Alex's Lemonade Stand Foundation for Childhood Cancer

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.

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

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.

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.