Boosting TET enzymes with vitamin C for acute myeloid leukemia
Official title: Enhancing TET activity for the treatment of hematological malignancy
Funded at University of Miami School of Medicine · NIH-11161573 · Data updated 2026-11-12
This project explores whether giving vitamin C can boost a natural enzyme and help adults with TET2‑mutant acute myeloid leukemia.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Miami School of Medicine NIH-funded |
| Lab location | 1 site (Coral Gables, United States) |
| Project start | August 1, 2023 |
| Project end | July 31, 2028 |
| Funding year | 2025 |
| Award (this year) | $351,131 |
| NIH project number | 5R01CA282453-03 |
| Project ID | NIH-11161573 on NIH RePORTER |
Who could benefit from this research
✓Could help
Adults (21+) with acute myeloid leukemia that carries a TET2 mutation, particularly those who cannot tolerate intensive chemotherapy, are the ideal candidates.
✕Less likely to help
Patients without TET2 mutations, children, or people whose leukemia is driven by different molecular changes are unlikely to benefit from this approach.
What this research studies
This project explores giving vitamin C (ascorbate) to increase activity of TET enzymes that help control DNA in blood stem cells. Many adults with AML carry one mutated TET2 gene, so researchers hope boosting the remaining TET2 activity can reverse harmful gene silencing. Preclinical work in cells and animal models shows vitamin C raises DNA hydroxymethylation, encourages leukemia cell differentiation, and slows disease progression. The team is carrying out laboratory and translational experiments aimed at developing vitamin C as a low‑toxicity adjuvant for TET2‑mutant AML.
Why it matters
Potential benefit: If successful, this could become a low‑toxicity treatment option that slows leukemia growth and improves outcomes for older adults with TET2‑mutant AML.
How similar studies have gone: Preclinical studies in cells and animals have shown promising effects of vitamin C on TET activity and leukemia biology, but clinical evidence in patients is still limited.
Where this research is happening
-
University of Miami School of Medicine
Coral Gables, United States Active
Researchers
- Principal investigator: Cimmino, Luisa — University of Miami School of Medicine
- Study coordinator: Cimmino, Luisa
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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.