Dr. Adam Durbin, Young Investigator: Neuroblastoma
Project title: Novel agents targeting EP300-regulated transcription in neuroblastoma
Project Funded: 2019-2021
Neuroblastoma is a pediatric tumor of the peripheral sympathetic nervous system, the nerves that comprise the nervous system outside of the brain and spinal cord. Neuroblastoma impacts nearly 700 children in the US each year. Current therapies for high-risk neuroblastoma contribute to a survival rate of only 50%. Unfortunately, even in those children that survive, treatment-induced long-term toxicities include deafness, cardiac insufficiency, infertility, and increased risk of second cancers.
Adam Durbin, MD, Ph.D. is a CureSearch Young Investigator conducting research at the St. Jude Children’s Research Hospital aiming to develop a new targeted therapy strategy for neuroblastoma. Dr. Durbin identified a protein (EP300) that is necessary for neuroblastoma growth. Upon identification of EP300 as a druggable target, Dr. Durbin worked to develop a novel, bioavailable compound that is able to selectively destroy EP300. During the course of his CureSearch project, Dr. Durbin will investigate this new compound’s ability to stop neuroblastoma growth. If successful, this project will not only offer an innovative treatment strategy for high-risk neuroblastoma, but has the potential to expand into other EP300-dependent pediatric tumors, including myeloid leukemia and rhabdomyosarcoma.
“As both a scientist and pediatric oncologist, I am focused on developing new drugs for children with challenging-to-treat cancers,” said Dr. Durbin. “Funding from CureSearch for Children’s Cancer is critical to the early steps of our work to try to deal with these issues by developing less toxic, more effective therapies for children with cancer.”
Project Update: As of May 2020, Dr.Durbin has produced data to demonstrate that his target, EP300, is essential for the maintenance of neuroblastoma cell survival. He has also generated preliminary data indicating that his drug, which destroys the target EP300 by directing it toward degradation, reduces the growth of tumor cells in neuroblastoma xenograft models; these data strongly support EP300’s role as a therapeutic target in neuroblastoma and subsequent experiments will reveal the dosing that yields optimal tumor growth inhibition while limiting side-effects
This project was supported by a grant from Genentech.