NIH-funded research R01 grant

Targeting metabolic weak spots in myelodysplastic syndrome stem cells

Official title: Metabolic targeting of heterogenous myelodysplastic syndrome stem cells

Funded at University of Colorado Denver · NIH-11188998 · Data updated 2026-11-12

The team is trying metabolism-based treatments to kill the stem cells that drive high-risk myelodysplastic syndrome (MDS).

More Myelodysplastic Syndrome research and trials

Quick facts

Grant typeR01 grant
Study typeNIH-funded research
Funding institutionUniversity of Colorado Denver
NIH-funded
Lab location1 site (Aurora, UNITED STATES)
Project startSeptember 19, 2023
Project endAugust 31, 2028
Funding year2025
Award (this year)$423,743
NIH project number5R01CA286717-03
Project IDNIH-11188998 on NIH RePORTER

Who could benefit from this research

Could help

Ideal candidates are people with high-risk MDS, particularly those enrolled in or eligible for clinical trials combining azacitidine with agents like omacetaxine.

Less likely to help

Patients with low-risk MDS or unrelated blood disorders are unlikely to directly benefit from this project at this time.

What this research studies

This project looks for metabolic weak points in the different kinds of MDS stem cells that keep the disease coming back. Researchers combine laboratory experiments with samples and data from an active clinical trial testing omacetaxine plus azacitidine (NCT03564873) to find which stem cell subsets survive current treatment. The goal is to map vulnerabilities in those resistant subsets and design next-generation approaches that are more precise and less toxic than global protein-translation blockers. If successful, the work aims to guide therapies that better eliminate the root cells that cause relapse.

Why it matters

Potential benefit: If successful, this work could lead to treatments that more completely eliminate the MDS stem cells and reduce relapses.

How similar studies have gone: An ongoing trial combining omacetaxine and azacitidine has produced promising responses but still shows some disease progression, so this project builds on those results to target remaining stem cell subsets.

Where this research is happening

  • University of Colorado Denver
    Aurora, United States
    Active

Researchers

  • Principal investigator: Pietras, Eric M — University of Colorado Denver
  • Study coordinator: Pietras, Eric M

Related research

Other NIH-funded projects on the same topics.

  • Targeting diverse stem cells in myelodysplastic syndrome to eradicate the disease

    Aimed at identifying weaknesses in the diverse stem cell populations of myelodysplastic syndrome so future treatments can wipe out the root cause of the disease.

    NIH research University of Colorado Denver · Aurora, UNITED STATES · NIH-11401024

  • New treatment approach for myelodysplastic syndrome and acute myeloid leukemia

    This project aims to develop a new antibody-based therapy to help people with MDS or AML, especially older or frail patients who cannot tolerate high-dose chemotherapy.

    NIH research Avantgen, INC. · San Diego, United States · NIH-11189625

  • Investigating the role of PI3 kinase in Myelodysplastic Syndrome

    This study is looking at how certain changes in genes might affect blood cell production in older adults with Myelodysplastic Syndrome (MDS), and it hopes to find new ways to help improve treatment for people with this condition.

    NIH research Albert Einstein College of Medicine · Bronx, United States · NIH-11112391

  • Identifying harmful and harmless genetic variations in blood cancer predisposition genes

    This study is looking at how certain changes in a gene called DDX41 might affect the chances of developing blood disorders like myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML), so that doctors can provide better advice and support for patients at risk.

    NIH research University of Wisconsin-Madison · Madison, United States · NIH-11090449

  • Investigating CCRL2's role in myelodysplastic syndrome

    This study is looking at how a specific gene called CCRL2 affects the growth of harmful cells in myelodysplastic syndrome (MDS), especially in men, to find new ways to improve treatment for people with this blood disorder.

    NIH research Johns Hopkins University · Baltimore, United States · NIH-11076301

  • Understanding how immune evasion occurs in high-risk myelodysplastic syndrome

    This study is looking at how a protein called VISTA helps high-risk myelodysplastic syndrome (HR-MDS) avoid being attacked by the immune system, with the goal of finding new ways to improve treatments for patients like you.

    NIH research Vanderbilt University · Nashville, UNITED STATES · NIH-11069224

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.