Platform development for target identification in high risk pediatric leukemias
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. Premise for this research is the recent development of high throughput forward genetic tools capable of analyzing genetic dependencies genome-wide and large scale drug libraries of bioactive compounds. The Crazy 8 discussion panel identified (i) the development of cell line panels representative of high risk leukemia groups and (ii) the establishment of effective protocols and tools for cell line and primary sample screening as important objectives aiming at identifying candidate drugs and drug targets of high translational value.
Project Update 2020:
This project has addressed the generation of new therapeutic platform for the identification of new drugs and drug targets for the treatment of high-risk pediatric leukemia patients. Our new cells culture systems allow the expansion of leukemia cells in laboratory conditions that recapitulate many features important for leukemia growth. These new discovery and therapeutic platforms offer new opportunities for advancing the discovery of drugs and drug combinations active in the treatment of high-risk patients.