Stem Cell Gene Therapy Advanced in Ireland

With the ultimate goal of being able to use stronger chemotherapy treatment with less severe side effects, Ireland Cancer Center researchers have recently made great strides in stem cell gene therapy research by transferring a new gene to cancer patients, via their own stem cells. In order to protect cells from the damage of chemotherapy regimens, a drug-resistance gene called MGMT isadded into purified hematopoietic stem cells.

Eight patients were enrolled in a trial where theywere infused with their own stem cells which were engineered to carry the MGMT gene. Two of the eight patients received a placebo. This presentation, which was made byStanton Gerson, MD, and colleagues from Ireland Cancer Center atthe annual American Society of Hematology meeting. It was one of 24 presentations that were made. Gerson and his team revealed significant new findings with this gene and drug combination. The gene was identified in three of the patient’s bone marrow or blood. Up to 28 weeks following administration, one of the patients still carried the gene.

Dr. Gerson, who is the Director of the Ireland Cancer Center and Case Comprehensive Cancer Center, said that “this study is the first to show the success of treatment with evidence that stem cells now carry the new gene.”

Gerson, along with his team of researchers, led the Phase I study.

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