April 2025

The 2025 X-Out Blood Cancer Campaign Kicks Off Tomorrow, May 1st!

Our 2025 X-Out Blood Cancer Campaign launches May 1st, and this year, our goal is to raise $50,000 to support RUNX1-FPD patients participating in the first-ever RUNX1-focused clinical trials. 

These groundbreaking studies bring us closer to real treatments for all patients, but for many families, the biggest barrier isn’t qualifying—it’s getting there.

That’s why we’re raising funds to help with travel, lodging, and other out-of-pocket costs for patients who would otherwise be unable to participate. These clinical trials could benefit everyone in our community, and we do not want people to miss an opportunity to take part because of finances. 

Please consider making a donation today and sharing these links with your family and friends: GoFundMe and Givebutter. No matter the size, every gift makes a difference and shows your support!

Coming Soon: RUNX1 Patient Tissue Bank Launching in June!

We’re excited to announce that the RUNX1 Patient Tissue Bank, a new opportunity to support research on RUNX1-FPD, will be launching at the beginning of June 2025!

This program, led by an RRP-funded team at the University of Pennsylvania (UPenn), will invite individuals with RUNX1-FPD to donate small amounts of blood or bone marrow aspirate during routine medical appointments in their hometown or city.

Samples will be securely stored and used by approved researchers to explore new methods for early cancer detection, improve quality of life, and work toward cancer prevention for those affected by RUNX1-FPD.

Participation will be simple:

• Set up an account in the online RUNX1 Research Portal.
• Provide your mailing address and the date of an upcoming scheduled clinical blood draw or bone marrow procedure.
• Receive a study team-provided sample collection kit in the mail, and bring it to your appointment.
• After your appointment, simply drop the sample kit off at your local FedEx using the prepaid packaging provided!

All samples will be de-identified to protect your privacy, and participation is completely voluntary.

Patients: Find out how you can contribute to advancing RUNX1-FPD research at our upcoming webinar:

Introduction to the RUNX1 Patient Tissue Bank Webinar

Tuesday, June 3rd
2:00 p.m. PDT  •  5:00 p.m. EDT  •  10:00 p.m. BST

We invite you to join us and the leaders of the study, Dr. Ximena Jordan Bruno and Dr. Martin Carroll from UPenn, for a special webinar to share more about the RUNX1 Patient Tissue Bank and how to get involved once it is ready for enrollment

You’ll have the chance to hear directly from the investigators, ask questions, and learn how your participation could help accelerate research and future treatments.

Together, we can build a resource that brings new hope for the RUNX1-FPD community!

Sirolimus Clinical Study Update: Investigator & Patient Perspectives

In a recent webinar, Dr. Courtney DiNardo (Primary Investigator - MD Anderson Cancer Center) shared updates on the Low-Dose Sirolimus Pilot Study for RUNX1-FPD patients, followed by a live Q&A with three individuals currently taking sirolimus.

Key Highlights:

• Dr. DiNardo explained the scientific rationale for the study: individuals with RUNX1-FPD show increased inflammatory signaling in their bone marrow, which may contribute to cancer risk. Sirolimus, an mTOR inhibitor, could potentially reduce this inflammation and lower long-term leukemia risk.
  
  
• Early findings from the first two pilot participants suggest that sirolimus may improve platelet function and stabilize bone marrow health, with some patients also reporting improvements in joint pain, allergy symptoms, and overall quality of life.
  
  
• Side effects reported were generally manageable, with some participants experiencing mild nausea, gastrointestinal symptoms, or headaches, particularly early in treatment.
  
  
• The study is still actively enrolling participants. Travel support and assistance with out-of-pocket costs are available through RRP.

In the second half of the session, three patients shared their personal experiences with sirolimus.

Each emphasized the manageable side effects and the importance of participating in research that could benefit the RUNX1-FPD community.

Several noted unexpected quality-of-life improvements and said they would recommend trial participation to others considering it.

