Why some blood disorders turn into acute leukemia
Identifying the mechanisms of leukemia progression
['FUNDING_OTHER'] · ST. JUDE CHILDREN'S RESEARCH HOSPITAL · NIH-11172458
Researchers are using patient samples, lab models, and CRISPR gene screens to find the genetic changes that make pediatric and adult blood disorders progress to acute myeloid leukemia so treatments can be improved.
Quick facts
| Phase | ['FUNDING_OTHER'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | ST. JUDE CHILDREN'S RESEARCH HOSPITAL (nih funded) |
| Locations | 1 site (MEMPHIS, UNITED STATES) |
| Trial ID | NIH-11172458 on ClinicalTrials.gov |
What this research studies
This project will compare how childhood and adult blood disorders evolve into acute leukemia by studying patient blood and bone marrow samples alongside animal and lab models. The team will use large-scale CRISPR/Cas9 screens to pinpoint genes and pathways that drive malignant transformation. They will study the effects of tumor suppressor gene disruption and test which approved or investigational drugs might block progression. Findings are intended to guide new prevention strategies and treatments for people at risk of developing acute leukemia.
Who could benefit from this research
Good fit: Ideal candidates would be adults or children with clonal blood disorders, myeloproliferative neoplasms, or acute myeloid leukemia who can provide blood or bone marrow samples for research.
Not a fit: People without blood cancers or related clonal disorders, or those unable to provide samples or attend study visits, are unlikely to benefit directly from this project.
Why it matters
Potential benefit: If successful, the work could lead to new ways to prevent or treat progression to acute leukemia and more targeted therapies for patients.
How similar studies have performed: Prior research—including the PI's discovery of GATA1 mutations in Down syndrome–associated leukemia—has successfully identified genetic drivers of leukemia and CRISPR and animal models have been useful, though turning those findings into proven therapies remains an ongoing challenge.
Where this research is happening
MEMPHIS, UNITED STATES
- ST. JUDE CHILDREN'S RESEARCH HOSPITAL — MEMPHIS, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: CRISPINO, JOHN D — ST. JUDE CHILDREN'S RESEARCH HOSPITAL
- Study coordinator: CRISPINO, JOHN D
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: Blood Diseases