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Thank you to everyone who joined us this September for the 9th Annual RUNX1 Scientific Conference and the RUNX1 Patient Meeting. Together, these gatherings created a powerful space for collaboration, discovery, and connection across our global community.
We had more than 160 registrants from 20 countries for this year’s RUNX1 Scientific Conference (Sept. 9–10). Attendee discussions centered around: -
The factors that may influence clonal evolution and the consistent pattern of co-occurring somatic mutations (BCOR, TET2, DNMT3a, PHF6, KRAS, SRSF2).
- How RUNX1 mutations affect immune system pathways.
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The role of a high-fiber plant-based diet in supporting a healthy gut and reducing inflammation.
- Developments in the allogeneic stem cell transplant landscape and the potential for gene editing therapies.
- Identification of a new drug target for RUNX1-FPD.
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The early encouraging results from ongoing clinical trials, particularly from the low-dose sirolimus study.
- Blood cancer risk in pediatric RUNX1-FPD individuals.
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The RUNX1 Patient Meeting (Sept. 19) reached its highest registration numbers yet, with 64 registrants from 8 countries. Families and individuals living with RUNX1-FPD came together to participate in discussions and expert-led presentations. Alongside scientific conference reflections for RUNX1 individuals and family members from RRP's Executive Director, Dr. Katrin Ericson, the meeting featured:
Community & Wellbeing - A wellbeing session led by patient community members, including a caregiver-focused discussion.
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A behavioral health expert shared findings of higher-than-average rates of anxiety among RUNX1-FPD individuals and offered proven techniques for managing this anxiety.
Clinical Trial Updates - Stem Cell Harvesting & Banking Study: Manageable, limited side effects reported; sufficient number of cells collected from the first two participants for potential future gene editing strategies.
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Imatinib Trial: Well-tolerated in the first participant, and now the study team has expanded eligibility criteria and reduced onsite visit requirements.
- Low-Dose Sirolimus Pilot Study: Early results among four participants show promise for improved blood health and the potential for reduced blood cancer risk based on improvements in platelet function and reduced inflammation markers.
NIH RUNX1 Clinical Research Study - Encouraging pregnancy and delivery outcomes for women with RUNX1-FPD, highlighting safe management with proper bleeding precautions.
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New age-specific data on blood cancer risk in RUNX1-FPD.
- Updated guidance from the NIH team based on new risk data:
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Family member testing of all potentially affected individuals when a family member is diagnosed with RUNX1-FPD.
- Blood testing in children every 3–6 months (2–4 times per year).
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Baseline bone marrow biopsy (BMB) for children, performed as soon as it is considered safe, with possible repeat biopsies over several years to establish a baseline pattern.
These are recommendations only. The decision to perform a bone marrow biopsy in a child should always be made jointly by the physician and the child’s parents/guardians.
We are deeply grateful to every presenter, facilitator, and participant who made this year’s meetings possible, and look forward to continuing the momentum next year in Atlanta, Georgia, at our 10th Annual Scientific Conference (May 6–7), and our RUNX1 Patient Meeting (May 7–8).
Click below for more details about each of these events and to register: |
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ATTENTION: Parents of Children with RUNX1-FPD
As we plan for the 2026 RUNX1 Patient Meeting, we’re exploring the possibility of offering sessions or activities designed specifically for patients under 18 with RUNX1-FPD.
When you register, you’ll see a section for parents and guardians to indicate whether you’d be interested in bringing your child(ren), along with their ages. Your responses will help us gauge interest and determine whether we can provide programming for kids in similar age groups.
Sharing your plans with us early will help us understand if this could be a valuable addition for families. RRP offers financial support for travel and lodging to RUNX1 patients and their close family members attending the meeting (this includes children).
Please register today and indicate your interest in the form! |
Congratulations to the 2025 RRP-ACS LEAP Grant Recipients |
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RRP and the American Cancer Society (ACS) are pleased to announce the recipients of the inaugural Leukemia Exploration And Prevention (LEAP) Team Grant. Project: “Intrinsic and extrinsic mechanisms driving clonal fitness and leukemic transformation in familial platelet disorder.”
Principal Investigator: Wenbin Xiao, MD, PhD – Memorial Sloan Kettering Cancer Center Team Principals: Robert Bowman, PhD, and Nancy A. Speck, PhD – University of Pennsylvania
Co-Investigators: Andriy Derkach, PhD, and Richard Koche, PhD – Memorial Sloan Kettering Cancer Center |
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Dr. Xiao previously received an Early Career Investigator Award from RRP and Alex’s Lemonade Stand Foundation, which he used to develop novel RUNX1 mouse models to study clonal evolution and identify key epigenetic dependencies in AML.
