Developing New Treatments for MLL-rearranged Leukemias
Development of Novel PROTACs Targeting the ENL YEATS Domain for Treating MLL-rearranged Leukemias
This project is creating new medicines called PROTACs to help children and infants with a very aggressive type of leukemia called MLL-rearranged leukemia.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Icahn School of Medicine at Mount Sinai NIH-funded |
| Lab location | 1 site (New York, United States) |
| Project ID | NIH-11057696 on NIH RePORTER |
What this research studies
We know that MLL-rearranged leukemias are very serious, especially in infants, and current treatments are not very effective. This research focuses on a specific part of the leukemia cells, called the ENL YEATS domain, which is crucial for the cancer cells to grow and survive. Our goal is to design special molecules, called PROTACs, that can specifically target and eliminate this ENL YEATS domain. By removing this key component, we hope to stop the leukemia cells from growing and ultimately cause them to die, offering a new way to fight this challenging disease.
Who could benefit from this research
Good fit: This research is focused on developing treatments for patients, particularly infants, diagnosed with MLL-rearranged acute lymphoblastic leukemia (ALL) or acute myeloid leukemia (AML).
Not a fit: Patients with other types of leukemia or cancers not involving MLL gene rearrangements would likely not benefit from this specific treatment approach.
Why it matters
Potential benefit: If successful, this work could lead to the first effective therapies for MLL-rearranged leukemias, which currently have a very poor prognosis.
How similar studies have performed: The concept of targeting specific proteins with PROTACs has shown promise in other cancer types, but this specific approach for MLL-rearranged leukemias is novel and currently untested in humans.
Where this research is happening
New York, United States
- Icahn School of Medicine at Mount Sinai — New York, United States (Active)
Researchers
- Principal investigator: Jin, Jian — Icahn School of Medicine at Mount Sinai
- Study coordinator: Jin, Jian
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.