Journal
PLOS ONE
Volume 11, Issue 3, Pages -Publisher
PUBLIC LIBRARY SCIENCE
DOI: 10.1371/journal.pone.0152196
Keywords
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Categories
Funding
- Neuroblastoma UK
- UK NIHR Biomedical Research Centre
- Wellcome Trust
- Great Ormond Street Hospital, Biomedical Research Centre
- Great Ormond Street Hospital Children's Charity
- Cancer Research UK [14779] Funding Source: researchfish
- Great Ormond Street Hospital Childrens Charity [V1243, W1006] Funding Source: researchfish
- National Institute for Health Research [CL-2009-18-011] Funding Source: researchfish
- Wellcome Trust [102803/Z/13/Z] Funding Source: researchfish
- Wellcome Trust [102803/Z/13/Z] Funding Source: Wellcome Trust
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Neuroblastoma is the commonest extra cranial solid cancer of childhood. Despite escalation of treatment regimens, a significant minority of patients die of their disease. Disialogan-glioside (GD2) is consistently expressed at high-levels in neuroblastoma tumors, which have been targeted with some success using therapeutic monoclonal antibodies. GD2 is also expressed in a range of other cancer but with the exception of some peripheral nerves is largely absent from non-transformed tissues. Chimeric Antigen Receptors (CARs) are artificial type I proteins which graft the specificity of a monoclonal antibody onto a T-cell. Clinical data with early CAR designs directed against GD2 have shown some promise in Neuroblastoma. Here, we describe a GD2-targeting CAR retroviral cassette, which has been optimized for CAR T-cell persistence, efficacy and safety.
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