Blocking BFL-1 to help treat acute myeloid leukemia
Targeting BFL-1 in Acute Myelogenous Leukemia
Researchers are developing drugs that block the BFL-1 protein to help people with acute myeloid leukemia who do not respond to current BCL-2 treatments.
Quick facts
| Grant type | P01 program project |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Dana-Farber Cancer Inst NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project ID | NIH-11196540 on NIH RePORTER |
What this research studies
This project focuses on creating drugs that target BFL-1, a protein that helps some AML cells avoid cell death. Investigators use BH3 profiling and a peptide called FS-1 to identify which patients' leukemia cells depend on BFL-1. Laboratory models and biochemical assays will guide the design and testing of small molecules that fit BFL-1's BH3-binding groove. Promising candidates could move toward clinical testing as a new option for patients with venetoclax-resistant AML.
Who could benefit from this research
Good fit: Adults with acute myeloid leukemia, particularly those with relapsed or refractory disease or who failed venetoclax, and whose cancer shows BFL-1 dependence by BH3 profiling are the most likely candidates.
Not a fit: Patients whose leukemia does not rely on BFL-1 are unlikely to benefit from a BFL-1-directed therapy.
Why it matters
Potential benefit: If successful, this could provide a new targeted treatment for AML patients who do not respond to existing BCL-2 inhibitors.
How similar studies have performed: BCL-2 inhibitors like venetoclax have improved outcomes for many AML patients, but direct targeting of BFL-1 is a newer approach that so far has mainly preclinical support.
Where this research is happening
Boston, United States
- Dana-Farber Cancer Inst — Boston, United States (Active)
Researchers
- Principal investigator: Letai, Anthony G — Dana-Farber Cancer Inst
- Study coordinator: Letai, Anthony G
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.