Investigating a new treatment for relapsed myelodysplastic syndrome and acute myeloid leukemia.

"A PHASE I STUDY INVESTIGATING THE SAFETY & EFFICACY OF DANVATIRSEN AS MONOTHERAPY FOLLOWED BY COMBINATION WITH VENETOCLAX IN PATIENTS WITH RELAPSED/REFRACTORY MDS & AML."

NIH-funded research Albert Einstein College of Medicine · NIH-10917227

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 typeR01 grant
Study typeNIH-funded research
Funding institutionAlbert Einstein College of Medicine NIH-funded
Lab location1 site (Bronx, United States)
Project IDNIH-10917227 on NIH RePORTER

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.

Who could benefit from this research

Good fit: Ideal candidates for this research are adults diagnosed with relapsed or refractory myelodysplastic syndrome or acute myeloid leukemia.

Not a fit: Patients with newly diagnosed MDS or AML who have not yet undergone treatment may not benefit from this research.

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 performed: 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

Bronx, 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.