Stanton Gerson, M.D., professor of medicine, has been leading the effort to introduce a gene into bone marrow cells that would protect the cells against the debilitating effects of chemo, thereby helping the patients maintain greater strength following chemotherapy.
June 6, at the American Society of Gene Therapy meeting in Minneapolis, Gerson and colleagues will present preliminary results of a Phase I clinical trial to test the safety of the method in humans. The study found no complications in five patients who were tested thus far, and found up to 41 percent transfer of the protective gene to the bone marrow, or blood stem cells.
Gerson, who also directs the Center for Stem Cell and Regenerative Medicine, said, "The results are encouraging and will help move this novel approach into new therapies."
Gerson's group, which includes Jane Reese and Omer Koc, M.D., has studied the gene mutant MGMT, that is able to protect stem cells from chemotherapy. In animal studies, they have found that this gene can provide stem cells with very high levels of survival advantage [more than 500 fold] compared to normal stem cells not carrying the gene.
Based on those preclinical animal results, they have begun this clinical trial in patients with advanced cancer. Blood stem cells are collected from patients and exposed to a retrovirus containing the gene, which inserts the gene into the cells. Patients are then infused with their own genetically-modified cells.
Patients are then treated with combination chemotherapy. Because stem cells have
Contact: George Stamatis
Case Western Reserve University