2026 Newsletters

Thanks For Contributing to the
2026 X-Out Blood Cancer Campaign!

Thanks to the incredible generosity of our community, the 2026 X-Out Blood Cancer Campaign raised over $22,000 for RUNX1 research. 

This year’s campaign featured video interviews with several RUNX1 researchers explaining the different approaches they are each taking to better understand how RUNX1 mutations lead to blood cancer and how to prevent it.

During a time when funding for rare disease research is more critical than ever, we appreciate everyone who donated, shared, and championed this campaign. Your support keeps vital research moving forward!

Congratulations to the 2026 RRP-ALSF RARE Grantee

“Modeling Clonal Hematopoiesis and Genetic Correction
in RUNX1-FPD Using Human iPSC-derived HSCs.”

The RUNX1 Research Program (RRP) and Alex’s Lemonade Stand Foundation (ALSF) are pleased to announce that Dr. Eirini Papapetrou and her team at the Icahn School of Medicine at Mount Sinai have been selected as the recipients of the 2026 Research Accelerating RUNX1 Exploration (RARE) Grant.

This award marks an important step toward advancing research into why some people with RUNX1-FPD develop blood cancer and how that process might one day be prevented or treated through new therapeutic approaches.

Congratulations to Dr. Papapetrou and her team!

Beyond the Blood: What RUNX1 May Be Doing in the Rest of Your Body

At this year's RUNX1 Patient Meeting, Dr. Katrin Ericson shared what we've learned so far about how RUNX1-FPD may affect the body beyond bleeding, bruising, and blood cancer risk.

In this new blog, she shares the key takeaways from her presentation, including possible connections to GI issues, skin symptoms, joint pain, migraines, fatigue, and the role inflammation may play across all of them.

The blog also covers what we still don’t know, why tracking symptoms over time matters, and how patients can use this information to have more informed conversations with their care team.

Watch the full video of the presentation or read the blog:

2026 RUNX1 Patient Meeting Summary

Earlier this month, we welcomed 65 patients, families, scientists, clinicians, and advocates to Atlanta for our 2026 RUNX1 Patient Meeting. 

The gathering featured meaningful patient stories, thoughtful community discussion, and timely updates from researchers and clinicians working to advance RUNX1-FPD research and care. Watch for an upcoming blog to learn more and watch recordings of select sessions.

We were honored to recognize Daphne Goldsmith as our 2026 RUNX1 Patient of the Year and Jill Williams as the 2025 X-Out Blood Cancer Challenge Top Fundraiser. Jill raised more than $12,000 to help support patients participating in the first RUNX1 clinical trials last year. 

Thank you to everyone who joined us and helped make this year’s RUNX1 Patient Meeting such a meaningful and energizing event. Stay tuned for an upcoming series of blogs featuring presenters from the Patient Meeting.

Reflections from Our May RUNX1-FPD Coffee Chat

Our May RUNX1 Patient & Family Coffee Chat brought together 12 patients and family members for an honest conversation about living with RUNX1-FPD. Participants shared experiences related to medical trauma, procedural anxiety, caregiving, self-advocacy, and the emotional challenges that can come with long-term uncertainty.

Several questions raised during the discussion (including those related to fertility, pregnancy, and whether certain physical traits, such as short stature, may be connected to RUNX1-FPD) reflect the kinds of questions the RUNX1 Patient Data Hub is designed to help answer over time through shared patient data and experiences. If you have not yet joined the RUNX1 Patient Data Hub, we encourage you to participate.

If you're looking for additional RUNX1-FPD resources or support, explore the "For Patients" and "For Clinicians" sections of our website, or email Amanda Eggen.

NIH RUNX1-FPD Clinical Research Study 

The NIH is currently enrolling individuals with RUNX1-FPD for a clinical research study focused on understanding why cancer risk varies so widely between affected families. 

Reported lifetime blood cancer risk in RUNX1-FPD ranges from approximately 11% in some families to nearly 100% in others. Researchers hope to identify the factors that influence why some individuals develop blood cancers while others do not.

This study is an important opportunity to advance understanding of RUNX1-FPD and support future efforts in risk assessment, early detection, and prevention. If you are interested in finding out if you may be eligible, please click below to learn more about the study and fill out the interest form:

10th Annual RUNX1 Scientific Conference Summary

Earlier this month, we welcomed scientists, clinicians, and patients to Atlanta for our 10th Annual RUNX1 Scientific Conference.

The gathering featured cutting-edge research presentations spanning clinical trials, genomics, and basic science, all advancing our understanding of RUNX1-FPD and how to better care for patients.

We were encouraged by meaningful progress on multiple fronts: early clinical trial data suggesting symptom benefit from low-dose therapies, growing insights into how somatic mutations like BCOR and TET2 shape disease progression, and emerging evidence that RUNX1-FPD may involve premature hematopoietic aging with developmental origins.

Researchers also presented promising new directions in epigenetic targeting, early progress on an active stem cell collection trial, and the first steps toward AI-assisted diagnostic tools.

Thank you to everyone who presented and participated in making this year's scientific conference such a productive and inspiring event.

RRP Research Webinar: “Mechanisms underlying aging-associated increased heterogeneity and selection for clonal hematopoiesis mutations in hematopoietic stem cell pools.”

Wednesday, June 3rd, 2026

9:00 a.m. PDT • 12:00 p.m. EDT • 5:00 p.m. BST

Aging plays a critical role in how blood stem cells function and how the risk for conditions like clonal hematopoiesis and leukemia evolves.

In this webinar, James DeGregori, PhD, will share his latest research on how aging reshapes hematopoietic stem cell fitness, including the role of RUNX1-related pathways and why certain mutations gain a selective advantage in this environment. These insights offer a deeper understanding of how leukemia risk develops, including in RUNX1-FPD.

Coming Soon: RRP–ALSF Early Career Investigator Grant

Applications will open soon for the RRP–ALSF Early Career Investigator (ECI) Grant, which supports early-career scientists studying inherited blood cancer predisposition disorders, including RUNX1-FPD. This program funds innovative research focused on intercepting the earliest changes that lead to disease.

Applications are due in December 2026. Learn more about past ECI grantees and their work:

RRP Medical Education Webinar: “Recognizing Pediatric Hereditary Hematologic Malignancies”

Thursday, June 11th, 2026

11:00 a.m. PDT • 2:00 p.m. EDT • 7:00 p.m. BST

Join us for an educational session on the germline origins of pediatric blood cancers and practical approaches to recognizing and managing hereditary conditions.

Featuring a presentation by Serine Avagyan, MD, PhD (University of California, San Francisco), who will be joined afterward for a live Q&A by Vivian Chang, MD (University of California, Los Angeles), and Kayla Hamilton, MS, CGC (Dana-Farber/Boston Children's Cancer and Blood Disorders Center).