New targeted treatment approaches for acute leukemias
Development of Novel Therapeutic Strategies in Human Leukemias
Testing new drugs that push leukemia cells to die and that target hard-to-drug genetic drivers for children and adults with acute leukemias.
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-11196538 on NIH RePORTER |
What this research studies
Researchers at Dana-Farber are developing medicines that change how leukemia cells regulate cell death using BH3 profiling to find compounds that lower the apoptotic threshold. They are creating targeted protein degradation strategies to eliminate proteins previously considered undruggable and are discovering agents that hit epigenetic and genetic dependencies in leukemia, including KMT2A-, NPM1-, and NUP98-rearranged tumors. Work uses laboratory models, molecular profiling, and early-phase clinical trials to move promising candidates from bench to bedside. The program builds on prior successes that brought FLT3 and BCL-2 inhibitors to patients and advances new compounds such as DOT1L and Menin inhibitors into trials.
Who could benefit from this research
Good fit: People with acute myeloid or related acute leukemias, including children, adolescents, and adults, especially those with KMT2A-, NPM1-, or NUP98-rearrangements or relapsed/refractory disease, would be the most likely candidates.
Not a fit: People without acute leukemia or with leukemia subtypes not targeted by this program (for example some chronic leukemias) are unlikely to benefit directly from these projects.
Why it matters
Potential benefit: If successful, this program could produce more effective, more specific therapies for acute leukemias and offer new options for patients who relapse or do not respond to current treatments.
How similar studies have performed: Related approaches have already led to approved drugs (FLT3 and BCL-2 inhibitors) and early clinical trials for DOT1L and Menin inhibitors, so this program builds on prior translational successes.
Where this research is happening
Boston, United States
- Dana-Farber Cancer Inst — Boston, United States (Active)
Researchers
- Principal investigator: Ebert, Benjamin Levine — Dana-Farber Cancer Inst
- Study coordinator: Ebert, Benjamin Levine
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.