Stopping harmful PANoptosis to help people with myelodysplastic syndromes
Official title: Targeting PANoptotic pathways for Myelodysplastic Syndromes treatment
Funded at Loyola University Chicago · NIH-11306607 · Data updated 2026-11-12
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 type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Loyola University Chicago NIH-funded |
| Lab location | 1 site (Maywood, United States) |
| Project start | April 1, 2025 |
| Project end | March 31, 2030 |
| Funding year | 2026 |
| Award (this year) | $344,111 |
| NIH project number | 5R01CA301049-02 |
| Project ID | NIH-11306607 on NIH RePORTER |
Who could benefit from this research
✓Could help
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.
✕Less likely to help
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.
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.
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 gone: 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
-
Loyola University Chicago
Maywood, United States Active
Researchers
- Principal investigator: Zhang, Jiwang — Loyola University Chicago
- Study coordinator: Zhang, Jiwang
Related research
Other NIH-funded projects on the same topics.
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Interferon-γ plus donor immune cells to fight AML/MDS after partial donor transplant
This project tests a therapy that adds a signaling protein, IFN-γ, with donor immune cells to help treat relapsed AML/MDS after a haploidentical stem cell transplant.
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Whole-genome sequencing to refine risk in myelodysplastic syndromes (MDS) for older adults
This project uses clinical whole-genome sequencing to improve genetic profiling and better predict how MDS will progress in older patients.
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Targeting gasdermin D to treat myelodysplastic syndromes in people with bone marrow inflammation
A research project aims to find new ways to treat myelodysplastic syndromes by blocking a protein (gasdermin D) involved in harmful bone marrow inflammation.
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Understanding why some MDS patients relapse after a stem cell transplant
A study of patients with myelodysplastic syndromes after stem cell transplant to learn the cellular changes and gene activity that lead to relapse, with the goal of guiding future treatments.
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Risk factors and inflammation biomarkers for cardiovascular disease in older adults with myelodysplastic syndromes
A study focuses on older adults with myelodysplastic syndromes to understand why they have higher cardiovascular risk and how inflammatory proteins in the blood might help predict it.
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Therapeutic targeting of transcription-epigenetic defects in SF3B1-mutant MDS
A training-focused grant to help a clinician-scientist develop therapies for adults with a common bone marrow failure condition called SF3B1-mutant myelodysplastic syndrome (MDS).
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.