RRP NEWSLETTER

2024 Newsletters

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The Latest Edition: December 2024

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December 2024

As we reflect on the progress made in 2024, we’re filled with gratitude for this community's unwavering support.

Your generosity and engagement drive groundbreaking research and programs that benefit RUNX1-FPD patients and families.

With this incredible year coming to a close, we invite you to join our Building Bridges, Breaking Barriers campaign to continue helping us support blood cancer prevention and intervention research.

Together, we’re building a future of hope and better outcomes for individuals with RUNX1-FPD and their families.

MAKE A DONATION TODAY

Thank you for your continued partnership. From all of us at RRP, we wish you and your loved ones a safe and joyful holiday season!

Clinical Trial Updates: Stem Cell Harvesting & Banking and Sirolimus

We’re excited to share updates on two important clinical trials supported by RRP:

Stem Cell Harvesting & Banking Clinical Trial: This study is now available in TWO locations: Houston, Texas, and Philadelphia, Pennsylvania. Click the button below to learn more:

STEM CELL HARVESTING & BANKING SUMMARY

Sirolimus Pilot Study: Recent protocol updates have added telemedicine options for those who live outside of Houston and are interested in enrolling. Several tests can now be performed with your home healthcare team and submitted to the study team. Click the button below for more information:

SIROLIMUS PILOT STUDY SUMMARY

In order to make both of these studies as accessible as possible, RRP is also offering assistance for U.S. domestic travel, lodging, and food for eligible patients. If you are interested or have questions about support options, please contact Amanda Eggen at aeggen@runx1-fpd.org.

For detailed information about these and other patient research study opportunities, click here to visit our website.

Webinar: RUNX1 Patient Data Hub: Getting Started on Matrix

Wednesday, January 15th

12 p.m. PST | 2 p.m. CST

3 p.m. EST

We’re excited to introduce the newly relaunched RUNX1 Patient Data Hub (RUNX1 PDH) on the Matrix platform! Whether you’re curious about the RUNX1 PDH, ready to set up your account, or transitioning from REDCap, this session is for you. Register today to learn more:

CLICK HERE TO REGISTER

What We’ll Discuss:

For New Users:
- Discover the goals, value, and key features of the RUNX1 PDH.
- Learn how to set up your account and get started.
For Existing Users: If you were previously enrolled through REDCap, learn how to:
- Set up your account on the Matrix platform
- Provide updated consent
- Access additional features of the platform

Q&A Session:
After the presentation, we’ll hold a live Q&A. If you have specific questions about your information or need help setting up your account, Amanda will share a link to schedule a 1-on-1 Zoom meeting for personalized support.

Please join us and take the next step in contributing to this vital resource for the RUNX1-FPD community! You can find more information about the RUNX1 PDH on our website:

RUNX1 PDH INFORMATION

Webinar: Stem Cell Harvesting & Banking Clinical Trial at
Children's Hospital of Philadelphia

Wednesday, January 29th

2 p.m. PST | 4 p.m. CST

5 p.m. EST

Join Dr. Tim Olson from the Children's Hospital of Philadelphia (CHOP) to learn about the second location of this groundbreaking clinical trial sponsored by RRP.

Dr. Olson will discuss the trial’s goals, procedures, and eligibility requirements, and answer attendee questions (anonymously if preferred). Register here:

CLICK HERE TO REGISTER

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.

The study team believes that this procedure could benefit patients in the future if gene editing technology advances. With more sophisticated technology, the RUNX1 variants in these stored cells could potentially be corrected and reinfused back to improve patients' health using their own cells.

Please Note: This webinar is designed for our patient audience; however, anyone interested in learning more about this study is welcome to attend.

Join The First Research Guided By Patients Committee Meeting of 2025!

Tuesday, January 28th

10 a.m. PST | 12 p.m. CST

1 p.m. EST

The Research Guided by Patients Committee (RGPC) consists of patients, family members, caregivers, researchers, and clinicians focused on including diverse RUNX1-FPD patients’ perspectives and preferences with respect to research, education, advocacy, and awareness initiatives for the RUNX1-FPD community.

Patients and family members are always welcome to join RGPC meetings, and the group is always open to new members!

There’s no need to register—just click the link below to join the meeting directly on January 28th at the scheduled time:

RGPC MEETING LINK

If you have an interest in learning more about the RGPC and how to get involved, please contact Amanda Eggen.

Scientific Advisory Board Meeting Update

On November 8, 2024, the RRP Scientific Advisory Board (SAB) met to review current research progress and identify priorities for advancing RUNX1-FPD research. The meeting was held the day after the annual scientific conference.

After careful evaluation, the SAB ranked 24 research questions and objectives to guide RRP’s funding decisions between 2025-2027.

The top priorities focus on the most critical areas for understanding and preventing disease progression:

Investigating RUNX1/BCOR and RUNX1/TET2 mutations to understand how these common genetic changes contribute to the development of blood cancers. Such studies can help identify therapeutic targets to stop or slow disease progression.
Exploring whether clones – groups of blood cells with the same genetic mutation (e.g., RUNX1/BCOR) – are adaptive (helpful), maladaptive (harmful), or context-dependent, which could lead to more precise approaches for targeting problematic clones while preserving healthy ones.
Studying how certain clones resist inflammation, a key driver of disease progression. Understanding these mechanisms could help develop therapies to control clonal growth.
Analyzing the clonal architecture of bone marrow samples from patients to uncover how specific cells evolve and interact. In other words how mutated cells (clones) grow and behave in the bone marrow to understand which ones lead to disease and why, offering critical insights into disease monitoring and potential interventions.

The SAB emphasized the importance of focusing on these areas while continuing to support existing clinical studies.

By prioritizing these efforts, RRP aims to accelerate research that addresses the underlying biology of RUNX1-FPD and advances strategies to delay or prevent blood cancer development in affected patients.