Retroviral-mediated delivery of BRCA1 gene therapy (LXN-BRCA1sv, a nor
mal splice variant form of BRCA1) was tested extensively in mouse mode
ls. It was found to be effective in reducing tumor burden and to be mi
nimally toxic. Twelve phase I clinical trial patients with recurrent c
ir persistent epithelial ovarian cancer were treated with one to three
cycles of intraperitoneal vector. There was minimal toxicity four pat
ients developed fevers (<102.5 degrees F) and three had sterile perito
nitis, which resolved within 48 hours. The vector was found to be fair
ly stable in some patients at 24 hours: as well as transferred into, a
nd expressed in patient tissues. Stable disease was noticed in 8 of th
e 12 patients, suggesting that the peritoneal cavity may be an appropr
iate site for gene therapy.