To overcome the myelosuppression that is a common side effect of chemotherapy, Adair et al. modified ex vivo the haematopoietic stem cells (HSCs) from three patients with glioblastoma to express a mutant form of 6-O-methylguanine-DNA methyltransferase (MGMT). This enzyme enhances the repair of DNA lesions caused by alkylating chemotherapies. Re-injection of the MGMT-modified HSCs, which successfully engrafted, enabled the patients to tolerate higher doses of chemotherapy, with some evidence of disease stabilization and improved survival.
ORIGINAL RESEARCH PAPER
Adair, J. E. et al. Extended survival of glioblastoma patients after chemoprotective HSC gene therapy. Sci. Transl Med. 4, 133ra57 (2012)Article
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Burgess, D. Enhancing efficacy by reducing side effects. Nat Rev Cancer 12, 377 (2012). https://doi.org/10.1038/nrc3292
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DOI: https://doi.org/10.1038/nrc3292