Why CSF3R‑mutant AML cells fail to mature into normal white blood cells

Mechanisms of differentiation blockade in CSF3R-mutant AML

NIH-funded research Oregon Health & Science University · NIH-11378903

Researchers are learning how CSF3R and related gene changes stop leukemia cells from maturing in people with CSF3R‑mutant acute myeloid leukemia.

Quick facts

Grant typeR01 grant
Study typeNIH-funded research
Funding institutionOregon Health & Science University NIH-funded
Lab location1 site (Portland, United States)
Project IDNIH-11378903 on NIH RePORTER

What this research studies

From a patient's point of view, the team is studying leukemia samples and lab models that carry CSF3R mutations together with changes in CEBPA or core binding factor (CBF) genes to see why the cancer cells stay immature. They will look at how these genetic changes alter signaling pathways (including STAT proteins) and the epigenetic control of myeloid cell development. The work combines analyses of human-derived samples with molecular and cellular experiments in the lab to map the mechanisms of differentiation arrest. Findings are intended to point toward targets that could be used to restore normal maturation of blood cells in this AML subtype.

Who could benefit from this research

Good fit: Ideal candidates are people diagnosed with acute myeloid leukemia whose leukemia carries CSF3R mutations, especially those with co‑occurring CEBPA mutations or CBF translocations.

Not a fit: Patients without CSF3R mutations (or with entirely different leukemia subtypes) are unlikely to benefit directly from this specific research.

Why it matters

Potential benefit: If successful, this work could point to new ways to make CSF3R‑mutant leukemia cells mature and potentially improve outcomes for patients with this subtype of AML.

How similar studies have performed: Differentiation therapy has been highly successful in a related AML subtype (APL), but targeting CSF3R‑driven AML is a newer, less‑tested approach.

Where this research is happening

Portland, United States

Researchers

About this research

  1. This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
  2. Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
  3. For full project details, budget, and progress reports, visit the official NIH RePORTER page below.
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.