Gamma-c gene therapy hematology data

Researchers at the hospital and colleagues reported long-term follow-up data from 9 boys treated with ex vivo gamma-c gene therapy showing that 8 patients were alive at a median follow-up of 9 years. Four patients developed acute leukemia, 1 of whom died. Transduced T cells were detected for up to 10.7 years following the gene therapy. Seven patients had sustained immune reconstitution, 3 of whom required immunoglobulin-replacement therapy.

A tenth patient treated in Australia had insufficient T cell reconstitution and was given an allograft hematopoietic stem cell transplant 26 months after gene therapy. That patient died from fungal pneumonia 18 months after the allograft. Data were published in the New England Journal of Medicine. ...