Targeting genetic weak spots in childhood cancers
Official title: Targeting Pediatric Cancer Vulnerabilities
Funded at Dana-Farber Cancer Inst · NIH-11160558 · Data updated 2026-11-12
Using lab gene-editing and screening tools to find and block weaknesses in childhood cancers so researchers can develop safer, more precise treatments for kids with leukemias and other pediatric tumors.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Dana-Farber Cancer Inst NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project start | September 19, 2023 |
| Project end | August 31, 2030 |
| Funding year | 2025 |
| Award (this year) | $1,065,492 |
| NIH project number | 5R35CA283977-03 |
| Project ID | NIH-11160558 on NIH RePORTER |
Who could benefit from this research
✓Could help
Children with fusion-driven pediatric cancers, such as certain forms of childhood acute myeloid leukemia, would be the most likely beneficiaries and potential future trial candidates.
✕Less likely to help
People with unrelated adult cancers, cancers without the specific fusion drivers, or those needing immediate standard therapy may not see direct benefit from this preclinical research.
What this research studies
This project uses CRISPR gene-editing screens and laboratory models to find critical vulnerabilities in cancers that affect children, especially fusion-driven leukemias. The team will confirm which molecular targets truly drive the cancer and then search for early chemical compounds that can hit those targets. Work will include studies in cells, model systems, and patient-derived samples to prioritize the most promising leads. The aim is to move validated targets toward future drug development with fewer toxic side effects than traditional chemo and radiation.
Why it matters
Potential benefit: If successful, this work could lead to targeted drugs for pediatric leukemias that are more effective and cause fewer long-term harms than current treatments.
How similar studies have gone: CRISPR screens have revealed useful drug targets in some cancers, but applying these approaches specifically to fusion-driven pediatric leukemias is less developed and relatively novel.
Where this research is happening
-
Dana-Farber Cancer Inst
Boston, United States Active
Researchers
- Principal investigator: Stegmaier, Kimberly — Dana-Farber Cancer Inst
- Study coordinator: Stegmaier, Kimberly
Related research
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Targeting childhood cancer vulnerabilities with CRISPR-based screens
A pediatric cancer research project uses CRISPR screens to find new, druggable weaknesses in childhood leukemias to guide safer, targeted therapies.
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Using children's tumor protein and genetic data to find better treatments
This project analyzes tumor DNA, RNA, proteins, and protein modifications to find potential treatment targets for children with brain tumors and leukemias.
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.