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JQ1 as an inhibitor of the MLL-PTD/BRD4 complex function in AML

Research Scholar

Felice Pepe, PhD internship (Italy)
Ramiro Garzon, faculty mentor


  • Hometown: Salerno, Italy
  • Degrees received: PhD in oncology, and molecular and clinical pathology‚ D'Annunzio University of Chieti-Pescara, Italy.

What is the issue or problem addresses in your research?

Acute myeloid leukemia (AML) is a cancer that rises from myeloid lineage cells that received mutations in specific genes. Different mutations have different effects on cells and can result in aggressive cancers. We observed that product of an AML commonly mutated gene (known as MLL-PTD mutation) is found in patients that respond poorly to chemotherapy. To develop new treatments, that are urgently needed to cure patients with this disease, we first need to understand the biology of these cells.

What methodology did you use in your research?

Using ChIP-seq we identified aberrant BRD4 protein binding in MLL-PTD leukemic cells. We studied whether treating MLL-PTD AML samples with JQ1, a BRD4-activity inhibitor, would restore BRD4 binding, leading to leukemic cells killing. We found that treating AML MLL-PTD transplanted mice with JQ1, resulted prolonged survival and altered BRD4 binding.

What are the purpose/rationale and implications of your research?

MLL-PTD\BRD4 complex could have a key role in the development/progression of AML. Our data showed that targeting BRD4 could be a valid option to treat this subset of patients. Finally, blocking BRD4 could be a strategy to study the genes deregulated by MLL-PTD and how this mutation lead to AML.