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Now Accepting Applications for the 2026 RARE Grant Application Deadline: Thursday, December 11, 2025 |
For nearly a decade, the RUNX1 Research Program (RRP) and Alex’s Lemonade Stand Foundation (ALSF) have partnered to fund innovative research aimed at improving outcomes for individuals and families living with RUNX1-FPD.
We are pleased to continue this successful collaboration by announcing that applications are now being accepted for the 2026 RRP-ALSF Research Accelerating RUNX1 Exploration (RARE) Grant.
The RARE Grant supports research focused on identifying druggable oncogenic pathways and advancing cancer interception and prevention strategies toward clinical application. Funded projects will demonstrate clear potential to benefit patients and families affected by RUNX1-FPD.
Total funding is $250,000 over two years (with a maximum of $125,000 per year). Review the full program guidelines and submit your application below: |
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RUNX1-FPD results from inherited mutations in RUNX1, significantly elevating leukemia risk through early clonal hematopoiesis driven by acquired somatic mutations, notably in BCOR.
Dr. Bowman’s team hypothesizes that combined RUNX1/ BCOR mutations trigger epigenetic dysregulation of the HoxA transcription factor family, dependent upon Menin for expression.
Leveraging Menin inhibitors, already recognized as breakthrough therapies in pediatric AML, Bowman’s group aims to suppress this HoxA-driven clonal expansion, potentially intercepting progression to leukemia.
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The project will also utilize CRISPR-based genetic screens to identify further therapeutic targets against RUNX1/BCOR-mutant clones. |
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Register Today For The 2026 10th Annual RUNX1 Scientific Conference
“Catalyzing Change: The Next Chapter in RUNX1" Wednesday, May 6 & Thursday, May 7, 2026 Atlanta, Georgia • Emory Conference Center Hotel |
Only a Few Days Left for Early Bird Registration!
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Register now to secure the early bird rate of $150 per person. After August 5, the rate increases to $300.
We’re excited to announce that the in-person 10th Annual RUNX1 Scientific Conference will be held on Wednesday, May 6, and Thursday, May 7, 2026, for the first time in Atlanta, Georgia, at the beautiful Emory Conference Center Hotel! The 2026 Conference theme, “Catalyzing Change: The Next Chapter in RUNX1,” highlights our shared commitment to advancing discovery and improving outcomes for individuals affected by RUNX1-FPD. Sessions will feature: -
Research presentations from leaders in the field
- Collaborative breakout discussions
- Poster presentations by graduate and postdoctoral researchers
Attendees will gain early access to new findings and explore emerging research directions that are helping to shape the future of RUNX1-FPD care. More information coming soon!
We hope to see you in Atlanta next year!
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Register Today For The 2026 RUNX1 Patient Meeting
Thursday, May 7 & Friday, May 8, 2026 Atlanta, Georgia • Emory Conference Center Hotel |
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We are excited to invite patients and family members to the 2026 RUNX1 Patient Meeting, hosted in person next spring at the beautiful Emory Conference Center Hotel in Atlanta, Georgia. This event is designed to support, inform, and connect individuals and families affected by RUNX1-FPD. Whether you're newly diagnosed, caring for a loved one, or looking to connect with others on a similar journey, we invite you to join us for this in-person gathering. Schedule for Patients and Families Thursday, May 7 | Welcome, Workshops, Poster Session & Keynote Dinner On Day 1, join fellow patients, families, researchers, and clinicians for an afternoon of workshops exploring the latest advancements and challenges in RUNX1-FPD treatment discovery and the impact RUNX1-FPD has on lived experience. Attend a poster session where you can learn directly from scientists about their research. Or if you’re interested, present a poster sharing your personal experience with RUNX1-FPD.
The evening will conclude with dinner, featuring an inspiring speaker and the announcement of the RUNX1 Award of X-Cellence winners. We welcome anyone from the RRP community who would like to participate!