Building on his prior work, this collaborative team will integrate single-cell technologies, experimental hematology, and computational epigenetics to: -
Characterize RUNX1-driven transcriptional and epigenetic programs in hematopoietic stem and progenitor cells (HSPCs)
- Model clonal evolution and leukemogenic progression in RUNX1-FPD
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Define molecular vulnerabilities and potential intervention points to inform prevention-based therapeutic strategies
This first award marks a significant milestone in advancing prevention-focused research in RUNX1-FPD and informing the broader field of leukemia interception. Congratulations to this grant recipient team!
The next RFA for the next LEAP Grant cycle is coming soon! - Mechanisms: Single-PI awards ($215,000/year × 4 years) and Team awards ($430,000/year × 4 years), plus indirects
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Scope: Mechanistic and translational research in clonal hematopoiesis, leukemogenesis, and cancer interception
- Review process: Formal progress review at Year 2 to assess progress and refine aims
Please check back here for more information: |
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Stem Cell Harvesting & Banking Clinical Trial at CHOP Wednesday, October 22nd
12:00pm PDT • 3:00pm EDT • 8:00pm BST |
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Join Dr. Tim Olson from the Children’s Hospital of Philadelphia (CHOP) to learn more about the expansion of this groundbreaking clinical trial. As the study lead at the CHOP site, Dr. Olson will present details on study goals, eligibility criteria, and participation opportunities.
This trial, also being conducted at MD Anderson Cancer Center in Houston, Texas, aims to evaluate the safety and feasibility of collecting and banking blood stem cells from RUNX1-FPD patients. This session is designed for RUNX1 patients and families interested in learning more about joining this research opportunity. However, anyone is welcome to attend! |
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UK Cancer Genetics Group Webinar for UK-based RUNX1 Patients
Thursday, October 23rd 4:00pm BST |
Join the UK Cancer Genetics Group (UKCGG) for a one-hour webinar designed for the UK RUNX1-FPD patient community. This event offers a unique opportunity for patients and their families to connect, ask questions, and learn more about resources and guidelines tailored to their specific region.
Presenters will share updates on the UK infrastructure for clinical pathways, including the National Inherited Cancer Predisposition Register, reproductive options for individuals with RUNX1, and UK clinical guidelines for patients and clinicians. The session will conclude with an open discussion and Q&A. Presenters: - Dr. Katie Snape – King’s College Hospital NHS Foundation Trust
- Dr. Terri McVeigh – The Royal Marsden NHS Foundation Trust
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Ms. Beverley Speight – Cambridge University Hospitals NHS Foundation Trust
- Ms. Clair Engelbrecht – King’s College Hospital NHS Foundation Trust
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The Value of Genetic Counselors for RUNX1-FPD and Rare Disease Care
By: Taylor Burg, Stanford University School of Medicine Genetic Counseling Student |
From explaining test results to connecting families with resources and support, genetic counselors are a crucial part of rare disease care. In this new blog, genetic counseling student Taylor Burg shares how counselors train, what they do for patients, and why their role matters for families with RUNX1-FPD. |
Interested in contributing to the blog? We'd love to hear from you!
Contact Catherine Pelton to learn more about writing as a guest author. |
For more information and/or questions about any of the articles in the "For Patients" section below, please email Amanda Eggen, RRP’s Director of Patient Engagement & Clinical Programs.
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Check Out Our Helpful Resources for RUNX1-FPD Patients & Families |
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Living with RUNX1-FPD can bring many questions about health, the latest research, and even the basics of understanding the disease. To help, we’ve created a section on our website with resources specifically designed for patients and families, along with a collection of videos from past webinars and events, and a blog written for patients and their families.
In our Patient & Family Resources section, you’ll find: - Downloadable guides and fact sheets to help you and your doctors understand RUNX1-FPD (algunas disponibles en español).
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One-page summaries of current RUNX1 clinical trials.
- Practical tools created by request, such as an informational card for first responders and a customizable letter for caregivers at camps or schools.
In our Video Library, you’ll find: -
Webinar recordings with updates on ongoing RUNX1 clinical trials.
- Tips for healthy living to limit cancer risk.
- Personal stories from patients and family members sharing their journeys.
On our RUNX1 Pulse Blog, you’ll find: -
Clear explanations of the latest RUNX1-FPD research findings.
- Stories and perspectives from others living with RUNX1-FPD.
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Information on how to make the greatest impact for the RUNX1-FPD community.
All of these resources are free and updated regularly, ensuring you always have access to the latest, trustworthy information. We hope you’ll visit and share these links with others in your family.
