Investigating a new treatment for relapsed myelodysplastic syndrome and acute myeloid leukemia.
Official title: "A PHASE I STUDY INVESTIGATING THE SAFETY & EFFICACY OF DANVATIRSEN AS MONOTHERAPY FOLLOWED BY COMBINATION WITH VENETOCLAX IN PATIENTS WITH RELAPSED/REFRACTORY MDS & AML."
Funded at Albert Einstein College of Medicine · NIH-10917227 · Data updated 2026-11-12
This study is testing a new drug called Danvatirsen to see how well it works and how safe it is for people with relapsed or hard-to-treat myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML), both on its own and when combined with another drug, Venetoclax, to help improve treatment results and reduce the chances of the cancer coming back.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Albert Einstein College of Medicine NIH-funded |
| Lab location | 1 site (Bronx, United States) |
| Project start | September 1, 2023 |
| Project end | May 31, 2028 |
| Funding year | 2025 |
| Award (this year) | $650,000 |
| NIH project number | 5R01FD007836-02 |
| Project ID | NIH-10917227 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are adults diagnosed with relapsed or refractory myelodysplastic syndrome or acute myeloid leukemia.
✕Less likely to help
Patients with newly diagnosed MDS or AML who have not yet undergone treatment may not benefit from this research.
What this research studies
This research focuses on evaluating the safety and effectiveness of a new drug called Danvatirsen, which targets specific cancer cells in patients with relapsed or refractory myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML). The study will first assess Danvatirsen as a standalone treatment and then in combination with another drug, Venetoclax, to see if this approach can improve patient outcomes. By targeting the underlying cancer stem cells, the research aims to reduce the chances of disease relapse and improve survival rates. Patients will be closely monitored for any side effects and overall response to the treatment.
Why it matters
Potential benefit: If successful, this research could lead to more effective treatment options for patients with relapsed MDS and AML, potentially improving survival rates.
How similar studies have gone: Previous research has shown promising results with similar approaches targeting cancer stem cells, suggesting potential for success in this study.
Where this research is happening
-
Albert Einstein College of Medicine
Bronx, United States Active
Researchers
- Principal investigator: Shastri, Aditi — Albert Einstein College of Medicine
- Study coordinator: Shastri, Aditi
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.