Turning genetic discoveries into better care for high-risk acute leukemia
Official title: Translating genomic discoveries to improved outcomes for high risk acute leukemia
Funded at St. Jude Children's Research Hospital · NIH-11178425 · Data updated 2026-11-12
Researchers are using genetic information to find new tests and targeted treatments for children and young people with high‑risk acute leukemia.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | St. Jude Children's Research Hospital NIH-funded |
| Lab location | 1 site (Memphis, United States) |
| Project start | January 19, 2017 |
| Project end | August 31, 2030 |
| Funding year | 2025 |
| Award (this year) | $902,351 |
| NIH project number | 5R35CA197695-10 |
| Project ID | NIH-11178425 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are children and young adults with newly diagnosed or relapsed high‑risk acute leukemia who could provide samples or join future targeted therapy trials.
✕Less likely to help
Patients with low‑risk leukemia subtypes or unrelated medical conditions are unlikely to receive direct benefit from this specific program.
What this research studies
You would be part of research that looks at the DNA changes that cause aggressive acute leukemia and make it hard to treat. Scientists compare patient samples with laboratory models to understand how specific gene changes (like enhancer errors, BCL11B problems, LMO2 activation, and STAG2 loss) and abnormal protein behavior drive the disease. They are testing new approaches, including drugs that mark disease-causing proteins for destruction, to target drivers that current treatments cannot. The goal is to turn these findings into better diagnostics and medicines that raise cure rates for young patients with high‑risk leukemia.
Why it matters
Potential benefit: If successful, this work could produce new diagnostic tests and targeted therapies that improve cure rates for patients with high‑risk acute leukemia.
How similar studies have gone: Prior studies have identified leukemia subtypes and shown early promise for targeted protein degradation, but many approaches remain experimental and not yet proven in patients.
Where this research is happening
-
St. Jude Children's Research Hospital
Memphis, United States Active
Researchers
- Principal investigator: Mullighan, Charles G — St. Jude Children's Research Hospital
- Study coordinator: Mullighan, Charles G
Related research
Other NIH-funded projects on the same topics.
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This study is looking at how certain proteins linked to leukemia affect kids and teens with high-risk blood cancers, and the researchers hope to find new ways to treat these conditions by learning more about how the disease works.
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Reducing leukemia disparities in Latino children and teens
A large, multi-project effort aims to understand why Latino children with acute lymphoblastic leukemia have worse outcomes and how to reduce those gaps.
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Genomic advances to improve outcomes in high-risk acute leukemia
A physician-scientist team is studying the genetic drivers of pediatric acute leukemia to find new ways to diagnose and treat it, with the goal of improving cure rates for kids.
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Understanding and Targeting Extramedullary Acute Leukemia
A clinician-research team is studying myeloid sarcoma, a form of AML that forms solid tumors outside the bone marrow, to develop new treatments for patients.
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Diversifying acute leukemia trial enrollment through multilevel approach
Aiming to broaden participation in acute leukemia trials by testing a multi-layer plan that addresses barriers for adults with AML.
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Blocking menin to treat acute leukemias with high HOX gene activity
This work develops new drugs that block a protein called menin to help people with certain aggressive acute leukemias driven by HOX gene activation.
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.