Advancing Stem Cell Gene Therapy

In a presentation by Ireland Cancer Center researchers at the annual American Society of Hematology meeting, Stanton Gerson, MD, and colleagues presented that eight patients were enrolled on the trial and six were infused with their own stem cells which were engineered to carry the MGMT gene. In three patients, stem cells carrying the gene were identified in their blood or bone marrow. In one patient, stem cells carrying the gene were detected up to 28 weeks after their administration. This significant finding has never been reported before with this gene and drug combination.

'This study is the first to show the success of treatment with evidence that stem cells now carry the new gene,'¨ says Dr. Gerson, Director of the Ireland Cancer Center and Case Comprehensive Cancer Center, who spearheaded the Phase I study along with a team of researchers. 'These patients show the success of treatment with evidence that their stem cells now carry the new genes. This is a breakthrough -- the first time selection with MGMT has been shown to occur in patients.'¨

Preclinical animal research, conducted by Dr. Gerson and his colleagues, has shown that the gene G156A-MGMT can provide stem cells with very high levels of drug resistance, compared to normal stem cells not carrying the gene. In the Phase I trial for patients with advanced malignancies, researchers collected peripheral blood stem cells from patients and exposed them to a retrovirus containing the G156A-MGMT gene.