X-Out Cancer Campaign 2024: Take Charge of Your Health & Make a Difference for RUNX1-FPD Patients in May! |
The campaign’s focus this year is “live healthy” and Kristen will share expert insights on the pivotal role of diet in managing cancer risk and the significance of addressing obesity. She'll also discuss the American Cancer Society's nutritional guidelines for cancer prevention and supporting survivorship.
Plus, find out how you can easily join our X-Out Cancer campaign in May to raise funds to support groundbreaking cancer prevention research. Help us turn hope into action by getting involved in this year’s campaign. There are several simple ways to participate: - Direct Donations: Each dollar furthers our research.
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Create a fundraising page on the X-Out Givebutter campaign page:
Share your story and connect with other community members as we come together to support RUNX1 research and cancer prevention. -
Email Campaign: Spread the word about the importance of our mission and get individuals in your network to support our work.
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Set up a Facebook fundraiser: Are you active on Facebook? Make an even bigger impact with a fundraising campaign on Facebook to extend your reach during the month of May.
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We’re excited to announce that John Crowley is the keynote speaker at this year’s Scientific Conference the evening of Day 1 (Wednesday, November 6th).
With a career spanning over two decades, John has been a pivotal figure in the biotech industry. He previously founded and led Amicus Therapeutics, a company dedicated to treating rare genetic diseases, from 2005 to 2022.
His journey into biotechnology began after his own children were diagnosed with Pompe disease in 1998, prompting him to leave Bristol-Myers Squibb and co-found Novazyme Pharmaceuticals, which significantly advanced treatment for the disease.
John’s relentless pursuit to find a treatment for his children’s rare disease inspired the film Extraordinary Measures, starring Harrison Ford and Brendan Fraser.
Please join us to hear John share his inspiring journey and speak about the power of innovation in biotechnology! This year’s keynote will be broadcast live via Zoom, so please stay tuned for the registration link to virtually join this not-to-be-missed event. If you are a scientist or clinician who would like to attend RRP’s Scientific Conference in Princeton, New Jersey, on November 6th & 7th in person, please click the button below to register. |
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Congratulations to RRP-ALSF RARE Grant Award Winner
Dr. Robert Bowman! |
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We are thrilled to announce that Dr. Robert Bowman, Assistant Professor of Cancer Biology at the University of Pennsylvania's Perelman School of Medicine, has been awarded the 2024 Research Accelerating RUNX1 Exploration (RARE) Grant.
Dr. Bowman and his team will use this grant to evaluate new medicines called Menin inhibitors that he and his colleagues hypothesize can specifically target cells with preleukemic BCOR mutations and prevent them from transforming into leukemia. |
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Get Your Imatinib Clinical Trial Questions Answered |
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Join us on Friday, May 17th to learn more about the groundbreaking Phase 1b clinical trial investigating the potential of the drug imatinib to increase RUNX1 protein levels in individuals with RUNX1-FPD.
Dr. Alan Cantor, Associate Investigator, and Dr. Lea Cunningham, Principal Investigator, will dive into the rationale behind this trial, discuss participant eligibility criteria, and answer general questions about engaging in a clinical trial. |
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If you’d like to learn more about the trial before attending the webinar, please click here for a one-page summary: |
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Register for the Next Patient & Family Coffee Chat in June!
Join us on Friday, June 7th to welcome and connect to our newest RUNX1-FPD patient & family community members for an informal discussion.
All you will need is a computer, tablet or phone equipped with a camera and microphone, as well as an internet connection. Click here or on the banner above to register. Hope to see you there! |
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Join the Next Research Guided by Patients Committee Meeting Thursday, May 30th 9 a.m. PT • 12 p.m. ET • 5 p.m. BST
Join us for an engaging presentation by Drs. Anupriya Agarwal, Alex Bick and Esther Obeng as they discuss their CZI-funded project, "Deciphering RUNX1-FPD at Single Cell Resolution."
During this session, they will outline the project's objectives, explain the methods used for accessing tissue samples, and share preliminary research findings. There will also be an opportunity for you to ask questions and engage directly with the researchers.
This event will be hosted in a Zoom meeting format, allowing you to join freely without prior registration. Simply click the button below on the day of the event to enter the meeting at any time: |
Save The Date: RUNX1-FPD Patient Regional Meeting Thursday, July 18th • 4:00 p.m. - 7:00 p.m.
Houston, Texas
RRP is thrilled to invite individuals with RUNX1-FPD and their close family members to an informative and supportive gathering focused on enhancing patient wellbeing and exploring new research opportunities, particularly those available at Texas institutions.
Event Details: - Location: Venue details to be announced
Agenda: -
Engaging Discussions: Topics will include patient wellbeing, advocacy and the latest research opportunities.
- Expert Insights: Local physicians and genetic counselors will be available to answer your RUNX1-related questions and provide guidance.
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Networking Opportunities: Connect with other families navigating similar experiences in a warm, supportive environment.
Special Offerings: -
Complimentary Meals: Refreshments and dinner will be provided on site.
- Travel Reimbursement: RRP will reimburse up to $300 toward travel expenses for the individual with RUNX1-FPD and one close family member.
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RRP RFA Announcement: Make An Impact On RUNX1-FPD Research |
RRP is excited to extend a unique opportunity to investigators passionate about advancing the understanding of RUNX1-FPD and its implications in blood cancer prevention.
We're inviting clinicians to submit proposals to share clinical and sequencing datasets for research-consented RUNX1-FPD individuals/families to be included in a newly-developed, ethics approved database called the Inherited Hematological Conditions Database (IHCSdb). Knowing that collecting and entering clinical and genomic patent data across multiple timepoints requires significant time and focused effort, RRP is providing financial support to allow clinicians and researchers to dedicate time toward this effort. There are three tiers of funding support designed to reflect the volume of information entered per patient medical record. Proposals are due by Tuesday, April 30, 2024, before 8:00 p.m. PT
Please click the button below to review the detailed application guidelines. For any questions, please contact Dr. Katrin Ericson. |
ISEH 2024 Society Award Winner: Ravi Majeti, M.D., Ph.D. |
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| We're proud to announce that RRP Scientific Advisory Board member, Dr. Ravi Majeti, has received the 2024 McCulloch & Till Award for his significant contributions to hematology and stem cell research, especially in acute myeloid leukemia (AML). For more details on his work and this award, click to read here. |
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Seminario Web de Educación Médica de RRP Lunes, 15 de Julio
2:00 p.m. PT • 4:00 p.m. CT • 5:00 p.m. ET "Neoplasias Hematológicas Hereditarias: No Tan Poco Frecuentes" |
We are thrilled to announce our first research webinar presented entirely in Spanish, facilitated by Dr. Cecilia Guillermo. Event Highlights: -
Presentation: Dr. Ximena Jordan-Bruno will deliver her insightful talk on "Neoplasias Hematológicas Hereditarias: No Tan Poco Frecuentes."