If you have any questions, would like to contribute a blog, or have ideas for additional resources, please email Amanda Eggen. We’d love your input!
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For more information and/or questions about any of the articles in the "For Researchers" section below, please email Dr. Katrin Ericson, RRP’s Executive Director. |
2026 RUNX1 Scientific Conference Wednesday, May 6 - Thursday, May 7, 2026
Emory Conference Center Hotel • Atlanta, Georgia |
Please register* to join us in Atlanta, Georgia, for the 10th Annual RUNX1 Scientific Conference, taking place in person on Wednesday, May 6, and Thursday, May 7, 2026, at the Emory Conference Center Hotel. The 2026 theme, “Catalyzing Change: The Next Chapter in RUNX1,” highlights our shared commitment to advancing discovery and improving outcomes for individuals affected by RUNX1-FPD. Through collaborative discussion and cross-disciplinary exchange, the conference aims to accelerate progress towards cancer interception therapies for RUNX1-FPD.
The conference will feature plenary talks, collaborative breakout sessions, and poster sessions dedicated to graduate and postdoctoral student researchers.
Attendees will gain access to the latest findings, forge new research partnerships, and help shape the future of RUNX1-FPD science. We look forward to welcoming you to Atlanta in May 2026!
*If you are a current RRP grantee or SAB member, please contact Lydia Harper for a registration promo code.
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Applications Open for the RRP-ALSF 2026 RARE Grant Deadline: Thursday, December 11, 2025 RRP and Alex’s Lemonade Stand Foundation (ALSF) are continuing their long-standing collaboration through the 2026 Research Accelerating RUNX1 Exploration (RARE) Grant. This $250,000, two-year award supports research that identifies druggable oncogenic pathways and advances cancer interception and prevention strategies toward clinical application. Click below for more information and application guidelines: |
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Updated Research Opportunities for RUNX1-FPD Patients If you care for any RUNX1-FPD patients and their families, please share these clinical research participation opportunities with them:
Stem Cell Harvesting & Banking Clinical Study (NCT06414889)
This trial is evaluating the feasibility of mobilizing, collecting, and cryopreserving autologous CD34+ cells in RUNX1-FPD patients for potential future gene correction strategies. Patients may enroll at both MD Anderson Cancer Center (Houston, TX) and the Children’s Hospital of Philadelphia (CHOP). Locations: CHOP – Philadelphia, PA; MD Anderson – Houston, TX
NIH RUNX1-FPD Clinical Research Study (NCT03854318)
A natural history study investigating genomic and clinical factors contributing to cancer penetrance in RUNX1-FPD through prospective follow-up and biospecimen collection.
Imatinib Phase 1b Clinical Trial (NCT06090669)
Determining the safety and optimal dosing of imatinib to evaluate its potential in restoring RUNX1 protein activity. Location: Both studies are at the NIH – Bethesda, MD Sirolimus Pilot Study (NCT06261060) Evaluating the safety and tolerability of low-dose sirolimus (rapamycin) to improve overall hematopoiesis and delay or prevent hematologic malignancy.
Location: MD Anderson – Houston, TX RUNX1 Patient Tissue Bank
Provides individuals with a confirmed germline RUNX1 variant (pathogenic, likely pathogenic, or VUS) the opportunity to collect and ship peripheral blood or bone marrow aspirate/biopsy during routine clinical care at their local institutions. These biospecimens are centrally banked and released to qualified investigators studying RUNX1-FPD disease mechanisms, early leukemogenesis, and cancer prevention strategies.
Location: Local blood/bone marrow collection site
Please Note: The NIH Clinical Research Study remains our highest priority. The Tissue Bank offers an alternative option for sample collection. We coordinate closely with NIH to prevent duplication of effort.
RUNX1 Patient Data Hub
A secure, anonymized platform gathering longitudinal patient-reported outcomes to map the clinical spectrum of RUNX1-FPD and inform future research. Participation is worldwide and web-based.
Location: Online You can find patient-friendly information, eligibility criteria, and downloadable summaries for each of these here: |
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Subscribe to RRP’s YouTube Channel!
Subscribe to our YouTube Channel for the latest updates on RUNX1-FPD research, patient resources, and educational webinars. By subscribing, you'll: -
Get notified when new webinars, updates, and stories are posted
- Watch expert presentations and patient Q&As
- Stay connected to the RUNX1-FPD research and support community
Subscribe today and turn on notifications to stay up to date! |
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Please consider a gift of any size today. RRP commits 100% of direct donations to fund research and educational programs. Thank you for being part of this effort to advance research and improve outcomes for the RUNX1-FPD community. |
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