Friday, May 8 | Patient Meeting Day 2 of the Patient Meeting includes: -
Research and clinical trial updates presented in accessible, patient-focused language
- Peer support and small-group conversations
- Wellness strategies for managing uncertainty
- Presentations and discussions led by the patient community
The event officially ends on Friday, and you are welcome to stay at the conference center through Saturday morning. There are currently no scheduled activities for the weekend, but we are happy to provide ideas for things to do in Atlanta if you’d like to relax or explore with friends and family!
Please Note: We can cover flight costs and hotel for Thursday and/or Friday night for each patient and one family member attending the meeting events together. We will follow up with more details soon to help support your travel planning. |
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Parenting in the Gray: Navigating the Uncertainty of RUNX1-FPD
By: Monica Babich |
When your child is diagnosed with a rare genetic disorder like RUNX1-FPD, you don’t just receive a clinical label. You enter a lifelong negotiation with uncertainty.
You go from being a parent to also becoming an advocate, and in many ways, an interpreter, reading between the lines of emerging science, evolving guidelines, your child’s day-to-day experience, and your gut instinct.
I know because I’ve been there … I am there. |
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. |
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2025 RRP Virtual RUNX1 Patient Meeting Friday, September 19 • 9:00 a.m. – 2:00 p.m. PDT |
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Join us online on Friday, September 19, 2025, for a supportive, patient-centered day featuring: - A personal patient story
- Breakout sessions on parenting, managing uncertainty, and caregiver support
- Wellness strategies
- A scientific conference research update breakdown
- Clinical trial updates from investigators leading the sirolimus, imatinib, and stem cell harvesting and banking studies
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Open discussion and live Q&A with experts and peers
Whether you're newly diagnosed or have been with us for years, this meeting is designed with you and your family in mind. For questions about the Patient Meeting, please contact Dr. Amanda Eggen, RRP’s Director of Patient Engagement and Clinical Programs. |
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Thursday, August 28 11:30 AM PDT • 2:30 PM EDT • 7:30 PM BST |
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Register today to join us for the August Research Guided by Patients Committee (RGPC) meeting!
This quarter’s discussion will focus on the RRP website. We’d love your input on the layout, content, and tools, including what’s working well, what feels confusing, and how we can make the site more useful for you. The RGPC brings together patients, families, caregivers, researchers, and clinicians to ensure diverse RUNX1-FPD perspectives help shape research, education, advocacy, and awareness initiatives. Patients and family members are always welcome, and the group is open to new members!
If you’d like to learn more about the RGPC and how to get involved, please contact Amanda Eggen. |
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New Support Group for RUNX1-FPD Parents Now on WhatsApp |
We’re excited to share that a private WhatsApp group is now available exclusively for parents and guardians of individuals with RUNX1-FPD. Created by and for RUNX1 parents, the group offers a welcoming space to share experiences, ask questions, and support one another through the ups and downs of this journey. The group is already active, with members exchanging advice, insights, and encouragement. To protect privacy, participation is by invitation only.
Interested in joining? Simply send an email to Amanda at aeggen@runx1-fpd.org with the mobile number linked to your WhatsApp account, and we’ll take care of the rest. New to WhatsApp? It’s a free, secure messaging app with end-to-end encryption. Learn more or download it here: https://www.whatsapp.com. |
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Need Help with the Patient Data Hub? Watch Our Tutorial! |
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Whether you’re new to the RUNX1 Patient Data Hub (RUNX1 PDH) or ready to retrieve your existing account on the new platform (Matrix), we’re here to help you get started! RRP’s Amanda Eggen hosted a webinar designed specifically for patients and families interested in learning more about how the RUNX1 PDH works and how participation supports improved care, more innovative research, and a deeper understanding of RUNX1-FPD. It walks you through everything you need to know: - What the RUNX1 PDH is and why your contribution matters
- How to set up an account and complete your consent
- Tools available to track your health, view data, and access insights
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We’ve also created three short demos that you can watch individually if you prefer:
If you have questions or need assistance with your account, please don't hesitate to contact Amanda directly at aeggen@runx1-fpd.org. Thank you for being part of the effort to improve the lives of individuals and families affected by RUNX1-FPD! |
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9th Annual Virtual RUNX1 Scientific Conference
Tuesday, September 9 & Wednesday, September 10 |
Join us this September as we explore “Navigating New Frontiers” in RUNX1-FPD science, innovation, and patient-centered care. |
This technical, science-focused event features presentations from RRP grantees and other leading experts across the field. Topics include: -
Modulating Inflammation to Control Clonal Evolution
- RUNX1 and the Immune System
- Advances in Transplants and Cell Therapies
- Controlling Inflammation with Diet in Blood Cancer Patients
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RUNX1 Mutations - Impact on Gene Regulatory Networks & Variant Classification
- RUNX1 Patient Experiences & Clinical Research
- Deciphering Maladaptive from Adaptive Somatic Mutations
For questions about the Scientific Conference, please contact Dr. Katrin Ericson, RRP’s Executive Director.