- Expert Panel Discussion: The presentation will be followed by a dynamic Q&A session with expert panelists Dr. María Noel Spangenberg and Dr. Elvira D. R. P. Velloso.
Please spread the word about this valuable opportunity to your Spanish-speaking colleagues and join us for an engaging learning experience!
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Objetivos de Aprendizaje: - Comprender el papel de las pruebas genéticas en los resultados de los pacientes con HM (HM es el acrónimo en inglés).
- Aprender la prevalencia de variantes germinales patógenas en pacientes con HM.
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Obtener herramientas sobre cómo sospechar y diagnosticar HM hereditario.
- Aprender cómo manejar a los pacientes con HM hereditario y a sus familiares en riesgo.
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| Learning Objectives: - Understand the role of genetic testing on patient outcomes for HM patients.
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Learn the prevalence of pathogenic germline variants in HM patients.
- Gain tools on how to suspect and diagnose hereditary HM.
- Learn how to manage hereditary HM patients and their at-risk family.
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Please support 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! |
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Did someone forward you this newsletter?
Sign up today to get your own copy delivered straight to your Inbox! |
2024 Scientific Conference Topics - Germline RUNX1 Mutations and Inflammation
- Clonal Hematopoiesis and Leukemic Progression
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Leveraging Insights from Patient Data and Human Cells
- Managing Predispositions/Precursor Conditions in the Clinic
Event Highlights -
Wednesday, November 6th: Conference kickoff in the afternoon followed by an inspiring Keynote Dinner.
- Thursday, November 7th: Day 2 of presentations, culminating in a Networking Dinner, Drinks & Poster Session.
Don't miss out! Please click the button below and register now to secure your spot:
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| From the moment Georgie Blackburn spoke to RRP co-founder Monica Babich and Dr. Amanda Eggen in 2019 after being blindsided by her son’s RUNX1-FPD diagnosis, she knew she wanted to get involved.
Five years later, Georgie is an active patient advocate and leader within the RUNX1 community, acting as chair of the Research Guided by Patients Committee (RGPC). She cherishes her role with RRP. “I have always been a cheerleader at heart. I love challenges. The driving force is the need for knowledge. It is empowering to be a member of RRP.”
Georgie and her husband, Charles Blackburn, are also longtime donors to RRP and exemplify the spirit of philanthropy. They have always been charitable and feel “that their donation contributes dollars for humankind.” As Georgie notes, “[Giving] is a must. Make a difference in your lifetime. I truly believe that helping others is helping yourself." By being donors to RRP, the Blackburns are helping to support world-class research, with the goal of preventing cancer. As Georgie says, “Why not contribute if your own family is involved. The research RRP is conducting is so meaningful.”
RRP is grateful to Georgie and Charles Blackburn for their ongoing commitment to our mission and their quest to, in their words, “eradicate blood cancer caused by RUNX1-FPD.” |
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Register Today For Our Next RRP Research Webinar! |
Join Dr. Hannah Carter for a presentation on how she and her colleagues employed SEUSS, a Perturb-seq-like methodology, to generate and assay mutations at physical interfaces of the RUNX1 Runt domain. All registrants will receive a link to the recorded presentation. Click the button below to register and please share this webinar with your colleagues! |
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RRP’s Dr. Ericson To Join The Rare Cancers Prevention And Interception Workshop |
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We are excited to announce that Dr. Katrin Ericson, Executive Director of RRP, has been invited to present at the Rare Cancers Prevention and Interception Virtual Workshop scheduled for Tuesday, May 7th, and Wednesday, May 8th, 2024. Presentation details are as follows: |
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"Synergizing Science and Advocacy: Strategies to Optimize Collaboration and Improve Patient Engagement"
Date: Wednesday, May 8th Time: 10:40 AM - 11:00 AM ET
The two-day workshop features an array of excellent presentations from leading experts and advocates in rare cancers. This is a prime opportunity to engage at the forefront of rare cancer research and advocacy. Click here to review the agenda and register. This important workshop is sponsored by the Division of Cancer Prevention, Center for Cancer Research, Division of Cancer Epidemiology and Genetics at the National Cancer Institute (NCI), and the Congressionally Directed Medical Research Programs at the Department of Defense (DoD). |
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New Educational Opportunities Available In 2024
For Patients & Family Members |
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While we are not planning to hold an in-person Patient Meeting this year in Princeton alongside the Scientific Conference, we are excited to share a few updates that we believe will be more inclusive of our broader patient community. Our goal is to continue to maximize learning and support opportunities for our patient community: -
4 Webinars: One-hour virtual sessions covering important topics including healthy living for cancer prevention, NIH RUNX1 Natural History Study update, RUNX1 Patient Data Hub update, and key takeaways from the Scientific Conference sessions by Dr. Katrin Ericson and Dr. Nancy Speck.
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3 Virtual Support Sessions: Three 1-hour virtual discussions will be offered throughout the year, facilitated by a counselor who focuses on managing emotions and stress in the face of uncertainty.
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2 Regional Meetings*: Instead of one Patient Meeting in Princeton this year, we're planning two 3-hour meet-ups in locations in the southern and western United States like Houston, Texas, and San Francisco, California. This way patients farther from Princeton have a chance to participate. These sessions are designed to facilitate networking and discussions on care and wellbeing with local specialists.
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1 Annual Scientific Conference*: While we won’t have dedicated patient sessions at the conference this year, if patients are interested in hearing the technical research presentations at the Scientific Conference in Princeton, please email Amanda Eggen.
If you are interested in joining one of the regional events, please click here to let us know:
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*RRP is able to reimburse up to $300 in travel costs for each RUNX1-FPD patient and up to $300 for one close family member to accompany this individual to attend ONE of these events. |
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Imatinib Clinical Trial Questions Answered By The Experts |
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Come and learn more about the groundbreaking Phase 1b clinical trial investigating the potential of the drug imatinib to increase RUNX1 protein levels in individuals with RUNX1-FPD. Dr. Alan Cantor, Associate Investigator, and Dr. Lea Cunningham, Principal Investigator, will dive into the rationale behind this trial, discuss participant eligibility criteria, and answer general questions about engaging in a clinical trial. Whether you're considering participation or simply seeking to learn more, this webinar is an invaluable opportunity to engage directly with its leaders!
In an effort to accommodate different schedules and time zones, we are offering the webinar on two different dates: Friday, April 12th at 12 p.m. PT | 3 p.m. ET |
Friday, May 17th at 7 a.m. PT | 10 a.m. ET |
If you’d like to learn more about the trial before attending the webinar, please click here for a one-page summary: |
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Dr. Paul Liu’s group at the National Institutes of Health (NIH) recently had their manuscript published in the journal Blood Advances detailing the first genomic findings from the RUNX1-FPD Clinical Research Study. Key discoveries include:
- Diverse Genetic Landscape: The team identified 26 unique RUNX1 gene variants across 27 families, showing the wide array of genetic mutations that contribute to RUNX1-FPD. This diversity underscores the complexity of diagnosing and treating this disorder.
