OHSU Expands Trial to Prevent Cancer in Rare Genetic Condition

New $1.89 million grant will test anti-inflammatory drug to delay or reduce leukemia risk in RUNX1 mutation carriers.

Apr. 8, 2026 at 3:20am

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A ghostly, translucent X-ray image of a human blood cell, its internal structures visible as glowing lines against a dark background, conceptually representing the cellular-level approach of a new cancer prevention trial.An experimental treatment aims to prevent blood cancer in people with a rare genetic disorder by targeting the underlying cellular processes.Boston Today

Researchers at Oregon Health & Science University have received a $1.89 million grant from the American Cancer Society and RUNX1 Research Program to expand a clinical trial evaluating whether an anti-inflammatory drug could help delay or reduce the risk of blood cancer in people with an inherited genetic condition known as RUNX1 familial platelet disorder (RUNX1-FPD). The trial will recruit 10-12 participants with RUNX1-FPD to test the safety and efficacy of low-dose sirolimus, an FDA-approved drug, in preventing the progression to leukemia.

Why it matters

RUNX1-FPD is a rare inherited condition that increases the risk of developing aggressive forms of blood cancer by 30-50%. If successful, this trial could lead to the first targeted cancer-prevention strategy for this high-risk group and serve as a model for people with other inherited cancer predispositions.

The details

The trial will evaluate the safety of a low daily dose of sirolimus taken over six months and assess its effects on blood and bone marrow cell function, immune response, inflammation, and cellular processes involved in the development of leukemia. Researchers will collect blood and bone marrow samples throughout the treatment to determine if the improvements seen in preclinical studies translate to people.

  • The trial is currently underway at MD Anderson Cancer Center and will expand to OHSU.
  • The $1.89 million, four-year grant was awarded in 2026.

The players

Anupriya Agarwal

Professor of medicine (hematology/medical oncology), cell and developmental and cancer biology, and molecular and medical genetics in the OHSU School of Medicine and OHSU Knight Cancer Institute, and the principal investigator at OHSU for the trial.

Courtney DiNardo

Team principal at MD Anderson Cancer Center, collaborating with Agarwal on the trial.

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What they’re saying

“The overarching goal is to intervene and potentially prevent the progression to leukemia. Considering RUNX1-FPD patients live in a pre-malignant state for many years, we see an opportunity to prevent or delay the development of leukemia.”

— Anupriya Agarwal, Principal Investigator

“We'll monitor each patient over the course of a year. We want to make sure they can take the medication over a long term while staying healthy and improving cellular function.”

— Anupriya Agarwal, Principal Investigator

What’s next

If the trial shows the low-dose sirolimus treatment is safe, the next step would be a larger clinical trial to evaluate whether this approach can delay or reduce the risk of cancer in RUNX1-FPD patients.

The takeaway

This trial represents a promising step towards a targeted cancer-prevention strategy for individuals with an inherited genetic condition that significantly increases their risk of developing aggressive blood cancers. If successful, the findings could have broader implications for other types of platelet disorders and inherited cancer predispositions.