Studies Will advance research on the role inflammation and the immune system play in the development of blood cancers
The Mark Foundation for Cancer Research and the RUNX1 Research Program have partnered to fund innovative scientific studies that could determine the role inflammation and the immune system play in the development of blood cancers in patients with RUNX1 familial platelet disorder (RUNX1-FPD). RUNX1-FPD is a rare hereditary blood disorder which increases the risk of developing blood cancer to 30x greater than the general population; these studies will provide an opportunity to study cancer initiation in this predisposed group.
The Seeking Transformational Research Venture (STRIVE) Grant supports researchers studying how inflammation and the immune system play a role in the evolution from preleukemia to full-scale blood cancer in those with RUNX1-FPD. The overarching goal of the program is to determine whether controlling inflammation or improving the function of the immune system with specific medicines could possibly be the key to cancer prevention treatments.
Recent results from close monitoring of RUNX1-FPD patients have shown that elevated levels of inflammation and immune disorders are commonplace. These findings, in parallel with the oncology community’s long-standing knowledge that chronic inflammation and a dysfunctional immune system is associated with cancer, led to the development of this grant program.
“We are excited to partner with the RUNX1 Research Program to support innovative research that will increase our understanding how germline RUNX1 mutations impart changes in the immune system and hopefully will lead to critical insights into cancer disease initiation and progression mechanisms,” says Ryan Schoenfeld, PhD, The Mark Foundation Interim CEO, Chief Scientific Officer.
RUNX1-FPD is caused by mutations in the RUNX1 gene that are passed down from generation to generation. The predominant type of blood cancer RUNX1-FPD patients develop is acute myeloid leukemia (AML), but other hematologic malignancies like myelodysplastic syndrome (MDS) may also occur. In the general population most people who develop blood cancer are in their 60s or older, but the median age for developing blood cancer for those with a RUNX1 mutation is 29.
The increased risk of blood cancer in RUNX1-FPD patients affords researchers a rare, real-time glimpse into the first stages of leukemia in the 30-50% who go on to develop it.
Through the STRIVE program, the foundations have already awarded a combined $750,000 in grants to three research groups:
- Lucio Castilla, Professor of Molecular, Cell and Cancer Biology at University of Massachusetts, and his team have generated a mouse model recapitulating a common RUNX1 mutation found in patients with RUNX1-FPD, which, like their human counterparts, are predisposed to developing hematologic malignancies. The team will treat these mice with the drug etanercept and monitor the effect this has on the mice.
- Lucy Godley, Professor of Medicine at University of Chicago, and her group will test the idea that the elevated inflammation characteristic of inherited RUNX1-mutations is due in part to altered epigenetic organization in stromal cells in the bone marrow that leads to pro-inflammatory gene expression.
- Ravi Majeti, Professor of Medicine at Stanford University, and his team are using computational tools and experimental methods to identify existing drugs that can be repurposed to target inflammation in RUNX1-FPD, and to model the effect of additional acquired mutations on inflammation and progression of RUNX1-FPD to acute myeloid leukemia (AML).
“Each of these projects will bring us one step closer to achieving our commitment to discover cancer prevention interventions for our community. These projects are highly focused and are designed to address critical questions within the research community. We are thrilled that The Mark Foundation for Cancer Research shares our enthusiasm for the importance of this work,” said Katrin Ericson, PhD, Executive Director of the RUNX1 Research Program.
About The Mark Foundation for Cancer Research
The Mark Foundation for Cancer Research actively partners with scientists to accelerate research that will transform the prevention, diagnosis, and treatment of cancer. The Mark Foundation fulfills its mission by supporting groundbreaking science carried out by individual investigators, multi-disciplinary teams, and inter-institutional collaborations in the United States, Europe, and across the globe. Recognizing the obstacles that prevent scientific advances from improving patient outcomes, The Mark Foundation maintains a nimble, high-impact approach to funding cancer research that bridges the gap between bench and bedside through grants and early-stage venture investments.
Since 2017, The Mark Foundation has awarded more than $125 million in grants to enable innovative basic, translational, and clinical cancer research, including drug discovery. The Mark Foundation also has a robust and growing portfolio of investments in oncology companies developing novel therapeutics and diagnostics. Through its research and venture arms, The Mark Foundation supports projects throughout their life cycle to ensure their highest chance of success in impacting the lives of patients with breakthroughs in cancer care.
To learn more please visit www.themarkfoundation.org.
About The RUNX1 Research Program
The RUNX1 Research Program (RRP) is the only non-profit advocacy and research-funding foundation in the world that supports patients with RUNX1 Familial Platelet Disorder (RUNX1-FPD). Our mission is to improve the quality of life and prevent cancer in RUNX1-FPD patients as we continue building awareness for this rare blood disorder. We have built a multi-stakeholder community who partners with us to facilitate world-class collaborative research. To learn more about RRP and RUNX1-FPD, visit www.runx1-fpd.org.