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Significant Mutations: Among 51 individuals without blood cancer, nearly half (49%) exhibited additional genetic variants, highlighting the complexity of RUNX1-FPD and potential new avenues for therapeutic research and intervention.
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BCOR emerged as the most frequently mutated gene (found in nine patients), with multiple BCOR mutations identified in four patients.
- Mutations in six other genes associated with clonal hematopoiesis of indeterminate potential (CHIP) or acute myeloid leukemia (AML) drivers (TET2, DNMT3A, KRAS, LRP1B, IDH1, and KMT2C) were also discovered in at least two patients or more.
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Three patients who are not related to each other (including one with myeloid malignancy) were found to have somatic mutations in NFE2, a gene important for erythroid and megakaryocytic differentiation.
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Evolving Genetic Mutations: The study's longitudinal approach revealed that genetic mutations evolved over time in 19 patients, showcasing the dynamic nature of RUNX1-FPD at the genetic level. This observation stresses the critical need for continuous monitoring to capture these evolutionary changes, which can significantly impact disease progression and the effectiveness of treatment strategies.
- Notably, older adult patients tended to have more stable clones, suggesting a relationship between the stability of genetic mutations and patient age. This detail adds an important dimension to understanding disease progression and could inform monitoring strategies and interventions based on patient age.
These findings not only advance our understanding of RUNX1-FPD, but also emphasize the critical role of early genetic screening and ongoing monitoring.
Identifying and tracking genetic mutations can inform proactive and preventive strategies, paving the way for personalized treatment options to manage RUNX1-FPD in the future.
Why Your Participation Matters By joining the NIH study, you can contribute to essential research that aims to improve our understanding and treatment of RUNX1-FPD. The greater the number of participants, the more significant insights researchers can gather about this condition.
Interested in contributing to this crucial research? Click here to learn more about the study and how to participate.
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RRP RFA Announcement: Make An Impact On RUNX1-FPD Research |
RRP is excited to extend a unique opportunity to investigators passionate about advancing the understanding of RUNX1-FPD and its implications in blood cancer prevention.
We're inviting clinicians to submit proposals to share clinical and sequencing datasets for research-consented RUNX1-FPD individuals/families to be included in a newly-developed, ethics approved database called the Inherited Hematological Conditions Database (IHCSdb). Knowing that collecting and entering clinical and genomic patent data across multiple timepoints requires significant time and focused effort, RRP is providing financial support to allow clinicians and researchers to dedicate time toward this effort. There are three tiers of funding support designed to reflect the volume of information entered per patient medical record. Proposals are due by Tuesday, April 30, 2024, before 8:00 p.m. PT
Please click the button below to review the detailed application guidelines. For any questions, please contact Dr. Katrin Ericson. |
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Seminario Web de Educación Médica de RRP Lunes, 15 de Julio 2:00 p.m. PT | 4:00 p.m. CT | 5:00 p.m. ET "Neoplasias Hematológicas Hereditarias: No Tan Poco Frecuentes" |
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We are thrilled to announce our first research webinar offered entirely in Spanish!
Dr. Ximena Jordan-Bruno will share her presentation "Neoplasias Hematológicas Hereditarias: No Tan Poco Frecuentes" and then will be joined by expert panelists for a Q&A session. Please share this webinar information widely with your Spanish-speaking colleagues! |
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Objetivos de Aprendizaje: - Comprender el papel de las pruebas genéticas en los resultados de los pacientes con HM. (HM es el acrónimo en inglés.)
- Aprender la prevalencia de variantes germinales patógenas en pacientes con HM.
- Obtener herramientas sobre cómo sospechar y diagnosticar HM hereditario.
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Aprender cómo manejar a los pacientes con HM hereditario y a sus familiares en riesgo.
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| Learning Objectives: - Understand the role of genetic testing on patient outcomes for HM patients.
- Learn the prevalence of pathogenic germline variants in HM patients.
- Gain tools on how to suspect and diagnose hereditary HM.
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Learn how to manage hereditary HM patients and their at-risk family.
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Please support 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! |
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Did someone forward you this newsletter?
Sign up today to get your own copy delivered straight to your Inbox! |
Please note: RRP's newsletter is best viewed in Light Mode versus Dark Mode on your device. |
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RRP's 8th Annual RUNX1 Scientific Conference |
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Attention RUNX1 Researchers and Clinicians: Please plan to join us in Princeton, New Jersey, on Wednesday, November 6th and Thursday, November 7th for our 8th Annual RUNX1 Scientific Conference. Anyone interested in advancing RUNX1-FPD research and improving patient outcomes are welcome to attend! Click the button below to pre-register, and we will be in touch soon with more information and a detailed agenda: |
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RRP is proud to announce that our organization’s story is once again showcased in the 2024 Giving List publication.
Click the banner above to read more and then please share our story with everyone you know to help us make an impact on our ultimate goal - to prevent cancer.
The Giving List began in 2020 as a special print book and digital platform for nonprofits to help drive new supporters through story driven narratives. Its creators and their highly philanthropic core investor group understood that story-driven marketing would create opportunities for the region’s nonprofit community to engage greater numbers of existing and new donors. |
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Imatinib Clinical Trial Is Now Open For Participation
The National Cancer Institute (NCI) at the National Institutes of Health (NIH) is seeking participants for a Phase 1b clinical trial studying the drug imatinib for individuals with RUNX1 Familial Platelet Disorder (RUNX1-FPD).
The study team believes that this medicine may be effective in increasing RUNX1 protein levels in individuals with RUNX1-FPD who have low RUNX1 protein levels. |
This phase of the trial is focused on determining the highest dose of the drug imatinib that can be tolerated with minimal side effects. Eligibility includes adults diagnosed with RUNX1-FPD and healthy volunteers.
For more detailed information, please click here to view a one-page summary of the study. |
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Join Us For The First Coffee Chat of 2024! |
No matter whether you're a newcomer or a familiar face, this is the perfect opportunity to meet and engage with fellow patients and family members. Come and share your journey or simply absorb the conversation - your presence is valuable and appreciated either way.
Register below and then join us on a device with internet access, a camera and a microphone (coffee is entirely optional!). |
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Join the Research Guided by Patients Committee (RGPC) First Meeting of 2024: Thursday, February 29th at 9:30 a.m. PT |
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The RGPC unites patients, caregivers, scientists and clinicians in order to combine their different perspectives on RRP's research support, patient education and awareness initiatives.
The meeting on February 29th will focus on outlining the committee's structure for the upcoming year, and identifying key areas where members can contribute to advancing RRP's mission.
We welcome new viewpoints to broaden our impact. If you're interested in learning more about the RGPC and exploring opportunities to contribute this year, please join us on the day of the meeting by clicking the button below:
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RGPC members participate in at least two quarterly, hour-long Zoom meetings each year and offer insights through email, phone or video calls. Please email Amanda Eggen, RRP’s Director of Patient Engagement and Clinical Programs, if you have any additional questions about membership. |
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NIH RUNX1-FPD Patient & Family Resources |
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The NIH RUNX1 Natural History study team has begun creating resources to support families through their challenges at home and to offer more accessible information about the disease.
