How FLT3 changes drive acute myeloid leukemia cell behavior
Regulation of FLT3 Signaling in Leukemia
['FUNDING_R01'] · CHILDREN'S HOSP OF PHILADELPHIA · NIH-11170526
Researchers are studying how a common FLT3 mutation changes where the protein sits inside blood stem cells and how that alters signals that can cause acute myeloid leukemia.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | CHILDREN'S HOSP OF PHILADELPHIA (nih funded) |
| Locations | 1 site (PHILADELPHIA, UNITED STATES) |
| Trial ID | NIH-11170526 on ClinicalTrials.gov |
What this research studies
This research looks at a common FLT3 mutation (FLT3-ITD) that is linked to worse outcomes in AML and explores how chemical tagging called palmitoylation controls where the FLT3 protein goes inside cells. The team found that an enzyme named ZDHHC6 adds this tag and that changing palmitoylation moves mutant FLT3 from the cell’s internal compartments to the surface, altering the signaling programs the cancer cells use. Scientists will use patient-derived blood stem/progenitor cells, cell lines, and molecular experiments to map these changes in location and downstream signaling through pathways like STAT5, AKT, and ERK. The goal is to reveal mechanisms that could point to new ways to block the abnormal signaling driving leukemia growth.
Who could benefit from this research
Good fit: People with acute myeloid leukemia whose cancer carries an FLT3-ITD mutation would be the most relevant candidates for future trials or for donating samples to support this research.
Not a fit: Patients without FLT3 mutations or those seeking immediate therapeutic benefit should not expect direct clinical benefit from this laboratory-focused research right now.
Why it matters
Potential benefit: If successful, this work could identify new targets or strategies to stop FLT3-driven leukemia and improve treatments for people with FLT3-mutant AML.
How similar studies have performed: Drugs that block FLT3 signaling have benefited some patients with FLT3-mutant AML, but targeting palmitoylation and protein localization is a newer and less-tested approach.
Where this research is happening
PHILADELPHIA, UNITED STATES
- CHILDREN'S HOSP OF PHILADELPHIA — PHILADELPHIA, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: TONG, WEI — CHILDREN'S HOSP OF PHILADELPHIA
- Study coordinator: TONG, WEI
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page below.