Blocking PRMT9 to help eliminate acute myeloid leukemia

Targeting Protein Arginine Methyltransferases to Eradicate Acute Myeloid Leukemia

NIH-funded research Beckman Research Institute/city of Hope · NIH-11234307

A new drug that blocks the PRMT9 enzyme aims to switch on immune signals in AML cells so the immune system can better attack acute myeloid leukemia, possibly together with PD‑1 immunotherapy.

Quick facts

Grant typeR01 grant
Study typeNIH-funded research
Funding institutionBeckman Research Institute/city of Hope NIH-funded
Lab location1 site (Duarte, United States)
Project IDNIH-11234307 on NIH RePORTER

What this research studies

If you have acute myeloid leukemia (AML), researchers are developing a drug that blocks an enzyme called PRMT9 to awaken immune signaling inside leukemia cells. They use samples from patients and mouse AML models to show that PRMT9 inhibition produces cGAMP and activates the cGAS‑STING/type I interferon pathway to prime immune responses. The team's PRMT9 inhibitor (LD2) cleared AML in animal experiments when given alone and especially when combined with a PD‑1 immune checkpoint blocker. These are preclinical results that could lead to future clinical trials testing this combination in people with AML.

Who could benefit from this research

Good fit: People with acute myeloid leukemia—particularly those with relapsed or treatment‑resistant disease—would be the most likely candidates for future trials based on this work.

Not a fit: People without AML, those with other types of blood cancer, or patients currently doing well on standard therapies would be unlikely to benefit directly from this AML‑focused research.

Why it matters

Potential benefit: If successful, this approach could become a new immune‑based treatment that helps patients' own immune systems clear AML and improve long‑term outcomes.

How similar studies have performed: Preclinical work, including the investigators' mouse studies, showed promising AML eradication when PRMT9 inhibition was combined with PD‑1 blockade, but this strategy is novel and has not yet been tested in patients.

Where this research is happening

Duarte, 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.