Childhood Cancer Research
Columbia University
New York, NY 10032
United States
Despite much progress in understanding the genetic basis and mechanism of leukemogenesis of high risk leukemia there are a limited number of targeted therapies available for the treatment of high risk patients. The goal of this project is to leverage new screening technologies for the identification of candidate drugs and druggable gene targets for the treatment of high risk leukemia patients.
Mentor: Robert Wechsler-Reya
Transcription factors are proteins that help to regulate genes. Our goal is to develop an entirely new treatment for neuroblastoma that is directed towards novel transcription factor targets and that uses an innovative strategy to do so. Our studies show that the transcription factors ATF5, CEBPB and CEBPD are required by neuroblastoma cells for their survival. Conventional thought is that targeting such factors clinically is not feasible.
Background
Early T cell precursor leukemia (ETP ALL) is an aggressive hematologic malignancy that requires treatment with intensified chemotherapy. Thus, further advances in the treatment of this disease require the development of effective and highly specific molecularly targeted anti-leukemic drugs. There is growing awareness that targeting the metabolic differences between tumor and normal cells holds promise as a novel anticancer strategy.
Background
Following on the completion of the Human Genome Project and using innovative sequencing tools researchers are rapidly annotating the complete repertoire of genetic alterations in cancer. However most of these lesions are located in genomic "dark matter" outside of well-annotated genes.
Background
Advanced stage neuroblastoma is frequently refractory to multimodality therapy. Immunotherapy targeting the PD-1/PD-L1 interaction has been shown to augment the host immune response following radiation therapy in mouse models of melanoma and breast carcinoma, improving local control. Neuroblastoma tumor cells also express PD-L1.
Survival following chemotherapy for pediatric malignancy remains one of the most stunning successes of modern medicine. Nevertheless, there remains a subset of patients with aggressive disease for whom novel therapies are required.
The Children's Hospital of New York-Presbyterian at Columbia University has one of the largest pediatric oncology programs in the United States. Residing within this division is the Pediatric Cancer Foundation Developmental Therapeutics Program (PCFDTP) led by Dr. Julia Glade Bender. The PCFDTP has demonstrated scientific leadership in the field of translational antiangiogenic research through investigations of novel agents which inhibit tumor blood vessel growth by blocking vascular endothelial growth factor.
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