Drugs that remove WDR5 to treat MLL‑rearranged acute leukemias
Discovery of First-in-class WDR5 PROTACs as a Novel Therapeutic Strategy for MLL-rearranged Leukemias
Developing small molecules that tag and destroy the WDR5 protein to help people with MLL‑rearranged acute leukemias.
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-11323452 on NIH RePORTER |
What this research studies
Researchers are creating first‑in‑class PROTAC drugs that bind WDR5 and mark it for destruction because WDR5 helps drive MLL‑rearranged leukemias. They will optimize these molecules in leukemia cells grown in the lab and measure cancer cell death, then test lead candidates for safety and effectiveness in animal models. The team will compare the degraders to existing WDR5 inhibitors and study how removing WDR5 affects cancer growth pathways. If promising, the best candidates would be prepared for future early human testing.
Who could benefit from this research
Good fit: People whose leukemia carries an MLL (MLL‑rearranged) genetic change — including infants and children with MLL‑rearranged ALL or AML and adults with relapsed or refractory disease — would be the intended candidates.
Not a fit: Patients whose leukemia does not have an MLL rearrangement or whose disease is driven by other genetic changes are unlikely to benefit from WDR5‑targeting degraders.
Why it matters
Potential benefit: If successful, this approach could produce a targeted therapy that kills MLL‑rearranged leukemia cells and offer new options for patients with these aggressive cancers.
How similar studies have performed: PROTAC degraders have shown promise for other protein targets in laboratory and early clinical work, but targeting WDR5 is a new approach that has not yet been proven in patients.
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.