Development of Hematopoietic Stem Cell-Engineered Invariant Natural Killer T Cell Therapy for Cancer.

Journal: 
Cell Stem Cell
Publication Year: 
2019
Authors: 
Yanni Zhu
Drake J Smith
Yang Zhou
Yan-Ruide Li
Jiaji Yu
Derek Lee
Yu-Chen Wang
Stefano Di Biase
Xi Wang
Christian Hardoy
Josh Ku
Tasha Tsao
Levina J Lin
Alexander T Pham
Heesung Moon
Jami McLaughlin
Donghui Cheng
Roger P Hollis
Beatriz Campo-Fernandez
Fabrizia Urbinati
Liu Wei
Larry Pang
Valerie Rezek
Beata Berent-Maoz
Mignonette H Macabali
David Gjertson
Xiaoyan Wang
Zoran Galic
Scott G Kitchen
Dong Sung An
Siwen Hu-Lieskovan
Paula J Kaplan-Lefko
Satiro N De Oliveira
Christopher S Seet
Sarah M Larson
Stephen J Forman
James R Heath
Jerome A Zack
Gay M Crooks
Caius G Radu
Antoni Ribas
Donald B Kohn
Owen N Witte
Lili Yang
PubMed link: 
31495780
Public Summary: 
Invariant natural killer T (iNKT) cells are potent immune cells for targeting cancer; however, their clinical application has been hindered by their low numbers in cancer patients. Here, we developed a proof-of-concept for hematopoietic stem cell-engineered iNKT (HSC-iNKT) cell therapy with the potential to provide therapeutic levels of iNKT cells for a patient's lifetime. Using a human HSC engrafted mouse model and a human iNKT TCR gene engineering approach, we demonstrated the efficient and long-term generation of HSC-iNKT cells in vivo. These HSC-iNKT cells closely resembled endogenous human iNKT cells, could deploy multiple mechanisms to attack tumor cells, and effectively suppressed tumor growth in vivo in multiple human tumor xenograft mouse models. Preclinical safety studies showed no toxicity or tumorigenicity of the HSC-iNKT cell therapy. Collectively, these results demonstrated the feasibility, safety, and cancer therapy potential of the proposed HSC-iNKT cell therapy and laid a foundation for future clinical development.
Scientific Abstract: 
Invariant natural killer T (iNKT) cells are potent immune cells for targeting cancer; however, their clinical application has been hindered by their low numbers in cancer patients. Here, we developed a proof-of-concept for hematopoietic stem cell-engineered iNKT (HSC-iNKT) cell therapy with the potential to provide therapeutic levels of iNKT cells for a patient's lifetime. Using a human HSC engrafted mouse model and a human iNKT TCR gene engineering approach, we demonstrated the efficient and long-term generation of HSC-iNKT cells in vivo. These HSC-iNKT cells closely resembled endogenous human iNKT cells, could deploy multiple mechanisms to attack tumor cells, and effectively suppressed tumor growth in vivo in multiple human tumor xenograft mouse models. Preclinical safety studies showed no toxicity or tumorigenicity of the HSC-iNKT cell therapy. Collectively, these results demonstrated the feasibility, safety, and cancer therapy potential of the proposed HSC-iNKT cell therapy and laid a foundation for future clinical development.