These resources include communication and wellbeing guides, and a RUNX1 fact sheet. You can find the resources at the bottom of their study page by clicking here. |
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Seminario Web de Educación Médica de RRP Lunes, 15 de Julio 2:00 p.m. PT | 4:00 p.m. CT | 5:00 p.m. ET "Neoplasias Hematológicas Hereditarias: No Tan Poco Frecuentes" |
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| We are thrilled to announce our first research webinar offered entirely in Spanish! Dr. Ximena Jordan-Bruno will share her presentation "Neoplasias Hematológicas Hereditarias: No Tan Poco Frecuentes" and then will be joined by expert panelists for a Q&A session.
Please share this webinar information widely with your Spanish-speaking colleagues! |
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Objetivos de Aprendizaje: - Comprender el papel de las pruebas genéticas en los resultados de los pacientes con HM. (HM es el acrónimo en inglés.)
- Aprender la prevalencia de variantes germinales patógenas en pacientes con HM.
- Obtener herramientas sobre cómo sospechar y diagnosticar HM hereditario.
- Aprender cómo manejar a los pacientes con HM hereditario y a sus familiares en riesgo.
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| Learning Objectives: -
Understand the role of genetic testing on patient outcomes for HM patients.
- Learn the prevalence of pathogenic germline variants in HM patients.
- Gain tools on how to suspect and diagnose hereditary HM.
- Learn how to manage hereditary HM patients and their at-risk family.
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Submit your RUNX1 research projects! The Vera and Joseph Dresner Foundation recently launched a funding initiative aiming to support cutting-edge research and related programs in Myelodysplastic Syndromes (MDS) to advance future standards of care and seek a cure.
The initiative will award three grants: two Early Career Awards providing up to $125,000 per year for two years, and one Established Investigator Award providing up to $250,000 per year for two years.
Click here for more information.
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We deeply appreciate every contribution from our patient community, particularly those who share their experiences through video. These stories are vital in helping us raise awareness about RUNX1-FPD and continue driving our mission forward.
We invite you to explore some of these inspiring stories by clicking on each of the pictures below: |
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If you're interested in sharing your own story, please reach out to RRP's Communication Manager, Catherine Pelton.
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Please support 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! |
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Did someone forward you this newsletter?
Sign up today to get your own copy delivered straight to your Inbox! |
New Monthly RRP Newsletter Schedule
In order to keep you more promptly informed of the latest RRP and RUNX1-related news and developments, we're shifting our newsletter from a bimonthly to a monthly publication.
Look out for the latest edition landing in your Inbox around mid-month, every month! If you have anything you’d like to share in our newsletter, please email Catherine Pelton, RRP’s Communication Manager.
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From significant research publications to impactful community engagements, the dedication and collaborative spirit of our community members last year continued to help us make groundbreaking progress.
Here are some of the highlights that made 2023 a year to remember: -
Our scientific community published nine pivotal RUNX1 research articles, including the first article from the NIH’s RUNX1 Clinical Research Study. You can find them all here: RUNX1 Radar Report.
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Partnered with scientists and healthcare providers to support the launch of the first two RUNX1-focused clinical trials.
- Celebrated the first anniversary of the RUNX1 Patient Data Hub, with 69 patients enrolled and 47 contributing valuable health data.
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Successfully conducted a series of educational webinars (engaging over 660 healthcare professionals), including:
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Dr. Ximena Jordan Bruno was awarded the inaugural RRP Early Career Investigator – Clinical Research Grant for her proposed RUNX1-FPD biorepository project.
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Received a Health Equity in RARE Impact Grant from Global Genes for our En Beneficio de su Salud (For the Benefit of Your Health) program designed to aid Spanish-speaking RUNX1-FPD individuals.
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Raised over $20,000 through the 2023 X-Out Cancer Challenge to support cancer prevention and RUNX1-FPD research.
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Hosted a virtual workshop with community members so they could learn more about RRP’s long-term research strategy and provide input to help ensure our community is moving in the right direction to support RUNX1-FPD patients and families.
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Awarded two RRP-ALSF RUNX1 Early Career Investigator Grants, supporting the promising research of Dirk Loeffler, PhD, and the launch of a coordinated plan to develop clinical consensus guidelines for RUNX1-FPD with leadership by Brittany Stewart, CGC, PhD, and Hetty Carraway, MD.
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Successfully conducted our 7th Annual RUNX1 Scientific Conference and 4th Annual Patient Meeting with more than 230 total attendees.
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Held a special event for our donor community, Fast Track To A Cure: Employing a Business Mindset to Accelerate Cancer Prevention Research, featuring a fireside chat with RRP co-founder Tim Babich and RRP Executive Director Dr. Katrin Ericson.
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Over $33,000 Raised for RUNX1 Research!
We're excited to announce that our 2023 From Labs to Lives year-end campaign was a success, raising over $33,000 for RUNX1 research and patient initiatives. This achievement is a direct result of our community's generosity and commitment to making a difference in the lives of those affected by RUNX1-FPD. And a big thank you to the anonymous donor who matched all donations dollar for dollar during the campaign!
We are deeply grateful for everyone who contributed, and look forward to continuing our work together in the coming year! |
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December Coffee Chat Summary: Imatinib Trail Insights |
At our December Coffee Chat we discussed the newly launched imatinib trial. Following the coffee chat, Dr. Lea Cunningham provided clarification to questions patients expressed during the chat: -
Location Requirements: Participants are not required to stay in Bethesda throughout the study. However, a brief onsite visit is needed initially and, for most, again on the 28th day of the study.
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Virtual Check-ins and Lab Work: The trial includes weekly telehealth appointments and home-based lab work. Importantly, the study will cover costs for specific lab tests needed for research purposes.
- International Participants: There's potential for accommodating patients from outside the U.S., expanding the scope of the study.
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Eligibility Queries: If you're uncertain about your eligibility, Dr. Cunningham is readily available for consultation. Find more details and contact information at NIH Clinical Studies.
Looking ahead, we have Coffee Chats scheduled for March, June, September, and December in 2024. Stay tuned for specific dates and registration information.
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Join the Research Guided by Patients Committee (RGPC) First Meeting of 2024: Thursday, February 29th at 9:30 a.m. PT |
The RGPC unites patients, scientists, physicians and genetic counselors in order to provide their different perspectives on RRP research, education and awareness efforts.
We're always welcoming new voices to enrich our discussions. Interested in contributing? It's simple. Members are expected to join quarterly one hour Zoom meetings, and contribute about an hour per month of material review and insights via email, phone or video call.
