Dr. Margarita Gutova – High Impact Research Grant
City of Hope
Dr. Margarita Gutova’s team at City of Hope Medical Center is partnering with Dr. Robert Wechsler-Reya at the Sanford-Burnham Medical Research Institute to test a novel neural stem cell therapy for the pediatric brain tumor medulloblastoma. Medulloblastomas are the most common malignant childhood brain tumors. Despite recent advances in treatment, current therapies are often severely damaging to brain development and skeletal growth. Therefore, new treatments are critically needed to improve the survival and quality of life of children with medulloblastoma. Major obstacles to successful treatment of pediatric brain tumors include the blood-brain-barrier, which prevents many anti-cancer agents from entering the central nervous system. Neural stem cells offer a novel way to overcome these obstacles because they can cross the blood-brain-barrier, migrate to and selectively target tumor cells throughout the brain. NSCs can be genetically modified to act as delivery vehicles for targeted anti-cancer therapy, thereby increasing the tumor-localized concentrations of a drug while minimizing toxicity to normal tissues.
The goal of this study is to determine the pre-clinical effectiveness of an NSC-mediated therapy against two subtypes of medulloblastoma. We will also investigate a novel, non-invasive route of NSC delivery to the central nervous system intranasal administration of therapeutic stem cells. This study has tremendous significance because NSC-based delivery of chemotherapy could improve survival and minimize the serious brain damage caused by current radiation and chemotherapy regimens, thus improving the quality of survivorship of children with brain tumors. This represents a key advance over traditional approaches, and provides a mechanism to eradicate invasive, residual tumor cells. Tests are currently underway in mice, with the goal of beginning human clinical trials within the next three to five years.
The field of stem cell research is promising, but still relatively new. There are many exciting directions to pursue in order to better understand stem cell function and development, with a wide array of potential clinical applications. City of Hope laboratories have expertise translating stem cell therapy to the clinic, and our infrastructure gives us an unparalleled opportunity to realize the promise of bench-to-bedside science on behalf of patients.