Targeting Epigenomics in Myeloid Neoplasms

点击翻译按钮获取中文摘要

DESCRIPTION (provided by applicant): Drug development in the era of biologically rational, "targeted therapeutics" in oncology requires specific expertise and complex skill sets. Far from previous paradigms in which drugs were plugged in to standard dose escalation schemata with dose limiting toxicity as the primary endpoint, today's investigators must develop creative trial designs which are appropriate for the specific agent and its specific targeted use. The investigator must be able to speak to the laboratory scientist working out the basic biological concepts, the pharmaceutical company which provides the drug, the analytic pharmacologists, biostatisticians, internal review boards and government regulators. Most importantly, the drug developer must be able to organize and lead a team effort, marshalling the resources at hand to design and execute a trial which is feasible, cost-effective, biologically and clinically sound, and which helps determine clinical and biological efficacy. The investigator must be able to glean information from the trial with which he or she can go back to the laboratory investigator and inform the basic investigation, forming an iterative process of discovery from bench to bedside to bench. Having acquired considerable unique expertise in the development of biologically driven therapies in myeloid malignancies, I am eager to spend more time mentoring developing investigators, transmitting my experience to a new generation. In the mentoring relationship, both mentor and trainee learn from each other and grow together. The Sidney Kimmel Comprehensive Cancer Center is a unique institution which provides outstanding resources for drug development in an academic center. I have organized a dynamic program focused on the translation of cutting edge basic science concerning the epigenetic regulation of gene expression to the rational non-cytotoxic treatment of myeloid malignancies. This program provides an outstanding venue for the training of young investigators; this K24 award will provide me with protected time to devote to that important endeavor and to further the accomplishment of my mission to develop effective therapies for this difficult group of patients.