On Monday, November 24th, we’ll launch our 2025 Annual Awareness and Fundraising Campaign, inviting our community to help us continue exploring new frontiers in improving the lives of individuals and families affected by RUNX1-FPD.
From new clinical trials and expanded patient resources to growing collaboration among researchers and clinicians, together we’ve made important strides in advancing research and improving care for RUNX1-FPD families. But our work is far from over.
100% of every contribution, no matter the size, helps accelerate progress toward blood cancer prevention and the development of better treatment options.
Stay tuned in the coming weeks for ways to get involved, spread the word, and help us start the next chapter of this journey.
Together, we’re navigating new frontiers, one act of generosity at a time.
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:
RUNX1 Clinical Trials Spotlight: Laying Foundations For Improved Health
Low-Dose Sirolimus Clinical Trial
Led by Dr. Courtney DiNardo at MD Anderson Cancer Center, this study is exploring whether a small daily amount of sirolimus can help improve blood health in individuals with RUNX1-FPD. Specifically, the doctors running the study are watching to see whether sirolimus can improve RUNX1-FPD platelet numbers and function, and whether it can slow the growth of precancerous cells in the bone marrow.
Participants are closely monitored to assess both safety and potential benefits. Findings from the initial patients taking sirolimus are encouraging and may offer valuable information about how to prevent blood cancer.
Stem Cell Harvesting and Banking Clinical Trial
Sponsored by RRP and happening at both MD Anderson Cancer Center and the Children’s Hospital of Philadelphia, this study is examining the safety and feasibility of collecting and banking blood stem cells from individuals with RUNX1-FPD.
The goal is to preserve these cells while they are “younger” for potential future use, should gene-editing technologies improve to the point where RUNX1 variants can be corrected. A participating patient’s own cells could then be edited to correct their RUNX1 variant and returned to their body, resulting in a blood system that functions more like a person without RUNX1-FPD.
Because human blood stem cells can self-replicate, collecting them is considered safe in healthy volunteers. In fact, the study follows the same procedures used to collect bone marrow from donors who are donating their blood stem cells to those who need them, such as a family member with a blood cancer.
Individuals interested in learning more about these or other ongoing RUNX1 clinical trials can visit our website for more information:
In our August newsletter, we shared how brothers Ethan and Logan, grandsons of longtime RRP community members Georgie and Chuck Blackburn, were making headlines on the cross-country course. We’re thrilled to share that their team has done it again!
Earlier this month, the University High School Hawks captured the Class AAAA state cross-country title for the seventh consecutive year, with both Ethan and Logan playing key roles in the victory.
Ethan, a senior, placed second overall and was recognized as the top senior in West Virginia. Logan, a junior, came in seventh overall in a highly competitive field of 104 runners.
Their dedication, teamwork, and determination continue to show that living with RUNX1-FPD doesn’t have to hold anyone back from pursuing their passions.
If you or your loved ones have good news to share, whether it’s an academic achievement, a personal milestone, or anything that makes you proud, we’d love to hear from you. Please email Catherine Pelton at cpelton@runx1-fpd.org so we can celebrate more moments like this with everyone in future newsletters.
Helping Your Family Understand a RUNX1-FPD Diagnosis
Understanding RUNX1-FPD and the value of testing for it can be truly empowering, as it gives individuals the chance to take charge of their health and work with their doctors to make proactive choices.
If you have relatives who haven’t yet had genetic testing or learned much about RUNX1, please refer them to RRP. By connecting them to us, your loved ones can learn what testing involves, connect with doctors in their area who are familiar with RUNX1-FPD, and find information and tools to guide their next steps.
If you are unsure how to start this conversation with your family members, you can reach out to Dr. Amanda Eggen. She can offer suggestions and even introduce you to the ConnectMyVariant team, who have expertise in counseling families with this challenge.
We’re here to help families avoid long delays in understanding their health, prepare for the future, and feel confident in managing their wellbeing. Whenever they’re ready to take the next step, they can connect with Amanda at aeggen@runx1-fpd.org or (805) 229-1393.
Register Today For The November Coffee Chat!
Please join us on Thursday, November 6th, for the last RUNX1 Patient & Family Coffee Chat of this year!
