Myeloid-targeted oligonucleotide STAT3 blocker with TMLI for AML treatment
Official title: Myeloid cell-selective, oligonucleotide-based STAT3 inhibition combined with total marrow and lymphoid irradiation for immunotherapy of acute myeloid leukemia
Funded at Beckman Research Institute/city of Hope · NIH-11361556 · Data updated 2026-11-12
A new approach combines targeted radiation with a special gene-silencing treatment to help people with treatment-refractory acute myeloid leukemia.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Beckman Research Institute/city of Hope NIH-funded |
| Lab location | 1 site (Duarte, United States) |
| Project start | July 19, 2023 |
| Project end | June 30, 2028 |
| Funding year | 2026 |
| Award (this year) | $715,641 |
| NIH project number | 5R01CA284593-04 |
| Project ID | NIH-11361556 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are adults with treatment-refractory AML who are eligible for total marrow and lymphoid irradiation followed by allogeneic transplantation and who can tolerate investigational oligonucleotide therapy.
✕Less likely to help
Patients whose disease biology or transplant plan does not align with this combined modality approach may not benefit.
What this research studies
This project aims to help AML patients who have not responded to standard therapies by using total marrow and lymphoid irradiation (TMLI) to intensify treatment while limiting damage to healthy tissues, paired with a targeted oligonucleotide therapy aimed at the STAT3 pathway. Researchers plan to deliver STAT3 inhibitors directly to cancer- and immune-related cells in the marrow environment to reduce leukemia growth and improve the body’s anti-leukemia response. The study builds on prior data showing radiation can trigger immune signals, but also can promote tumor growth unless STAT3 is controlled. By combining these modalities, the team hopes to reduce relapse rates and improve overall survival after transplantation. The work involves a translational approach that could lead to new treatment options for patients with AML who have limited options.
Why it matters
Potential benefit: If successful, the approach could improve survival and reduce relapse for AML patients undergoing transplant by targeting both leukemia cells and the supportive tumor environment.
How similar studies have gone: Previous work suggests timing and combination strategies with radiation and immune-targeting agents can alter outcomes, but this exact oligonucleotide STAT3 inhibition with TMLI is novel.
Where this research is happening
-
Beckman Research Institute/city of Hope
Duarte, United States Active
Researchers
- Principal investigator: Kortylewski, Marcin — Beckman Research Institute/city of Hope
- Study coordinator: Kortylewski, Marcin
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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.