| Explore the 2024 Impact Report |
We are proud to share the 2024 Impact Report, which highlights the progress made in blood cancer prevention research, patient engagement, and health equity initiatives last year, thanks to the support of the RUNX1-FPD community.
Report Highlights - Launch of two new clinical studies, including low-dose sirolimus and stem cell harvesting for future gene therapy.
-
Enrollment of 69 participants in the RUNX1 Patient Data Hub and the launch of a mobile-friendly platform.
- The first Regional Patient Meeting in Houston brought patients and clinicians together.
-
Expansion of Spanish-language clinician education through the En Beneficio de su Salud program.
-
Community-driven fundraising success, including $32,500 raised by a RUNX1-FPD patient.
- Launch of the LEAP Grant Program, co-funded with the American Cancer Society, committing $2.8 million to cancer prevention research.
This report is an excellent reminder of how far we have come and the momentum driving the next chapter of RUNX1 research.
|
|
|
2025 RUNX1 Virtual Scientific Conference & Patient Meeting |
|
|
September is a big month for our community, with two major annual events just around the corner. Don’t forget to register and join us on the following dates:
9th Annual Virtual RUNX1 Scientific Conference
September 9–10, 2025 • View Agenda
Designed for researchers and clinicians, this science-focused event will highlight the latest findings in RUNX1-FPD and other relevant research. |
Virtual RUNX1 Patient Meeting
September 19, 2025 • View Agenda Created for patients and families, this supportive event will feature personal stories, breakout sessions, wellness strategies, and updates on clinical trials. |
We hope to see you online in September! |
|
|
Hereditary Hematologic Malignancies: Not That Rare Wednesday, October 1st 12:30pm PDT • 3:30pm EDT • 8:30pm BST |
|
|
Join Dr. Kelly Bolton (Washington University) for an in-depth discussion on the role of genetic testing in hereditary hematologic malignancies (HHMs) and its impact on patient outcomes.
Dr. Bolton will review the prevalence of pathogenic germline variants, best practices for diagnosis and management, and how comprehensive genetic testing informs treatment decisions, including selecting appropriate stem cell donors for transplantation.
The session will conclude with an expert panel discussion featuring Dr. Sravanti Rangaraju (University of Alabama at Birmingham) and Kelcy Smith-Simmer, MMSc, CGC (University of Wisconsin). |
|
|
Stem Cell Harvesting & Banking Clinical Trial at CHOP Wednesday, October 22nd 12:00pm PDT • 3:00pm EDT • 8:00pm BST |
|
|
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! |
|
|
UK Cancer Genetics Group Webinar for UK-based RUNX1 Patients Thursday, October 23rd 8:00am PDT • 11:00am EDT • 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 is a unique opportunity for patients and families to connect, ask questions, and learn more about resources and guidelines tailored to their 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
- Ms. Beverley Speight – Cambridge University Hospitals NHS Foundation Trust
- Ms. Clair Engelbrecht – King's College Hospital NHS Foundation Trust
|
|
|
2026 RUNX1 Scientific Conference Wednesday, May 6 - Thursday, May 7, 2026
Emory Conference Center Hotel • Atlanta, Georgia |
Register today for the 2026 RUNX1 Scientific Conference, which will take place in-person for the first time in Atlanta, Georgia, at the beautiful Emory Conference Center Hotel. With the theme Catalyzing Change: The Next Chapter in RUNX1, the program will feature:
- Research presentations from leading scientists and clinicians.
- Collaborative breakout discussions.
- Poster sessions from graduate and postdoctoral researchers.
-
Attendees will gain early access to new findings and explore emerging directions in RUNX1-FPD research, prevention, and care.
We hope to see you in Atlanta next year! *If you are a current RRP grantee, please reach out for a registration promo code. |
|
|
2026 RUNX1 Patient Meeting
Thursday, May 7 - Friday, May 8, 2026 Emory Conference Center Hotel • Atlanta, Georgia |
We are excited to invite patients and family members to join us in Atlanta next spring for the 2026 RUNX1 Patient Meeting. This in-person gathering is designed to support, inform, and connect individuals with RUNX1-FPD and their families. There is no registration fee, and RRP will provide travel support and cover the cost of the hotel room. Schedule Highlights: Thursday, May 7 - Welcome session and workshops exploring the latest research and lived experiences.
-
Poster session with opportunities to learn from scientists or present your own personal RUNX1-FPD story.
-
Evening keynote dinner and announcement of the RUNX1 X-Cellence Award winners.
Friday, May 8 -
Patient-focused research and clinical trial updates.
- Peer support and small-group discussions.
- Wellness strategies for managing uncertainty.
- Sessions led by members of the patient community.
The meeting officially concludes Friday evening, though guests are welcome to extend their stay through Saturday morning. For those staying longer, we will provide suggestions for enjoying Atlanta with family and friends.
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. Please register today and indicate your interest in the form! Travel Support
RRP will cover flight costs and hotel accommodations for Thursday and/or Friday night for each patient and one accompanying family member attending the meeting. Additional details will be provided soon to help with your planning.
