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Researcher honored for studies of neural stem cells, gene therapy

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 Researcher honored for studies of neural stem cells, gene therapy 

  


By Shawn Le


The American Society of Gene Therapy (ASGT) recently honored Karen Aboody, M.D., for her pioneering work with neural stem cells and cancer and significant contributions to the field of gene therapy.

Aboody, assistant professor in the departments of Hematology & Hematopoietic Cell Transplantation and Neurosciences, received the 2008 ASGT Outstanding New Investigator Award, a highly competitive honor that recognizes active members who conduct original research in basic sciences, technology development or clinical translation in the field of gene therapy.

Karen Aboody (Photo by Marcelo Coelho)
“This great recognition is highly deserved for all you have accomplished in the field,” wrote Donald Kohn, M.D., chair of the ASGT advisory council, in his congratulations.

Said Aboody: “I am honored to have the ASGT recognize the neural stem cell-mediated cancer gene therapy approach we are developing at City of Hope.

“This novel tumor-selective treatment has the potential to overcome current obstacles limiting the success of surgical, radiation, chemotherapy and gene therapy for malignant brain tumors and other invasive cancers. Using neural stem cells as delivery vehicles for therapy may allow us to localize concentrated treatment specifically to tumor sites, while decreasing toxicity to normal tissues.”

Aboody studies how neural stem cells naturally gravitate to tumors, a property that can be exploited for targeting gene therapy directly to cancer cells.

She is developing a therapy that uses genetically modified neural stem cells to deliver therapeutic genes to cancer sites. These activating genes can unleash cancer-fighting compounds when they interact with a prodrug — an inactive drug that metabolizes only in the presence of an activating agent. The potential therapy would concentrate the compounds on the tumor cells, minimizing damage to surrounding healthy tissue.

“In our preclinical studies, we have demonstrated significant therapeutic efficacy in models of glioma, melanoma brain metastases, medulloblastoma and disseminated neuroblastoma,” Aboody said. “We have designed a phase I human clinical trial that is currently being developed for regulatory review. Following approval from the NIH [National Institutes of Health] Recombinant Advisory Committee for our cells and protocol, Drs. Jana Portnow, Behnam Badie and I hope to bring this therapy to patients with recurrent glioma in the near future.

“At the same time, we are exploring various stem-cell-mediated therapeutic applications for other fatal cancers.”

Aboody received the ASGT award during the society’s annual meeting in late May in Boston. She received a $1,000 honorarium and delivered an overview of the neural stem cell work during a plenary lecture attended by more than 1,500 colleagues.

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