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Research Opportunities For Your RUNX1-FPD Patients |
If you care for any RUNX1-FPD patients and their families, please consider referring them to one or more of the following clinical studies. NEW: RUNX1 Patient Tissue Bank (Tissue Bank)
(Location: Local blood/bone marrow collection site)
The RUNX1 Tissue Bank offers individuals with a confirmed germline RUNX1 variant classified as pathogenic, likely pathogenic, or a variant of uncertain significance (VUS) the opportunity to contribute peripheral blood or bone marrow aspirate during routine clinical care at their local institutions. These biospecimens are securely banked and made available to qualified investigators researching RUNX1-FPD disease mechanisms, early leukemogenesis, and cancer prevention strategies.
Please note: The NIH Clinical Research Study remains our highest priority. However, for the ~30% of patients unable or unwilling to participate in this study, the Tissue Bank offers an alternative way to contribute to research. We coordinate closely with the NIH team to avoid duplication and ensure alignment. If you have a patient who is enrolled at the NIH but is not having samples collected, please contact us so that we can help coordinate this.
RUNX1 Patient Data Hub
(Location: web-based)
A secure, anonymized platform for collecting longitudinal patient-reported outcomes to map the timeline and spectrum of clinical manifestations associated with RUNX1-FPD, to inform future research design and development, and to optimize care. Patients may participate from anywhere worldwide.
Sirolimus Pilot Study (Location: MD Anderson Cancer Center - Houston, TX) Evaluating the safety and tolerability of low-dose sirolimus (rapamycin) to improve overall hematopoiesis and delay or prevent hematologic malignancy development.
Stem Cell Harvesting & Banking Clinical Trial (Locations: MD Anderson Cancer Center and Children’s Hospital of Philadelphia - Philadelphia, PA). Assessing the feasibility of mobilizing, collecting, and cryopreserving autologous CD34+ cells in RUNX1-FPD patients for potential future gene correction strategies.
NIH RUNX1-FPD Clinical Research Study (Location: NIH - Bethesda, Maryland)
A natural history study, investigating genomic and clinical factors that contribute to variable cancer penetrance in RUNX1-FPD individuals through prospective longitudinal follow-up and biospecimen collection.
Imatinib Phase 1b Trial (Location: NIH - Bethesda, Maryland) Determining the safety and optimal dosing of imatinib in patients with RUNX1-FPD to evaluate its potential in restoring RUNX1 protein activity.
You can find patient-friendly, detailed information, eligibility criteria, and downloadable summaries for each of these here, including links to their clinicaltrials.gov entries: | |
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Stay current with the latest peer-reviewed studies in RUNX1-FPD science and clinical research!
The RUNX1 Radar tracks and shares newly published findings from across the field, including work from RRP-funded investigators and leading researchers worldwide.
Explore recent research articles in Science Translational Medicine, Blood Advances, The Journal of Clinical Investigation, Genes & Development, and more. |
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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. |
Thanks for following our progress and being a part of the RRP community! Visit www.runx1-fpd.org to learn more and keep up-to-date between newsletters. |
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