NIH-funded research R01 grant

Boosting TET Activity to Help Treat AML with Vitamin C

Official title: Enhancing TET activity for the treatment of hematological malignancy

Funded at University of Miami School of Medicine · NIH-11398136 · Data updated 2026-11-12

Vitamin C may help people with a type of blood cancer (AML) by boosting a natural anti-cancer pathway, potentially improving outcomes for adults with this disease.

Quick facts

Grant typeR01 grant
Study typeNIH-funded research
Funding institutionUniversity of Miami School of Medicine
NIH-funded
Lab location1 site (Coral Gables, United States)
Project startAugust 1, 2023
Project endJuly 31, 2028
Funding year2026
Award (this year)$351,131
NIH project number5R01CA282453-04
Project IDNIH-11398136 on NIH RePORTER

Who could benefit from this research

Could help

Adults with AML (including TET2-mutant forms) who may be candidates for adjuvant or novel therapies.

Less likely to help

Patients whose AML does not rely on TET2 pathways or who cannot tolerate vitamin C–related regimens may not benefit.

What this research studies

This grant explores whether high-dose vitamin C can activate TET enzymes in AML cells, helping to reprogram the cancer’s DNA and slow disease progression. The approach combines cellular and animal model studies to understand how residual TET2 activity could be enhanced in patients with TET2-mutant AML. If successful, the work could point toward a non-toxic adjuvant therapy that complements existing treatments, especially for older adults who may not tolerate aggressive chemotherapy. The project is led by Luisa Cimmino at the University of Miami and spans several years to translate lab findings toward potential clinical testing.

Why it matters

Potential benefit: If successful, this research could lead to a safer, vitamin C–based approach that improves survival or quality of life for AML patients, especially those with TET2 mutations.

How similar studies have gone: Preclinical studies have shown that ascorbate can alter AML epigenetics and slow leukemia progression in models, supporting further exploration.

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

Related research

Other NIH-funded projects on the same topics.

  • Why people with sickle cell disease have higher risk of blood cancers

    This project is checking if oxidative DNA damage in blood stem cells explains the higher risk of blood cancers after transplants or gene therapy in adults with sickle cell disease.

    NIH research Cincinnati Childrens Hosp Med Ctr · Cincinnati, United States · NIH-11310848

  • Boosting TET enzymes with vitamin C for acute myeloid leukemia

    This project explores whether giving vitamin C can boost a natural enzyme and help adults with TET2‑mutant acute myeloid leukemia.

    NIH research University of Miami School of Medicine · Coral Gables, United States · NIH-11161573

  • Understanding why sickle cell disease increases the risk of blood cancers.

    This study is looking at why adults with sickle cell disease might have a higher chance of developing blood cancers, and it aims to find out how stress on their cells and DNA damage could play a role, which could help improve treatments for people with sickle cell disease.

    NIH research Cincinnati Childrens Hosp Med Ctr · Cincinnati, United States · NIH-11110191

  • Using vitamin C to improve treatment for acute myeloid leukemia

    This study is looking at whether vitamin C can help treat people with acute myeloid leukemia, especially those with a specific gene mutation, by boosting the body's ability to fight cancer and making other treatments work better.

    NIH research University of Miami School of Medicine · Coral Gables, United States · NIH-10895423

  • Detecting cancer risk and treatment response in NF1 nerve tumors using cfDNA markers

    A doctor-led study will test blood-based DNA markers to detect when NF1-related nerve tumors become malignant and to monitor how well treatment is working.

    NIH research Mayo Clinic Rochester · Rochester, United States · NIH-11452312

  • Epigenetic Basis for Controlling Aggressive IDH-Mutant Gliomas

    Aims to understand and shape the epigenetic changes in IDH-mutant brain tumors to restrain their growth, which could lead to new, targeted treatment options for patients.

    NIH research State University New York Stony Brook · Stony Brook, United States · NIH-11449546

About this research

  1. This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
  2. Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
  3. For full project details, budget, and progress reports, visit the official NIH RePORTER page.
Last reviewed 2026-11-14 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.