The session closed with a discussion about ongoing efforts to reduce financial barriers for patients interested in joining the study. You can view either part of the webinar below:

Registration Now Open: 2025 Virtual RUNX1 Conference

Join us virtually this September as we explore “Navigating New Frontiers” in RUNX1-FPD science, innovation, and patient-centered care. More details to come on topics and presenters for both events. We’re excited to see you all again online this year!

Scientific Conference (Researcher & Clinician Focused)

Tuesday, September 9th: 8:30 a.m - 2:00 p.m. PDT

Wednesday, September 10th: 9:00 a.m. - 3:00 p.m. PDT

This scientific-focused event features research updates from leading RUNX1-FPD experts, including RRP grantees and other distinguished researchers in the field. Both days will include a poster session open to graduate students and postdocs—stay tuned for application details!

For questions about the Scientific Conference or if you are interested in presenting, please contact Dr. Katrin Ericson, RRP’s Executive Director.

Patient Meeting (Patient & Family Focused)

Friday, September 19th: 9:00 a.m. PT - 2:00 p.m. PT

This patient-focused series of discussions will include a translation of key takeaways from this year’s Scientific Conference into accessible insights. This event will also feature expert presentations tailored to patient interests, covering research updates, clinical trials, and strategies for managing RUNX1 patients’ and families’ overall well-being.

For questions or suggestions regarding the Patient Meeting, please contact Dr. Amanda Eggen, RRP’s Director of Patient Engagement and Clinical Programs.

We’re excited to spotlight Dr. Kristy Stengel, an exceptional researcher and advocate for the RUNX1-FPD community, in this month’s newsletter. 

Dr. Stengel serves as an Assistant Professor in the Department of Cell Biology at Albert Einstein College of Medicine and is a member of the Stem Cell & Cancer Biology Program at the NCI-designated Montefiore Einstein Comprehensive Cancer Center.

In 2022, Dr. Stengel received an Early Career Investigator Grant through the RUNX1 Research Program in partnership with Alex’s Lemonade Stand Foundation. That early-stage support laid the foundation for her most recent achievement: a five-year, $2.6 million R01 grant from the National Heart, Lung, and Blood Institute (NHLBI, part of NIH).

This new grant will enable her team to explore how RUNX1 regulates blood cell development—and what goes wrong when RUNX1 is mutated, as it is in individuals with RUNX1-FPD.

We were thrilled to host Dr. Stengel at a recent RRP webinar, where she generously shared her team’s latest research findings.

We’re honored to have supported her early work and look forward to following her continued success.

Why AML Awareness Matters for RUNX1-FPD Families:

Prevention, Support, and Hope

AML World Awareness Day may have passed, but its message this year—highlighting the need for greater education and support around the long-term physical and psychological effects of treatment for acute myeloid leukemia (AML)—still matters. Especially for individuals and families living with RUNX1-FPD.

We're always looking for relevant RUNX1 content to add to the blog. Would you like to contribute? If you’re interested in being a guest author, please contact Catherine Pelton at cpelton@runx1-fpd.org.

Join Us For Our Next RUNX1 Patient & Family Coffee Chat!

Thursday, May 8th
9:00 a.m. PDT  •  11:00 a.m. CDT  •  5:00 p.m. BST

Join us for the next RRP Patient & Family Coffee Chat! These informal gatherings are a great opportunity to connect with other patients and family members in the community.

Whether you're newly diagnosed or have been part of the community for years, your voice matters. Bring a cup of coffee, your questions, or simply yourself—everyone is welcome.

After you register, Amanda Eggen will follow up with event details. Questions? Reach out to Amanda at aeggen@runx1-fpd.org.

Help Advance RUNX1-FPD Research Progress Through the RUNX1 Patient Data Hub

Eligible Participants Receive a $50 Gift Card

We’re continuing to enroll participants in the RUNX1 Patient Data Hub (RUNX1 PDH), a secure platform where individuals and families affected by RUNX1-FPD can share their health experiences to help drive research, improve care, and support the community.

By contributing your information, you’re helping researchers and clinicians better understand the complexities of RUNX1-FPD. Each participant brings us closer to answering critical questions about symptoms, treatments, trends, and long-term outcomes.

