NIH R01 · 2025
Ph1/2 Study of the Imipridone ONC201 for Treatment of AML IND125,203 (12/23/2014)
Project Summary/Abstract Survival rates (app. 30 %) of acute myeloid leukemia (AML) have not been improved over 4 decades, except in some specialized instances. The long term aim of this study is to increase the cure rates of AML through clinical implementation of targeting a new cellular survival mechanism, i.e. mitochondrial (mt) unfolded protein response (mtUPR). We are proposing to conduct a clinical Phase 1/2 trial of ONC201, a first-in-class imipridone and to confirm and further investigate the underlying novel mechanism of action (MOA). We discovered in extensive preclinical studies that ONC201 induces apoptosis in AML but not in normal cells. Importantly, ONC201 has great efficacy…
From the public funding record at NIH RePORTER. Describes the funded project, not the reviews below.