A Huge Milestone for RUNX1

By Giving List Staff   |   July 9, 2024

There is currently no treatment for RUNX1 familial platelet disorder (RUNX1-FPD). However, this year marks a significant milestone with the opening of two of the first-ever RUNX1-FPD patient-focused clinical trials.

Researchers are testing the drugs imatinib and sirolimus – two FDA-approved drugs that have shown promise in improving some of the blood-related issues RUNX1-FPD patients face. Imatinib could potentially raise RUNX1 protein levels up to normal levels, the same as those without RUNX1-FPD. Sirolimus might help restore normal blood cell production and improve the overall health of the blood system by blocking elevated levels of a protein called mTORC1.

Interestingly, blocking mTORC1 is also associated with extending lifespan in many different animal models. In mice it has reduced the incidence of cancer. The RUNX1 Research Program (RRP) is committed to providing comprehensive information to the patient community about these clinical trial opportunities, helping them make informed decisions about participation. Learn more on our Patient Research Opportunities page.


RUNX1 Research Program

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(916) 212-0410
Director of Development : Alex Gonzalez


To improve the quality of life and prevent cancer in patients with RUNX1-FPD. We do this by promoting awareness and funding world-class collaborative research with an empowered patient community. 

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We know you care about where your money goes and how it is used. Connect with this organization’s leadership in order to begin to build this important relationship. Your email will be sent directly to this organization’s Director of Development and/or Executive Director.

Before we started the RUNX1 Research Program, we didn’t know of anyone else having RUNX1-FPD beyond our family – but we knew others like us were out there, waiting to be found. Over the past six years, we’ve close to 400 patients join our community from across the world, and we know there are so many more. We are proud that RRP has granted over $11M to support 31 research projects towards finding a cure to benefit all our families.
Monica Babich, RRP co-founder, wife and mother of patients

Stop Cancer in Its Tracks

As part of their mission to stop cancer in its tracks, all donations to the RUNX1 Research Program fund cancer prevention initiatives – a mission that has never been more important. Medical professionals predict that, in the next year, cancer will become the number one cause of death in the United States. Your donations to the RUNX1 Research Program will fund research at the forefront of cancer detection and intervention. By studying the earliest signs of cancer today, we can prevent a fatal diagnosis tomorrow.

Key Supporters

Tim and Monica Babich
Georgetta and Charles Blackburn
Alex’s Lemonade Stand Foundation
The Edward P. Evans Foundation
Leukemia & Lymphoma Society
Mark Foundation for Cancer Research
Chan Zuckerberg