Developing new treatments for a type of leukemia linked to MLL gene rearrangements

Discovery of First-in-class WDR5 PROTACs as a Novel Therapeutic Strategy for MLL-rearranged Leukemias

NIH-funded research Icahn School of Medicine at Mount Sinai · NIH-11066502

This study is looking at a type of leukemia that affects babies and is testing a new treatment that targets a specific protein to help fight the cancer more effectively, especially for those who haven't had much success with current therapies.

Quick facts

Grant typeR01 grant
Study typeNIH-funded research
Funding institutionIcahn School of Medicine at Mount Sinai NIH-funded
Lab location1 site (New York, United States)
Project IDNIH-11066502 on NIH RePORTER

What this research studies

This research focuses on a specific type of leukemia caused by rearrangements of the MLL gene, which is particularly prevalent in infants. The study aims to develop a novel therapeutic strategy using WDR5 PROTACs, which are designed to degrade the WDR5 protein that plays a crucial role in the growth of these cancer cells. By targeting WDR5, the researchers hope to create a more effective treatment option for patients who currently have limited responses to existing therapies. The approach involves pharmacological degradation rather than traditional inhibition, which may lead to better outcomes for patients with MLL-rearranged leukemias.

Who could benefit from this research

Good fit: Ideal candidates for this research are infants and children diagnosed with acute lymphoblastic leukemia or acute myeloid leukemia characterized by MLL gene rearrangements.

Not a fit: Patients with other types of leukemia or those who do not have MLL gene rearrangements may not benefit from this research.

Why it matters

Potential benefit: If successful, this research could provide a new, effective treatment option for patients suffering from MLL-rearranged leukemias.

How similar studies have performed: While the approach of targeting WDR5 is novel, previous research has shown promise in targeting similar pathways in cancer treatment.

Where this research is happening

New York, United States

Researchers

About this research

  1. This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
  2. Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
  3. For full project details, budget, and progress reports, visit the official NIH RePORTER page below.
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.