered cells were administered to patients and were well tolerated, with durable engraftment observed for the study duration. These encouraging observations pave the way for future trials to study CRISPR-engineered cancer immunotherapies. Science , this issue p. eaba7365 ; see also p. 976

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