OVARIAN-CANCER GENE-THERAPY

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.