Our February 29th meeting will focus on the future of the RUNX1 Patient Data Hub. Patients not currently in the RGPC are also invited to participate. For more information or to join, please email Amanda Eggen, RRP’s Director of Patient Engagement and Clinical Programs. |
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First RRP MedEd Webinar of 2024
"Pulling the Molecular Thread Through Pediatric Thrombocytopenia, MDS & AML" |
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On January 8th, Dr. Michele Lambert and Dr. Timothy S. Olson of Children’s Hospital of Philadelphia were joined by Dr. Tanja Andrea Gruber of Lucile Packard Children's Hospital - Stanford, to present on making an accurate diagnosis by leveraging comprehensive molecular profiling, and management implications for pediatric patients with thrombocytopenia, MDS and AML. If you were unable to attend you can click the button below to watch the recording: |
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Please consider a gift of any size today to support cancer prevention research and help find treatments for RUNX1-FPD patients. Thank you for your continued support! |
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Did someone forward you this newsletter?
Sign up today to get your own copy delivered straight to your Inbox! |
RRP's 8th Annual RUNX1 Scientific Conference |
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Attention RUNX1 Researchers and Clinicians: Please plan to join us in Princeton, New Jersey, on Wednesday, November 6th and Thursday, November 7th for our 8th Annual RUNX1 Scientific Conference. Anyone interested in advancing RUNX1-FPD research and improving patient outcomes are welcome to attend! Click the button below to pre-register, and we will be in touch soon with more information and a detailed agenda: |
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RRP is proud to announce that our organization’s story is once again showcased in the 2024 Giving List publication.
Click the banner above to read more and then please share our story with everyone you know to help us make an impact on our ultimate goal - to prevent cancer.
The Giving List began in 2020 as a special print book and digital platform for nonprofits to help drive new supporters through story driven narratives. Its creators and their highly philanthropic core investor group understood that story-driven marketing would create opportunities for the region’s nonprofit community to engage greater numbers of existing and new donors. |
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|
Imatinib Clinical Trial Is Now Open For Participation
The National Cancer Institute (NCI) at the National Institutes of Health (NIH) is seeking participants for a Phase 1b clinical trial studying the drug imatinib for individuals with RUNX1 Familial Platelet Disorder (RUNX1-FPD).
The study team believes that this medicine may be effective in increasing RUNX1 protein levels in individuals with RUNX1-FPD who have low RUNX1 protein levels. |
This phase of the trial is focused on determining the highest dose of the drug imatinib that can be tolerated with minimal side effects. Eligibility includes adults diagnosed with RUNX1-FPD and healthy volunteers.
For more detailed information, please click here to view a one-page summary of the study. |
|
|
Join Us For The First Coffee Chat of 2024! |
No matter whether you're a newcomer or a familiar face, this is the perfect opportunity to meet and engage with fellow patients and family members. Come and share your journey or simply absorb the conversation - your presence is valuable and appreciated either way.
Register below and then join us on a device with internet access, a camera and a microphone (coffee is entirely optional!). |
|
|
Join the Research Guided by Patients Committee (RGPC) First Meeting of 2024: Thursday, February 29th at 9:30 a.m. PT |
|
|
The RGPC unites patients, caregivers, scientists and clinicians in order to combine their different perspectives on RRP's research support, patient education and awareness initiatives.
The meeting on February 29th will focus on outlining the committee's structure for the upcoming year, and identifying key areas where members can contribute to advancing RRP's mission.
We welcome new viewpoints to broaden our impact. If you're interested in learning more about the RGPC and exploring opportunities to contribute this year, please join us on the day of the meeting by clicking the button below:
|
RGPC members participate in at least two quarterly, hour-long Zoom meetings each year and offer insights through email, phone or video calls. Please email Amanda Eggen, RRP’s Director of Patient Engagement and Clinical Programs, if you have any additional questions about membership. |
|
|
NIH RUNX1-FPD Patient & Family Resources |
|
|
The NIH RUNX1 Natural History study team has begun creating resources to support families through their challenges at home and to offer more accessible information about the disease.
These resources include communication and wellbeing guides, and a RUNX1 fact sheet. You can find the resources at the bottom of their study page by clicking here. |
|
|
Seminario Web de Educación Médica de RRP Lunes, 15 de Julio 2:00 p.m. PT | 4:00 p.m. CT | 5:00 p.m. ET "Neoplasias Hematológicas Hereditarias: No Tan Poco Frecuentes" |
|
|
| We are thrilled to announce our first research webinar offered entirely in Spanish! Dr. Ximena Jordan-Bruno will share her presentation "Neoplasias Hematológicas Hereditarias: No Tan Poco Frecuentes" and then will be joined by expert panelists for a Q&A session.
Please share this webinar information widely with your Spanish-speaking colleagues! |
|
|
Objetivos de Aprendizaje: - Comprender el papel de las pruebas genéticas en los resultados de los pacientes con HM. (HM es el acrónimo en inglés.)
- Aprender la prevalencia de variantes germinales patógenas en pacientes con HM.
- Obtener herramientas sobre cómo sospechar y diagnosticar HM hereditario.
- Aprender cómo manejar a los pacientes con HM hereditario y a sus familiares en riesgo.
|
| Learning Objectives: -
Understand the role of genetic testing on patient outcomes for HM patients.
- Learn the prevalence of pathogenic germline variants in HM patients.
- Gain tools on how to suspect and diagnose hereditary HM.
- Learn how to manage hereditary HM patients and their at-risk family.
|
|
|
Submit your RUNX1 research projects! The Vera and Joseph Dresner Foundation recently launched a funding initiative aiming to support cutting-edge research and related programs in Myelodysplastic Syndromes (MDS) to advance future standards of care and seek a cure.
The initiative will award three grants: two Early Career Awards providing up to $125,000 per year for two years, and one Established Investigator Award providing up to $250,000 per year for two years.
Click here for more information.
|
|
|
We deeply appreciate every contribution from our patient community, particularly those who share their experiences through video. These stories are vital in helping us raise awareness about RUNX1-FPD and continue driving our mission forward.
We invite you to explore some of these inspiring stories by clicking on each of the pictures below: |
|
|
If you're interested in sharing your own story, please reach out to RRP's Communication Manager, Catherine Pelton.
|
|
|
Please support 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! |
|
|
Did someone forward you this newsletter?
Sign up today to get your own copy delivered straight to your Inbox! |
Please note: RRP's newsletter is best viewed in Light Mode versus Dark Mode on your device. |
|
|
Our Contact Information
*{{Organization Name}}*
*{{Organization Address}}*
*{{Organization Phone}}*
*{{Organization Website}}*
*{{Unsubscribe}}* |
| Follow us on social media for the latest news and updates! |
|
|
|
New Monthly RRP Newsletter Schedule
In order to keep you more promptly informed of the latest RRP and RUNX1-related news and developments, we're shifting our newsletter from a bimonthly to a monthly publication.
Look out for the latest edition landing in your Inbox around mid-month, every month! If you have anything you’d like to share in our newsletter, please email Catherine Pelton, RRP’s Communication Manager.
|
|
|
From significant research publications to impactful community engagements, the dedication and collaborative spirit of our community members last year continued to help us make groundbreaking progress.
