Drug intelligence / Profile preview

autologous MGMT P140K-transduced hematopoietic stem cells (Fred Hutch)

Development stage
Unknown
Lead developer
Fred Hutchinson Cancer Center
Modality
Stem Cell Therapies → Cell Therapies, Gene Therapies
Administration
Intravenous
01

Overview

Autologous transduced hematopoietic cells developed by Fred Hutchinson Cancer Center is an experimental ex vivo gene therapy for the treatment of HIV-associated Hodgkin and non-Hodgkin lymphoma. The therapy involves the collection of a patient's own peripheral blood stem cells, which are then genetically modified using a lentiviral vector to express a gene conferring HIV resistance and a mutant form of the O6-methylguanine-DNA methyltransferase (MGMT P140K) gene. Following high-dose chemotherapy and autologous transplantation of these modified cells, patients receive treatment with O6-benzylguanine (O6BG) and carmustine (BCNU). The MGMT P140K mutation protects the modified cells from the toxic effects of this combination, allowing for the in vivo enrichment of the HIV-resistant cell population. This approach aims to reconstitute the immune system with cells that are refractory to HIV infection while simultaneously treating the underlying lymphoma.

Other names
MGMT P140K-transduced HSCsHIV-resistant hematopoietic stem cell therapy

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