Stopping harmful PANoptosis to help people with myelodysplastic syndromes

Targeting PANoptotic pathways for Myelodysplastic Syndromes treatment

NIH-funded research Loyola University Chicago · NIH-11306607

Researchers aim to block a form of inflammatory cell death called PANoptosis to try to improve blood counts and reduce disease progression in people with myelodysplastic syndromes (MDS).

Quick facts

Grant typeR01 grant
Study typeNIH-funded research
Funding institutionLoyola University Chicago NIH-funded
Lab location1 site (Maywood, United States)
Project IDNIH-11306607 on NIH RePORTER

What this research studies

This research looks at bone marrow samples from people with MDS to understand how PANoptosis (a combined form of programmed cell death) contributes to low blood counts and disease progression. The team will study patient-derived cells and use laboratory and animal models to test genetic and drug approaches that reduce PANoptosis in mutant hematopoietic stem and progenitor cells. They will measure whether blocking PANoptosis improves healthy blood cell production and reduces the survival or expansion of the disease-causing clones. Findings will guide development of new therapies aimed at preventing cytopenias and transformation to acute myeloid leukemia.

Who could benefit from this research

Good fit: People diagnosed with myelodysplastic syndromes, especially those with spliceosome mutations such as SF3B1 or with persistent cytopenias, would be the most relevant candidates for related clinical work.

Not a fit: Patients whose bone marrow disease is driven by unrelated mechanisms or who are too frail for future therapies may not benefit from approaches targeting PANoptosis.

Why it matters

Potential benefit: If successful, this work could lead to treatments that raise blood counts, lower transfusion needs, and reduce the risk of progression to acute leukemia for people with MDS.

How similar studies have performed: Targeting PANoptosis is a relatively new idea with limited direct human data, though early clinical work on spliceosome inhibitors and related cell-death pathway approaches has shown promising signals.

Where this research is happening

Maywood, United States

Researchers

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 below.
Last reviewed 2026-06-13 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.