Here are some of the highlights that made 2023 a year to remember: -
Our scientific community published nine pivotal RUNX1 research articles, including the first article from the NIH’s RUNX1 Clinical Research Study. You can find them all here: RUNX1 Radar Report.
-
Partnered with scientists and healthcare providers to support the launch of the first two RUNX1-focused clinical trials.
- Celebrated the first anniversary of the RUNX1 Patient Data Hub, with 69 patients enrolled and 47 contributing valuable health data.
-
Successfully conducted a series of educational webinars (engaging over 660 healthcare professionals), including:
-
Dr. Ximena Jordan Bruno was awarded the inaugural RRP Early Career Investigator – Clinical Research Grant for her proposed RUNX1-FPD biorepository project.
-
Received a Health Equity in RARE Impact Grant from Global Genes for our En Beneficio de su Salud (For the Benefit of Your Health) program designed to aid Spanish-speaking RUNX1-FPD individuals.
-
Raised over $20,000 through the 2023 X-Out Cancer Challenge to support cancer prevention and RUNX1-FPD research.
-
Hosted a virtual workshop with community members so they could learn more about RRP’s long-term research strategy and provide input to help ensure our community is moving in the right direction to support RUNX1-FPD patients and families.
-
Awarded two RRP-ALSF RUNX1 Early Career Investigator Grants, supporting the promising research of Dirk Loeffler, PhD, and the launch of a coordinated plan to develop clinical consensus guidelines for RUNX1-FPD with leadership by Brittany Stewart, CGC, PhD, and Hetty Carraway, MD.
-
Successfully conducted our 7th Annual RUNX1 Scientific Conference and 4th Annual Patient Meeting with more than 230 total attendees.
-
Held a special event for our donor community, Fast Track To A Cure: Employing a Business Mindset to Accelerate Cancer Prevention Research, featuring a fireside chat with RRP co-founder Tim Babich and RRP Executive Director Dr. Katrin Ericson.
|
|
|
Over $33,000 Raised for RUNX1 Research!
We're excited to announce that our 2023 From Labs to Lives year-end campaign was a success, raising over $33,000 for RUNX1 research and patient initiatives. This achievement is a direct result of our community's generosity and commitment to making a difference in the lives of those affected by RUNX1-FPD. And a big thank you to the anonymous donor who matched all donations dollar for dollar during the campaign!
We are deeply grateful for everyone who contributed, and look forward to continuing our work together in the coming year! |
|
|
December Coffee Chat Summary: Imatinib Trail Insights |
At our December Coffee Chat we discussed the newly launched imatinib trial. Following the coffee chat, Dr. Lea Cunningham provided clarification to questions patients expressed during the chat: -
Location Requirements: Participants are not required to stay in Bethesda throughout the study. However, a brief onsite visit is needed initially and, for most, again on the 28th day of the study.
-
Virtual Check-ins and Lab Work: The trial includes weekly telehealth appointments and home-based lab work. Importantly, the study will cover costs for specific lab tests needed for research purposes.
- International Participants: There's potential for accommodating patients from outside the U.S., expanding the scope of the study.
-
Eligibility Queries: If you're uncertain about your eligibility, Dr. Cunningham is readily available for consultation. Find more details and contact information at NIH Clinical Studies.
Looking ahead, we have Coffee Chats scheduled for March, June, September, and December in 2024. Stay tuned for specific dates and registration information.
|
|
|
Join the Research Guided by Patients Committee (RGPC) First Meeting of 2024: Thursday, February 29th at 9:30 a.m. PT |
The RGPC unites patients, scientists, physicians and genetic counselors in order to provide their different perspectives on RRP research, education and awareness efforts.
We're always welcoming new voices to enrich our discussions. Interested in contributing? It's simple. Members are expected to join quarterly one hour Zoom meetings, and contribute about an hour per month of material review and insights via email, phone or video call.
Our February 29th meeting will focus on the future of the RUNX1 Patient Data Hub. Patients not currently in the RGPC are also invited to participate. For more information or to join, please email Amanda Eggen, RRP’s Director of Patient Engagement and Clinical Programs. |
|
|
First RRP MedEd Webinar of 2024
"Pulling the Molecular Thread Through Pediatric Thrombocytopenia, MDS & AML" |
|
|
On January 8th, Dr. Michele Lambert and Dr. Timothy S. Olson of Children’s Hospital of Philadelphia were joined by Dr. Tanja Andrea Gruber of Lucile Packard Children's Hospital - Stanford, to present on making an accurate diagnosis by leveraging comprehensive molecular profiling, and management implications for pediatric patients with thrombocytopenia, MDS and AML. If you were unable to attend you can click the button below to watch the recording: |
|
|
Please consider a gift of any size today to support cancer prevention research and help find treatments for RUNX1-FPD patients. Thank you for your continued support! |
|
|
Did someone forward you this newsletter?
Sign up today to get your own copy delivered straight to your Inbox! |
Please note: RRP's newsletter is best viewed in Light Mode versus Dark Mode on your device. |
|
|
Our Contact Information
*{{Organization Name}}*
*{{Organization Address}}*
*{{Organization Phone}}*
*{{Organization Website}}*
*{{Unsubscribe}}* |
| Follow us on social media for the latest news and updates! |
|
|
|
RRP's 8th Annual RUNX1 Scientific Conference |
|
|
Attention RUNX1 Researchers and Clinicians: Please plan to join us in Princeton, New Jersey, on Wednesday, November 6th and Thursday, November 7th for our 8th Annual RUNX1 Scientific Conference. Anyone interested in advancing RUNX1-FPD research and improving patient outcomes are welcome to attend! Click the button below to pre-register, and we will be in touch soon with more information and a detailed agenda: |
|
|
RRP is proud to announce that our organization’s story is once again showcased in the 2024 Giving List publication.
Click the banner above to read more and then please share our story with everyone you know to help us make an impact on our ultimate goal - to prevent cancer.
The Giving List began in 2020 as a special print book and digital platform for nonprofits to help drive new supporters through story driven narratives. Its creators and their highly philanthropic core investor group understood that story-driven marketing would create opportunities for the region’s nonprofit community to engage greater numbers of existing and new donors. |
|
|
Imatinib Clinical Trial Is Now Open For Participation
The National Cancer Institute (NCI) at the National Institutes of Health (NIH) is seeking participants for a Phase 1b clinical trial studying the drug imatinib for individuals with RUNX1 Familial Platelet Disorder (RUNX1-FPD).
The study team believes that this medicine may be effective in increasing RUNX1 protein levels in individuals with RUNX1-FPD who have low RUNX1 protein levels. |
This phase of the trial is focused on determining the highest dose of the drug imatinib that can be tolerated with minimal side effects. Eligibility includes adults diagnosed with RUNX1-FPD and healthy volunteers.
For more detailed information, please click here to view a one-page summary of the study. |
|
|
Join Us For The First Coffee Chat of 2024! |
No matter whether you're a newcomer or a familiar face, this is the perfect opportunity to meet and engage with fellow patients and family members. Come and share your journey or simply absorb the conversation - your presence is valuable and appreciated either way.
