保护基因 MGMT：让干细胞勇往直前

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爱尔兰癌症中心的研究人员干细胞基因治疗进展
耐药基因MGMT
Stem Cell Gene Therapy Advanced By Ireland Cancer Center Researchers

Ireland Cancer Center of University Hospitals Case Medical 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, with the ultimate goal of being able to use stronger chemotherapy treatment with less severe side effects. Under this protocol, MGMT, a drug-resistance gene, is added into purified hematopoietic stem cells to protect these cells from the damage of chemotherapy regimens.

In one of 24 presentations 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.

In addition to this promising research, Ireland Cancer Center scientists presented 24 oral and poster presentations at ASH. \"The breadth and depth of this innovative hematologic research at the Ireland Cancer Center are outstanding,\"says Alvin Schmaier, MD, Chief of Hematology/Oncology at UHCMC and Case Western Reserve University School of Medicine. ?Our faculty is making tremendous advances in these fields which is reflected in their being chosen for oral and poster presentations.\"