Come and connect with fellow patients and family members by sharing how you learned about RUNX1-FPD, or join to listen and learn from others. Click the button below to register:
Please note that you can join with your camera on to make it feel as close to a relaxed conversation as possible. Or if you prefer, you can simply call in by phone and listen.
We’re excited to invite RUNX1-FPD patients and their families to join us in Atlanta next spring for the 2026 RUNX1 Patient Meeting. This in-person event is a special opportunity to learn, connect, and share experiences with others who truly understand the RUNX1-FPD journey.
There is no registration fee, and RRP covers travel, lodging, and meals for individuals with RUNX1-FPD, children traveling with them, and one close family member.
If you’re a parent or guardian of a RUNX1-FPD child, we’d love your input! Please take a moment to register and complete the “Children’s Programming Interest” section of the form by Tuesday, November 11th, to assist us in deciding whether to offer children’s sessions.
Here’s what we’re considering:
Ages 4–7: Gentle, play-based sessions led by a facilitator, focused on comfort in medical settings and easing anxiety about doctor visits (no heavy disease talk).
Ages 8–12: Hands-on, creative learning activities designed to help kids feel connected and included.
Ages 13–17: Small, discussion-based sessions where teens can ask questions, learn basic science, and openly discuss experiences or emotions.
If we don’t have enough children in similar age ranges, families are still encouraged to attend, and children can join the main sessions alongside parents and caregivers.
Even if you don’t have children attending, early registration helps us plan the best possible experience for everyone.
Whether you are newly diagnosed, caring for a loved one, or looking to reconnect with the RUNX1-FPD community, we hope to see you there!
Join the RUNX1 and Inherited Hematologic Malignancies Consortium
The RUNX1 and Inherited Hematologic Malignancies Consortium unites healthcare professionals with a shared mission: to improve care and outcomes for individuals with inherited blood cancer predisposition syndromes.
Members collaborate through monthly meetings, focused working groups, and shared tools to develop research partnerships and create clinical consensus guidelines.
Whether you are a hematologist, genetic counselor, or researcher, your expertise can help shape the future of patient care. See current members and join today: runx1-fpd.org/runx1-clinicians
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 reach out to Catherine Pelton at cpelton@runx1-fpd.org for a registration promo code.
2025 Hereditary Hematologic Malignancies: Not That Rare Webinar
On Wednesday, October 1st, Dr. Kelly Bolton (Washington University School of Medicine) presented information on how germline genetic variants influence the development of blood cancers as part of our Hereditary Hematologic Malignancies: Not That Rare medical education series.
Dr. Bolton discussed the role of clonal hematopoiesis (CH) as the bridge between inherited and acquired mutations, highlighted the importance of germline testing, and outlined emerging strategies for early intervention. She was joined by Dr. Sravanti Rangaraju (University of Birmingham) and genetic counselor Kelcy Smith-Simmer (University of Wisconsin) for an engaging audience Q&A session.
Researchers are laying the groundwork for a future where a person with RUNX1-FPD could benefit from their own corrected stem cells.
RRP is sponsoring this important clinical trial at two locations, the Children’s Hospital of Philadelphia (CHOP) and MD Anderson Cancer Center (MDACC), to evaluate the safety of collecting and storing blood stem cells from individuals with RUNX1-FPD for potential future use, should stem cell correction technologies advance.
In this webinar, Dr. Olson, the lead investigator at the CHOP site, shared:
Why the teams are studying the safety of stem cell collection and storage.
Why each participant’s stored cells are intended only for their own possible future clinical use.
How future advances in gene editing might help correct RUNX1 variants in these cells.
Who may be eligible and what participation involves.
Click the link below to watch the recording and learn more about what this opportunity could mean for individuals with RUNX1-FPD.
On Thursday, October 23, the UK Cancer Genetics Group (UKCGG) held a webinar for RUNX1-FPD patients and families in the United Kingdom to discuss how RUNX1-FPD is diagnosed and managed within the UK’s National Health Service (NHS).
The session brought together leading clinicians to explain care pathways, testing options, and reproductive planning resources available to UK patients and families.
The panel discussed diagnostic and predictive testing, hematology care coordination, and individualized surveillance approaches. They also reviewed reproductive options approved for RUNX1-FPD in the UK in 2023 and shared new patient information leaflets developed for RUNX1 patients.
None of the news you just read is possible without support from our generous donors. 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!
