The RUNX1 Research Program 10th Annual Scientific Conference and Patient Meeting

About This Grant

PROJECT SUMMARY The main goal of the RUNX1 Research Program 10th Annual Scientific Conference and Patient Meeting is to create a dynamic convening to foster collaboration and provide opportunities for knowledge and data sharing among a diverse set of researchers, clinicians, and patients and their families for a rare disease called RUNX1 familial platelet disorder with predisposition to hematologic malignancies (RUNX1-FPD or FPDMM). Individuals with the disorder have a 35-50% lifetime risk of developing a hematologic malignancy. Of those who develop a malignancy, over 50% are diagnosed with AML. It is the only convening of its kind and continues to be an important part of deepening the communities’ understanding of the disease, of driving collaborations, and facilitating the discovery of future therapies for patients. Equally important is the opportunity for early career investigators, posts, and graduate students to present their research and receive feedback from a diverse, multidisciplinary community, a critical aspect of their training. RRP is committed to cultivating the next generation of RUNX1-FPD basic, translational, and clinical researchers. Conference participation has grown since our first conference in 2017, which had only 18 attendees. By 2021 we had over 181 attendees for the virtual conference. In 2022, we had 130 individuals - 40 patients and 90 scientists, clinicians, and genetic counselors - who attended in person in Princeton, NJ. Thanks to NCATS/NIH funding, we were able to award five (5) deserving young investigators with travel scholarships to attend the conference in 2022 and present at the poster sessions. In 2023, we returned to a virtual format with 397 registrants and a total of 219 participants. Once again, we held a poster session with four (4) young investigators.At our 2024 in-person scientific conference in Princeton, NJ we had 84 scientists, clinicians, and genetic counselors. As a result of NCI funding for the conference, we awarded travel scholarships to six (6) trainees.In 2024, we did not host a formal patient meeting alongside the scientific conference because we decided to pilot a smaller regional patient meeting in Houston to better reach patients outside of the northeast. This decision was influenced by a patient community-wide survey we conducted. Regardless, there were still seven patients and patient family members who attended the scientific conference. In 2025, our scientific conference and patient meeting will be held in September. The agenda includes research-specific sessions open to all stakeholders as well as a track dedicated to RUNX1-FPD patients and family members. The objective is to promote and drive patient-engaged research forward, with direct patient input in the development and planning of the sessions via our 23-member Research Guided by Patients Committee (RGPC). The research-focused portion of the meeting will bring together experts from the fields of hematology, immunology, oncology, cell therapy, and basic science who are committed to uncovering the mechanisms underlying the pathogenesis of RUNX1-FPD with the goal of developing therapeutic interventions that impact the bleeding and immune dysfunction issues and most importantly the predisposition to malignancy. The audience will include investigators devoted to studying RUNX1, leukemia progression, clonal hematopoiesis, gene editing, and hematopoietic stem cell transplants. It has become increasingly clear through the NHGRI-sponsored natural history study of RUNX1-FPD that immune dysfunction and inflammatory disorders are common. RUNX1-FPD includes a unique patient population where there may be a nexus between immunology and oncology based entirely on the reduced and/or altered activity of a single transcription factor, RUNX1.