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Thanks to the incredible generosity of many RRP community members during the 2024 year-end Building Bridges, Breaking Barriers campaign, we raised a total of $39,213.60.
And with the support of an anonymous donor matching nearly all contributions, we achieved a combined total of $73,165.56. 100% of this will be used to fund vital research and patient support initiatives.
As we move into 2025, we’re excited to continue breaking barriers and supporting research focused on creating real-life solutions for RUNX1-FPD patients.
Thank you for being an essential part of our community! |
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Dr. Anupriya Agarwal, RRP-Edward P. Evans Foundation Focused Impact REsearch (FIRE) grantee, and her team are advancing our understanding of inflammation’s role in RUNX1-FPD and its connection to blood cancer risk. Dr. Agarwal and colleagues published their latest findings in a top tier medical journal, Science Translational Medicine. |
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Previous research has shown that inflammation is a major contributor to the development of blood cancer, and Dr. Agarwal’s research sheds light on specific pathways and targets that could lead to new treatments for RUNX1-FPD. Her team identified CD74, a protein found on the surface of blood cells, as being elevated in RUNX1-FPD. CD74 is closely linked to mTORC1 and plays a key role in driving inflammation, making it a promising therapeutic target and potential biomarker for blood cancer risk.
Even more excitingly, her findings have shown that existing drugs, such as sirolimus (an mTORC1 inhibitor), may effectively target these inflammatory pathways. This research directly led to the launch of the clinical trial testing sirolimus in RUNX1-FPD patients, a significant step forward for the community.
Dr. Agarwal’s work highlights how patient-centered research can lead to real-world clinical innovations.
Read the full blog to learn more about this important research and what it could mean for RUNX1-FPD patients. Click here to read the scientific paper. |
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Revealing the Genetic Underpinnings of Cytopenias: Navigating the Path to Genetic Testing Thursday, February 20th
9 a.m. PST • 11 a.m. CST • 12 p.m. EST |
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Join us for a presentation by medical geneticist Dr. Moon Ley Tung discussing the challenges and best practices in managing and diagnosing adult patients with inherited cytopenias. Dr. Tung will use unique patient cases to highlight the clues she and her colleagues identified that indicated the need for deeper analyses, including genetic testing.
Following Dr. Tung’s presentation, she will be joined by Dr. Sioban Keel of the University of Washington and Dr. Ilene Weitz of the University of Southern California for a Q&A discussion. Both hematologists, Dr. Keel is an expert in inherited bone marrow failure syndromes, and Dr. Weitz specializes in cytopenias and clotting disorders. All three panelists will offer their perspectives on the reviewed cases and answer audience questions.
Learning Objectives:
• Understand the prevalence of inherited cytopenias
• Learn how to suspect germline genetic conditions
• Gain tools on how to order diagnostics for cytopenias
• Discuss what to consider when caring for patients and their family members |
Introducing the RUNX1 X-Change: A New Space for Patient Conversations
Wednesday, March 12th
2:30 PM PDT • 4:30 PM CDT • 9:30 PM GMT |
Join Amanda Eggen and Katrin Ericson for a patient-only, informal discussion space where you can share your thoughts, feelings, and questions on hot topics.
These quarterly chats are a chance for open, reciprocal conversations—we’ll introduce a specific topic for the event, and the discussion will be a freeform exchange. We want to hear your perspectives, learn from your experiences, and better understand what’s on your mind.
Whether you have specific questions or want to hear from others, we invite you to be part of the conversation. Your voice helps shape the topics and ensures we address what’s most important to you. Stay tuned for the next topic and join us March 12th for an open, supportive discussion! |
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We Look Forward to Seeing You at the First Coffee Chat of 2025! Wednesday, February 19th 3 p.m. PST • 5 p.m. CST • 11 p.m. GMT |
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Join us for the first RUNX1 Patient & Family Coffee Chat of 2025 and connect with others in the RRP community. If you’ve joined RRP in the past year, this is a great opportunity to introduce yourself, meet others, and share your story (only if you feel comfortable).
For those who have been part of the community longer, we invite you to join as well! Your experiences as a RUNX1-FPD patient or caregiver can help support and guide our newest members.
All you need is a computer, tablet, or phone with a camera and microphone, plus an internet connection—coffee is optional! After you register, Amanda Eggen will send you more details before the event. Questions? Email Amanda at aeggen@runx1-fpd.org. We look forward to seeing you there! |
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Patient and Family Support for Those in the United Kingdom |
There is a growing group of United Kingdom-based individuals and their family members who have gathered to support one another utilizing a private WhatsApp group. If you are interested in joining this private group, please email Amanda Eggen at aeggen@runx1-fpd.org.
The group is also coordinating a virtual meetup on Zoom and plans to convene several times this year. If you're interested in joining the discussion, please click below to complete the Doodle Poll with your preferred dates and times.
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This clinical consortium consists of 105 physicians, genetic counselors and scientists from 43 different institutions across the U.S., Canada, South America and Britain, with particular expertise in inherited hematologic malignancies.
The major focal areas for the overarching consortium is to develop cross-institutional clinical research and clinical consensus guidelines. Cross-institutional collaboration and practice learning opportunities continue to arise through this dynamic group. Working groups and committees include: - RUNX1 Clinical Consensus Guidelines Steering Committee
- Clinical Research Steering Committee
- Genetic Counselor Working Group
- RUNX1 Clinical Database Team
- RUNX1 Tissue Bank Biorepository Team
The larger consortium meets once per month on Tuesdays, and each working group has its own separate meeting time. If you are interested in joining the consortium, click below to complete the form: |
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On January 15th, 2025, Amanda Eggen introduced the newly relaunched RUNX1 Patient Data Hub (RUNX1 PDH) built on the Matrix platform. The session was designed to guide both new and existing users through the platform's features and updates.
Key Highlights: - Explored the goals and value of the RUNX1 PDH
- Demonstrated valuable patient tools offered by the Matrix software platform.
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Provided step-by-step guidance on setting up accounts, providing consent, and utilizing patient tools.
If you missed the live event, you can still watch the recording by clicking the button below, or contacting Amanda at aeggen@runx1-fpd.org. |
On January 29th, 2025, Dr. Tim Olson from the Children’s Hospital of Philadelphia (CHOP) led an insightful webinar about the second location of this groundbreaking clinical trial sponsored by RRP. Key Highlights: -
Dr. Olson provided an overview of the trial’s aim: to evaluate the safety and feasibility of collecting and banking stem cells that could be used at a later date for patient care.
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He also discussed the potential future benefits of collecting and banking blood stem cells from RUNX1-FPD patients, particularly as gene editing technologies continue to advance.
- He shared study eligibility requirements and procedures, offering valuable details for anyone considering participation.
This trial, which also underway at MD Anderson Cancer Center in Houston, Texas, represents an exciting step forward in RUNX1-FPD research. Watch the full webinar to learn more about this innovative study: |
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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