In recognition of your time and contribution, we are pleased to offer a $50 gift card to all patients who qualify and complete the required surveys.

If you haven’t joined yet, now is the perfect time to get involved and help build a stronger future for yourself, your family, and the entire RUNX1-FPD community.

Have questions? Reach out to Amanda Eggen at aeggen@runx1-fpd.org.

Please Join Our Upcoming RGPC Meeting: Understanding how RUNX1 mutations drive inflammation by analyzing single cells from patient samples

Monday, May 19th
9:00 a.m. PDT  •  12:00 p.m. EDT  •  5:00 p.m. BST

Our next Research Guided by Patients Committee (RGPC) meeting is coming up and we hope you’ll join us!

This month’s session will feature an update from the CZI-funded research team, who are investigating how RUNX1 mutations may drive inflammation by analyzing single cells from patient samples. The team will share early insights from this exciting study and answer questions from attendees. 

The RGPC brings together patients, families, caregivers, researchers, and clinicians to ensure the lived experience of those affected by RUNX1-FPD helps shape research, education, advocacy, and awareness efforts.

Members of the CZI-funded research team.

New members are always welcome, and patients and family members are especially encouraged to attend. Want to learn more or get involved? Contact Amanda Eggen at aeggen@runx1-fpd.org.

Attention: Parents of Children with RUNX1-FPD

Many parents in our community are asking the same kinds of questions:

• When is the right time to talk to my child about RUNX1-FPD?
• Is it safe for them to play contact sports?
• How do I support my child during blood draws or bone marrow biopsies?

We know how overwhelming this can feel, but you’re not alone. Other parents are looking to connect with those who truly understand.

We’re exploring a small parents group to help families share experiences, ask questions, and support one another. If you're interested, please take a moment to complete this short survey:

We’ll begin reaching out to connect parents within the next month. You don’t have to navigate this alone—we’re here to help!

"Hematopoietic Stem Cell Division is Governed by Distinct RUNX1 Binding Partners”

Tuesday, May 6th
11:00 a.m. PDT  •  2:00 p.m. EDT  •  7:00 p.m. BST

Join RRP Scientific Advisory Board member, Dr. Leonard Zon as he presents his team’s latest discoveries on modulating RUNX1 to enhance hematopoietic stem cell (HSC) expansion.

Dr. Zon’s research identifies a novel mechanism by which the RUNX1 inhibitor Ro5-3335 promotes HSC self-renewal and enhances clonal diversity by interacting with ELF1, revealing potential therapeutic strategies for improving blood system health.

“Transcription Factor Associations Fine-Tune Expression of the RUNX1 Gene Network to Confer Regulatory Logic to Hematopoietic Gene Expression Programs”

RRP grantee Dr. Kristy Stengel recently presented her team’s latest research findings on lineage-specific transcription factors and their role in hematopoietic gene regulation.

Dr. Stengel and her team used acute depletion of RUNX1 combined with nascent transcriptomics to define RUNX1-regulated gene targets and identify the enhancers responsible for this regulation, offering new insights into how transcription factors fine-tune blood cell development. Her presentation shed light on the molecular mechanisms behind RUNX1-FPD and how these discoveries may inform future approaches to disease prevention and treatment.

Click below to watch the full webinar:

"Hereditary Hematologic Malignancies: Not That Rare”

In our latest medical education webinar, Dr. Kelly Bolton (Washington University School of Medicine) broke down how inherited genetic mutations influence blood cancer risk, and why germline testing should be routine in hematologic care.

Dr. Bolton shared findings from large-scale population studies, discussed how germline and somatic mutations interact, and explored emerging opportunities for early intervention in high-risk individuals. The session also features insights from Dr. Gina Keefer and Dr. Abhishek Mangaonkar during an expert Q&A panel discussion.

Click below to watch the full webinar:

Please support blood cancer prevention research and help find treatments for RUNX1-FPD patients. RRP commits 100% of direct donations to fund research and educational programs.

Thank you!

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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