Register below and then join us on a device with internet access, a camera and a microphone (coffee is entirely optional!). |
|
|
Join the Research Guided by Patients Committee (RGPC) First Meeting of 2024: Thursday, February 29th at 9:30 a.m. PT |
|
|
The RGPC unites patients, caregivers, scientists and clinicians in order to combine their different perspectives on RRP's research support, patient education and awareness initiatives.
The meeting on February 29th will focus on outlining the committee's structure for the upcoming year, and identifying key areas where members can contribute to advancing RRP's mission.
We welcome new viewpoints to broaden our impact. If you're interested in learning more about the RGPC and exploring opportunities to contribute this year, please join us on the day of the meeting by clicking the button below:
|
RGPC members participate in at least two quarterly, hour-long Zoom meetings each year and offer insights through email, phone or video calls. Please email Amanda Eggen, RRP’s Director of Patient Engagement and Clinical Programs, if you have any additional questions about membership. |
|
|
NIH RUNX1-FPD Patient & Family Resources |
|
|
The NIH RUNX1 Natural History study team has begun creating resources to support families through their challenges at home and to offer more accessible information about the disease.
These resources include communication and wellbeing guides, and a RUNX1 fact sheet. You can find the resources at the bottom of their study page by clicking here. |
|
|
Seminario Web de Educación Médica de RRP Lunes, 15 de Julio 2:00 p.m. PT | 4:00 p.m. CT | 5:00 p.m. ET "Neoplasias Hematológicas Hereditarias: No Tan Poco Frecuentes" |
|
|
| We are thrilled to announce our first research webinar offered entirely in Spanish! Dr. Ximena Jordan-Bruno will share her presentation "Neoplasias Hematológicas Hereditarias: No Tan Poco Frecuentes" and then will be joined by expert panelists for a Q&A session.
Please share this webinar information widely with your Spanish-speaking colleagues! |
|
|
Objetivos de Aprendizaje: - Comprender el papel de las pruebas genéticas en los resultados de los pacientes con HM. (HM es el acrónimo en inglés.)
- Aprender la prevalencia de variantes germinales patógenas en pacientes con HM.
- Obtener herramientas sobre cómo sospechar y diagnosticar HM hereditario.
- Aprender cómo manejar a los pacientes con HM hereditario y a sus familiares en riesgo.
|
| Learning Objectives: -
Understand the role of genetic testing on patient outcomes for HM patients.
- Learn the prevalence of pathogenic germline variants in HM patients.
- Gain tools on how to suspect and diagnose hereditary HM.
- Learn how to manage hereditary HM patients and their at-risk family.
|
|
|
Submit your RUNX1 research projects! The Vera and Joseph Dresner Foundation recently launched a funding initiative aiming to support cutting-edge research and related programs in Myelodysplastic Syndromes (MDS) to advance future standards of care and seek a cure.
The initiative will award three grants: two Early Career Awards providing up to $125,000 per year for two years, and one Established Investigator Award providing up to $250,000 per year for two years.
Click here for more information.
|
|
|
We deeply appreciate every contribution from our patient community, particularly those who share their experiences through video. These stories are vital in helping us raise awareness about RUNX1-FPD and continue driving our mission forward.
We invite you to explore some of these inspiring stories by clicking on each of the pictures below: |
|
|
If you're interested in sharing your own story, please reach out to RRP's Communication Manager, Catherine Pelton.
|
|
|
Please support 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! |
|
|
RRP's 8th Annual RUNX1 Scientific Conference |
|
|
Attention RUNX1 Researchers and Clinicians: Please plan to join us in Princeton, New Jersey, on Wednesday, November 6th and Thursday, November 7th for our 8th Annual RUNX1 Scientific Conference. Anyone interested in advancing RUNX1-FPD research and improving patient outcomes are welcome to attend! Click the button below to pre-register, and we will be in touch soon with more information and a detailed agenda: |
|
|
RRP is proud to announce that our organization’s story is once again showcased in the 2024 Giving List publication.
Click the banner above to read more and then please share our story with everyone you know to help us make an impact on our ultimate goal - to prevent cancer.
The Giving List began in 2020 as a special print book and digital platform for nonprofits to help drive new supporters through story driven narratives. Its creators and their highly philanthropic core investor group understood that story-driven marketing would create opportunities for the region’s nonprofit community to engage greater numbers of existing and new donors. |
|
|
Imatinib Clinical Trial Is Now Open For Participation
The National Cancer Institute (NCI) at the National Institutes of Health (NIH) is seeking participants for a Phase 1b clinical trial studying the drug imatinib for individuals with RUNX1 Familial Platelet Disorder (RUNX1-FPD).
The study team believes that this medicine may be effective in increasing RUNX1 protein levels in individuals with RUNX1-FPD who have low RUNX1 protein levels. |
This phase of the trial is focused on determining the highest dose of the drug imatinib that can be tolerated with minimal side effects. Eligibility includes adults diagnosed with RUNX1-FPD and healthy volunteers.
For more detailed information, please click here to view a one-page summary of the study. |
|
|
Join Us For The First Coffee Chat of 2024! |
No matter whether you're a newcomer or a familiar face, this is the perfect opportunity to meet and engage with fellow patients and family members. Come and share your journey or simply absorb the conversation - your presence is valuable and appreciated either way.
Register below and then join us on a device with internet access, a camera and a microphone (coffee is entirely optional!). |
|
|
Join the Research Guided by Patients Committee (RGPC) First Meeting of 2024: Thursday, February 29th at 9:30 a.m. PT |
|
|
The RGPC unites patients, caregivers, scientists and clinicians in order to combine their different perspectives on RRP's research support, patient education and awareness initiatives.
The meeting on February 29th will focus on outlining the committee's structure for the upcoming year, and identifying key areas where members can contribute to advancing RRP's mission.
We welcome new viewpoints to broaden our impact. If you're interested in learning more about the RGPC and exploring opportunities to contribute this year, please join us on the day of the meeting by clicking the button below:
|
RGPC members participate in at least two quarterly, hour-long Zoom meetings each year and offer insights through email, phone or video calls. Please email Amanda Eggen, RRP’s Director of Patient Engagement and Clinical Programs, if you have any additional questions about membership. |
|
|
NIH RUNX1-FPD Patient & Family Resources |
|
|
The NIH RUNX1 Natural History study team has begun creating resources to support families through their challenges at home and to offer more accessible information about the disease.
These resources include communication and wellbeing guides, and a RUNX1 fact sheet. You can find the resources at the bottom of their study page by clicking here. |
|
|
Seminario Web de Educación Médica de RRP Lunes, 15 de Julio 2:00 p.m. PT | 4:00 p.m. CT | 5:00 p.m. ET "Neoplasias Hematológicas Hereditarias: No Tan Poco Frecuentes" |
|
|
| We are thrilled to announce our first research webinar offered entirely in Spanish! Dr. Ximena Jordan-Bruno will share her presentation "Neoplasias Hematológicas Hereditarias: No Tan Poco Frecuentes" and then will be joined by expert panelists for a Q&A session.
