Targeting a specific protein to treat aggressive leukemia in children
Targeting MBNL1-mediated alternative splicing in MLL-fusion leukemia
['FUNDING_CAREER'] · CINCINNATI CHILDRENS HOSP MED CTR · NIH-11032743
This study is looking at a protein called MBNL1 that helps certain aggressive leukemias in babies and young kids survive, and it aims to find a way to block this protein to help stop the cancer from growing.
Quick facts
| Phase | ['FUNDING_CAREER'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | CINCINNATI CHILDRENS HOSP MED CTR (nih funded) |
| Locations | 1 site (CINCINNATI, UNITED STATES) |
| Trial ID | NIH-11032743 on ClinicalTrials.gov |
What this research studies
This research investigates the role of a protein called MBNL1 in a type of aggressive leukemia that affects infants and young children. The researchers have found that this leukemia often involves a genetic rearrangement and that MBNL1 is crucial for the cancer cells' survival. By inhibiting MBNL1, the study aims to disrupt the cancer cell growth and promote cell death. The approach includes understanding the mechanisms of how MBNL1 supports leukemia and developing a small-molecule inhibitor as a potential treatment.
Who could benefit from this research
Good fit: Ideal candidates for this research are infants and children diagnosed with MLL-fusion leukemia or related acute myeloid leukemias.
Not a fit: Patients with leukemia types that do not involve MLL gene rearrangements may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to a new targeted therapy for treating aggressive leukemia in young patients.
How similar studies have performed: While targeting alternative splicing in cancer is a novel approach, preliminary studies have shown promising results in similar contexts.
Where this research is happening
CINCINNATI, UNITED STATES
- CINCINNATI CHILDRENS HOSP MED CTR — CINCINNATI, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: LEE, LYNN HUA — CINCINNATI CHILDRENS HOSP MED CTR
- Study coordinator: LEE, LYNN HUA
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.
Conditions: anti-cancer therapy