My laboratory has generated and analyzed large high-throughput gene and microRNA (miRNA) expression data sets from chronic myeloid leukemia (CML) and acute myeloid leukemia (AML) patient samples to identify genes, miRNAs, and pathways that contribute to leukemogenesis and therapy resistance. I have investigated how specific candidates 1) contribute to disease, 2) can be targeted by existing or new therapeutic strategies and 3) can be used to predict outcomes. Specific to this proposal, together with Pamela Becker MD, PhD, I have developed a high-throughput drug screening tool to investigate drug resistance and guide selection of therapy in hematologic malignancies. A clinical trial to select next-line therapy in real-time is under evaluation in refractory and resistant AML patients (NCT02551718).
In funded work I am examining mechanisms of CML disease progression and therapy resistance. Data from high throughput drug screening are being used in conjunction with transcriptome analysis and clonal mutational evolution. In addition to these translational research efforts, I am PI on a number of clinical trials in AML and CML and am familiar with the clinical profile of many of the drugs under evaluation. I can rapidly facilitate translation to investigator-initiated clinical trials. I will serve as co-PI of this application expanding my HTS panels of targeted cancer therapeutics to examine efficacy against SARS-Co-V-2 with the goal of rapid translation to clinical trials.