Integration of epigenetic and non-coding RNA mechanisms in leukemia

NIH RePORTER · NIH · R37 · $356,850 · view on reporter.nih.gov ↗

Abstract

ABSTRACT Acute myeloid leukemia (AML) is the most common (30-40%) of all leukemias and has the poorest survival (25%) of any leukemia. Mutations in the DNA methyltransferase DNMT3A and internal tandem duplications of the FLT3 receptor tyrosine kinase (FLT3-ITD) and are two of the most frequent events in over 50% of AML and commonly co-occur in patients conferring increased resistance to chemotherapy, the standard treatment for this subtype of AML. DNMT3A/FLT3-mutant AML have more adverse clinical outcome than AML with either mutation alone. Thus, there is a dire need for a better understanding of the biological mechanisms underlying this disease to address pressing therapeutic challenges. Our preliminary data suggest that DNMT3A/FLT3-ITD AML downregulate innate immune signaling through Toll-like receptors (TLRs) to maintain stemness and block differentiation. Our data also indicate that microRNA may play an important role in the dysregulation of TLR pathways in AML cells, suggesting a novel crosstalk between epigenetic/signaling mutations, microRNA, and innate immune signaling in AML. Despite evidence that suggests TLR signaling is an important contributor to the pathogenesis of myelodysplastic syndrome (MDS), very little is known about TLR signaling in AML. Thus, we are focusing on defining the mechanisms that deregulate TLR signaling in AML, understanding the consequences of suppressed TLR signaling in AML pathogenesis, and finally whether TLR signaling can be leveraged to treat AML.

Key facts

NIH application ID
9968169
Project number
5R37CA226433-03
Recipient
THOMAS JEFFERSON UNIVERSITY
Principal Investigator
Sara E Meyer
Activity code
R37
Funding institute
NIH
Fiscal year
2020
Award amount
$356,850
Award type
5
Project period
2018-07-01 → 2023-06-30