Investigating a new treatment for aggressive nerve sheath tumors with specific genetic changes

Phase II Study of ASTX727 in Patients with PRC2 loss Malignant Peripheral Nerve Sheath Tumor (MPNST)

NIH-funded research Sloan-Kettering Inst Can Research · NIH-10707164

This study is testing a new drug for patients with malignant peripheral nerve sheath tumors (MPNST) that may help improve treatment outcomes, especially for those whose tumors have specific genetic changes.

Quick facts

Grant typeR01 grant
Study typeNIH-funded research
Funding institutionSloan-Kettering Inst Can Research NIH-funded
Lab location1 site (New York, United States)
Project IDNIH-10707164 on NIH RePORTER

What this research studies

This research focuses on malignant peripheral nerve sheath tumors (MPNST), which are aggressive cancers often associated with genetic changes in the PRC2 pathway. The study aims to evaluate the effectiveness of a new treatment using a drug that targets DNMT1, a protein that may enhance the cancer's response to therapy. Patients with MPNST who have lost PRC2 function will be treated with this drug to see if it improves outcomes compared to existing therapies. The approach involves advanced genetic screening and tailored treatment strategies to address the unique characteristics of these tumors.

Who could benefit from this research

Good fit: Ideal candidates for this research are patients diagnosed with malignant peripheral nerve sheath tumors that exhibit loss of PRC2 function.

Not a fit: Patients with MPNST who do not have PRC2 loss or those with other types of tumors may not benefit from this research.

Why it matters

Potential benefit: If successful, this research could lead to a new effective treatment option for patients with MPNST, potentially improving survival rates and quality of life.

How similar studies have performed: While research on MPNST is ongoing, this specific approach targeting DNMT1 in the context of PRC2 loss is novel and has not been extensively tested in clinical settings.

Where this research is happening

New York, 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.