Please share this webinar information widely with your Spanish-speaking colleagues! |
|
|
Objetivos de Aprendizaje: - Comprender el papel de las pruebas genéticas en los resultados de los pacientes con HM. (HM es el acrónimo en inglés.)
- Aprender la prevalencia de variantes germinales patógenas en pacientes con HM.
- Obtener herramientas sobre cómo sospechar y diagnosticar HM hereditario.
- Aprender cómo manejar a los pacientes con HM hereditario y a sus familiares en riesgo.
|
| Learning Objectives: -
Understand the role of genetic testing on patient outcomes for HM patients.
- Learn the prevalence of pathogenic germline variants in HM patients.
- Gain tools on how to suspect and diagnose hereditary HM.
- Learn how to manage hereditary HM patients and their at-risk family.
|
|
|
Submit your RUNX1 research projects! The Vera and Joseph Dresner Foundation recently launched a funding initiative aiming to support cutting-edge research and related programs in Myelodysplastic Syndromes (MDS) to advance future standards of care and seek a cure.
The initiative will award three grants: two Early Career Awards providing up to $125,000 per year for two years, and one Established Investigator Award providing up to $250,000 per year for two years.
Click here for more information.
|
|
|
We deeply appreciate every contribution from our patient community, particularly those who share their experiences through video. These stories are vital in helping us raise awareness about RUNX1-FPD and continue driving our mission forward.
We invite you to explore some of these inspiring stories by clicking on each of the pictures below: |
|
|
If you're interested in sharing your own story, please reach out to RRP's Communication Manager, Catherine Pelton.
|
|
|
Please support 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! |
|
|
New Monthly RRP Newsletter Schedule
In order to keep you more promptly informed of the latest RRP and RUNX1-related news and developments, we're shifting our newsletter from a bimonthly to a monthly publication.
Look out for the latest edition landing in your Inbox around mid-month, every month! If you have anything you’d like to share in our newsletter, please email Catherine Pelton, RRP’s Communication Manager.
|
|
|
From significant research publications to impactful community engagements, the dedication and collaborative spirit of our community members last year continued to help us make groundbreaking progress.
Here are some of the highlights that made 2023 a year to remember: -
Our scientific community published nine pivotal RUNX1 research articles, including the first article from the NIH’s RUNX1 Clinical Research Study. You can find them all here: RUNX1 Radar Report.
-
Partnered with scientists and healthcare providers to support the launch of the first two RUNX1-focused clinical trials.
- Celebrated the first anniversary of the RUNX1 Patient Data Hub, with 69 patients enrolled and 47 contributing valuable health data.
-
Successfully conducted a series of educational webinars (engaging over 660 healthcare professionals), including:
-
Dr. Ximena Jordan Bruno was awarded the inaugural RRP Early Career Investigator – Clinical Research Grant for her proposed RUNX1-FPD biorepository project.
-
Received a Health Equity in RARE Impact Grant from Global Genes for our En Beneficio de su Salud (For the Benefit of Your Health) program designed to aid Spanish-speaking RUNX1-FPD individuals.
-
Raised over $20,000 through the 2023 X-Out Cancer Challenge to support cancer prevention and RUNX1-FPD research.
-
Hosted a virtual workshop with community members so they could learn more about RRP’s long-term research strategy and provide input to help ensure our community is moving in the right direction to support RUNX1-FPD patients and families.
-
Awarded two RRP-ALSF RUNX1 Early Career Investigator Grants, supporting the promising research of Dirk Loeffler, PhD, and the launch of a coordinated plan to develop clinical consensus guidelines for RUNX1-FPD with leadership by Brittany Stewart, CGC, PhD, and Hetty Carraway, MD.
-
Successfully conducted our 7th Annual RUNX1 Scientific Conference and 4th Annual Patient Meeting with more than 230 total attendees.
-
Held a special event for our donor community, Fast Track To A Cure: Employing a Business Mindset to Accelerate Cancer Prevention Research, featuring a fireside chat with RRP co-founder Tim Babich and RRP Executive Director Dr. Katrin Ericson.
|
|
|
Over $33,000 Raised for RUNX1 Research!
We're excited to announce that our 2023 From Labs to Lives year-end campaign was a success, raising over $33,000 for RUNX1 research and patient initiatives. This achievement is a direct result of our community's generosity and commitment to making a difference in the lives of those affected by RUNX1-FPD. And a big thank you to the anonymous donor who matched all donations dollar for dollar during the campaign!
We are deeply grateful for everyone who contributed, and look forward to continuing our work together in the coming year! |
|
|
December Coffee Chat Summary: Imatinib Trail Insights |
At our December Coffee Chat we discussed the newly launched imatinib trial. Following the coffee chat, Dr. Lea Cunningham provided clarification to questions patients expressed during the chat: -
Location Requirements: Participants are not required to stay in Bethesda throughout the study. However, a brief onsite visit is needed initially and, for most, again on the 28th day of the study.
-
Virtual Check-ins and Lab Work: The trial includes weekly telehealth appointments and home-based lab work. Importantly, the study will cover costs for specific lab tests needed for research purposes.
- International Participants: There's potential for accommodating patients from outside the U.S., expanding the scope of the study.
-
Eligibility Queries: If you're uncertain about your eligibility, Dr. Cunningham is readily available for consultation. Find more details and contact information at NIH Clinical Studies.
Looking ahead, we have Coffee Chats scheduled for March, June, September, and December in 2024. Stay tuned for specific dates and registration information.
|
|
|
Join the Research Guided by Patients Committee (RGPC) First Meeting of 2024: Thursday, February 29th at 9:30 a.m. PT |
The RGPC unites patients, scientists, physicians and genetic counselors in order to provide their different perspectives on RRP research, education and awareness efforts.
We're always welcoming new voices to enrich our discussions. Interested in contributing? It's simple. Members are expected to join quarterly one hour Zoom meetings, and contribute about an hour per month of material review and insights via email, phone or video call.
Our February 29th meeting will focus on the future of the RUNX1 Patient Data Hub. Patients not currently in the RGPC are also invited to participate. For more information or to join, please email Amanda Eggen, RRP’s Director of Patient Engagement and Clinical Programs. |
|
|
First RRP MedEd Webinar of 2024
"Pulling the Molecular Thread Through Pediatric Thrombocytopenia, MDS & AML" |
|
|
On January 8th, Dr. Michele Lambert and Dr. Timothy S. Olson of Children’s Hospital of Philadelphia were joined by Dr. Tanja Andrea Gruber of Lucile Packard Children's Hospital - Stanford, to present on making an accurate diagnosis by leveraging comprehensive molecular profiling, and management implications for pediatric patients with thrombocytopenia, MDS and AML. If you were unable to attend you can click the button below to watch the recording: |
|
|
Please consider a gift of any size today to support cancer prevention research and help find treatments for RUNX1-FPD patients. Thank you for your continued support! |
|
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