Targeting genetic weak spots in childhood cancers
Targeting Pediatric Cancer Vulnerabilities
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 ID | NIH-11160558 on NIH RePORTER |
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.
Who could benefit from this research
Good fit: 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.
Not a fit: 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.
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 performed: 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
Boston, United States
- Dana-Farber Cancer Inst — Boston, United States (Active)
Researchers
- Principal investigator: Stegmaier, Kimberly — Dana-Farber Cancer Inst
- Study coordinator: Stegmaier, Kimberly
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.