Journal
NATURE COMMUNICATIONS
Volume 11, Issue 1, Pages -Publisher
NATURE PUBLISHING GROUP
DOI: 10.1038/s41467-020-17175-8
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Funding
- Stand Up To Cancer (SU2C)-St. Baldrick's Pediatric Cancer Dream Team Translational Research Grant [SU2C-AACR-DT1113]
- Cancer Prevention Research Institute of Texas (CPRIT) [RP101335]
- Alex's Lemonade Stand Pediatric Cancer Foundation
- V Foundation for Cancer Research
- Triumph Over Kid Cancer Foundation
- Cookies for Kids' CancerTM Foundation
- Clinical Research Center at Texas Children's Hospital
- Dan L. Duncan Cancer Center Support Grant [P30CA125123]
- National Cancer Institute of the National Institute for Health under the Cancer Moonshot U54 project [1U54CA232568-01]
- State of Texas CPRIT training program [RP160283]
- National Cancer Institute of the National Institutes for Health [K12CA090433]
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Refractory metastatic rhabdomyosarcoma is largely incurable. Here we analyze the response of a child with refractory bone marrow metastatic rhabdomyosarcoma to autologous HER2 CAR T cells. Three cycles of HER2 CAR T cells given after lymphodepleting chemotherapy induces remission which is consolidated with four more CAR T-cell infusions without lymphodepletion. Longitudinal immune-monitoring reveals remodeling of the T-cell receptor repertoire with immunodominant clones and serum autoantibodies reactive to oncogenic signaling pathway proteins. The disease relapses in the bone marrow at six months off-therapy. A second remission is achieved after one cycle of lymphodepletion and HER2 CAR T cells. Response consolidation with additional CAR T-cell infusions includes pembrolizumab to improve their efficacy. The patient described here is a participant in an ongoing phase I trial (NCT00902044; active, not recruiting), and is 20 months off T-cell infusions with no detectable disease at the time of this report. Recurrent metastatic rhabdomyosarcoma remains largely incurable. Here, the authors describe a child with metastatic rhabdomyosarcoma who has durable response to HER2-specific CAR T cells and shows endogenous immune reactivity.
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