Whether you are newly diagnosed, caring for a loved one, or seeking connection with others who share your journey, we hope to see you there! |
|
|
Why Our Health Equity Grants Are More Important Than Ever Before By: Dr. Gwen Nichols |
For 40 years, I’ve poured my life into fighting cancer. Working alongside doctors, nurses, social workers, researchers—and of course patients and their loved ones—we’ve made incredible progress. But what keeps me up at night after all these years is the realization that, for too long, the health sector didn’t focus on the holes in that progress. As we collectively sprinted towards better treatments, we’ve left some patients behind. Here’s the ugly truth of cancer care: through no fault of their own, factors like a patient’s income, race, age, or even zip code can significantly impact their health outcomes. |
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 the articles below,
please contact Amanda Eggen, RRP’s Director of Patient Engagement & Clinical Programs. |
|
|
Patient Spotlight: Ethan and Logan |
|
|
This fall, brothers Ethan and Logan, grandsons of longtime RRP community members Georgie and Chuck Blackburn, are making headlines on the cross-country course, reminding us that RUNX1-FPD doesn’t have to hold anyone back from pursuing their passions.
Ethan, a high school senior, has placed in the top 10 at the Class AAA state meet for three consecutive years and is aiming for his fourth this fall. At last year’s state cross-country meet, he ran a 5K in 16:07.35, which is an incredible achievement. His coach notes that finishing in the top 10 for three consecutive years is a rare feat in program history. Logan, a junior, continues to shave seconds off his best times and is running stronger with each meet. At their first official race of the season, both showed remarkable grit and determination, with Logan finishing second and Ethan third. They are now preparing for a major invitational race at California State University. Their story is a powerful reminder that while RUNX1-FPD can bring unique challenges, it doesn’t have to stop anyone from achieving goals, competing at a high level, or enjoying life to the fullest. Please join us in wishing Ethan and Logan the best of luck this season! |
|
|
Join the RUNX1 Patient Data Hub and Receive a $50 Gift Card as a Thank You |
|
|
We are continuing to enroll participants in the RUNX1 Patient Data Hub (RUNX1 PDH), a secure platform where patients share their health experiences to help drive research, improve care, and support the RUNX1-FPD community. By contributing your experiences, you help researchers and clinicians better understand this rare disorder and pave the way for new discoveries in care and prevention.
As a thank you for your time and participation, eligible patients who complete the required surveys will receive a $50 gift card.
If you have not enrolled yet, now is a great time to join and help build a stronger future for yourself, your family, and the entire RUNX1-FPD community. |
|
|
RUNX1 Patient Research Opportunities Spotlight: NIH Studies on RUNX1-FPD |
Two National Institutes of Health (NIH) studies focused on RUNX1-FPD are currently enrolling. If you are eligible, you may be able to participate in both during the same visit, making the most of your time at the NIH. -
RUNX1-FPD Clinical Research Study (NIH Natural History Study)
This long-term observational study follows participants over time to better understand why some people with RUNX1-FPD develop blood cancer while others do not.
- Imatinib Clinical Trial
This trial is testing the safety and tolerability of imatinib in RUNX1-FPD. Researchers hope to determine whether the drug can safely increase RUNX1 protein levels and support healthy blood cell development.
Why participate? - Care from leading experts: The NIH team has evaluated more individuals with RUNX1-FPD than any other group worldwide. Participants receive thorough evaluations based on the latest scientific knowledge and clinical experience.
- Direct impact on future care: Each participant helps build critical knowledge that may lead to prevention strategies and future treatment options for RUNX1-FPD.
Who may be eligible? Eligibility depends on your health history and interest. Some patients may qualify for one study, while others may be able to join both.
Interested in learning more? Contact Amanda Eggen for details on qualifications and to be introduced to the NIH study team. |
|
|
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: |
RRP–ALSF Funded Research: Dr. Lucio Castilla |
|
|
|
In 2019, Lucio Castilla, Ph.D. (UMass Chan Medical School) was awarded the RRP–ALSF Familial RUNX1 Research Grant to investigate how RUNX1 mutations drive disease risk. Dr. Castilla’s group developed mouse models carrying the same RUNX1 mutations seen in patients, providing essential tools to study how these genetic changes alter blood stem cell behavior. Their work revealed that:
Germline RUNX1 mutations induce inflammation in hematopoietic stem and progenitor cells. |
|
|
RUNX1 mutations may impair DNA damage repair mechanisms, leaving cells more vulnerable to stress and the acquisition of secondary cancer-associated mutations.
Over time, these effects lead to the overgrowth of the myeloid compartment in the blood and increased predisposition to hematologic malignancies.
The study, published in Blood Advances, suggests that anti-inflammatory strategies may help reduce clonal expansion and cancer risk in RUNX1-FPD. Read the publication here: Runx1-R188Q germ line mutation induces inflammation and predisposition to hematologic malignancies in mice
|
|
|
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. |
Did someone forward you this newsletter? Sign up today to get your own copy delivered straight to your Inbox! |
|
|
|
