Ireland Cancer Center researchers advance stem cell gene therapy

December 12, 2007

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 ¡V 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.¡¨

The presentations include:
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About University Hospitals

With 150 locations throughout Northeast Ohio, University Hospitals serves the needs of patients through an integrated network of hospitals, outpatient centers and primary care physicians. At the core of our Health System is University Hospitals Case Medical Center. The primary affiliate of Case Western Reserve University School of Medicine, University Hospitals Case Medical Center is home to some of the most prestigious clinical and research centers of excellence in the nation and the world, including cancer, pediatrics, women's health, orthopedics and spine, radiology and radiation oncology, neurosurgery and neuroscience, cardiology and cardiovascular surgery, organ transplantation and human genetics. Its main campus includes the internationally celebrated Rainbow Babies & Children's Hospital, ranked best in the Midwest and first in the nation for the care of critically ill newborns; MacDonald Women's Hospital, Ohio's only hospital for women; and Ireland Cancer Center, which holds the nation's highest designation by the National Cancer Institute of Comprehensive Cancer Center. For more information, go to www.uhhospitals.org.

University Hospitals of Cleveland

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