The RARE Grant Program

The RARE Grant Program - Research Accelerating RUNX1 Exploration is jointly supported by the RUNX1 Research Program and Alex's Lemonade Stand Foundation. The RUNX1 Research Program is a research and advocacy initiative focused on improving quality of life and preventing blood cancer in people with RUNX1-FPD, a hereditary blood cancer predisposition syndrome, through awareness, collaborative research, and engagement with the patient community. Alex's Lemonade Stand Foundation is a nonprofit organization focused on childhood cancer through research funding, awareness, family support, and efforts to advance cures. The organizations continue their partnership through the RARE Grant to support research directed at RUNX1-FPD. The program's key objective is to accelerate discovery of druggable oncogenic pathways and advance cancer interception and cancer prevention therapeutics for children and family members with RUNX1-FPD. The two-year award supports research intended either to intercept progression from a precancerous state, including clonal hematopoiesis, to myelodysplastic syndrome or acute myeloid leukemia, or to prevent blood cancer before clonal hematopoiesis occurs. Proposed aims must be relevant to preventing hematologic malignancies in RUNX1-FPD. Projects with clear translational potential from bench to bedside receive priority, although novel and compelling research across the continuum is welcomed. Priority areas include clonal hematopoiesis in the context of germline RUNX1 mutations, the functional distinction between adaptive and maladaptive clones, and mechanisms by which clones resist or adapt to inflammatory pressures. The RARE Grant provides up to $250,000 USD in direct costs over two years, with no more than $125,000 in total costs requested in either year. Budgets should be proportional to the proposed project. The RUNX1 Research Program and Alex's Lemonade Stand Foundation follow the NIH salary cap for principal investigators and co-investigators, and indirect costs are not allowed. Allowable budget categories may include fringe benefits, reasonable travel to research meetings, supplies, small equipment, computer and software expenses, and properly budgeted subcontracts or subawards. Tuition remission is prohibited. Subcontract and subaward costs must be included in the project budget, and the principal investigator's institution is responsible for disbursing those funds. The award is not renewable, although one no-cost extension may be requested. No cost-share or matching contribution is stated as a requirement. Applicant institutions may be located in the United States or outside the United States, and applicants do not need to be U.S. citizens. Award funds must be granted to nonprofit institutions or organizations. Applicants must hold an MD, PhD, MD/PhD, DO, MBBS, or equivalent degree and must have research experience and deep understanding of normal or malignant hematopoiesis and/or immunology. Applicants may hold grants from other funding sources during the RARE award period when clear documentation shows how scientific and budgetary overlap will be avoided. Research involving human embryonic stem cells or non-human primates is not funded, while research using human induced pluripotent stem cells is permissible. Awardees are expected to share unique resources developed through the grant and must submit a Resource Sharing Plan describing the resources, sharing mechanisms, timing, characterization, and resources necessary for dissemination. A complete proposal must be assembled as one PDF not exceeding 20 MB and submitted through Proposal Central. An ORCID is required. The application includes a cover page, table of contents, scientific abstract, impact statement, budget and justification, NIH-format biographical sketches for the principal investigator and key personnel, and a research plan containing specific aims, significance, innovation, approach, milestones, expected outcomes, potential problems, contingency plans, and a simple Gantt chart. Applicants must also include the required Resource Sharing section, a plan for clinical translation within a five-year horizon, applicable human-subjects and vertebrate-animal documentation, literature cited, relevant letters of support, and any permitted appendix material. Applicants may save an application in progress, upload the single PDF through the Attachments tab, and submit it through Proposal Central. A confirmation email is expected within approximately 10 minutes, and an application may be unsubmitted and revised before the deadline. Applications are reviewed by an independent expert panel using an NIH-recognized peer-review process. Review considerations include the applicant's training and education record, publication record, grant and research awards or recognition, overall proposal quality under the NIH 9-point scoring system, evidence of collaboration between research groups, alignment with prevention of hematologic malignancies in RUNX1-FPD, and the feasibility of translating findings into the clinic. Resource-sharing plans are also assessed for how dissemination will enhance or diminish the impact of the work. Funded investigators must provide six-month interim and annual progress reports, with continued funding contingent on satisfactory scientific progress and appropriate expenditures, and must submit a final report. Investigators are also expected to present project progress at the annual RUNX1 Research Program scientific meeting. Full proposals were due December 11, 2025 at 8:00 PM ET. Award notification was scheduled for February 2026, with a projected project period from April 2026 through April 2028. The guidelines do not state a recurring application schedule or a confirmed opening date for a subsequent round, so no future deadline is inferred. Questions about eligibility or research-topic suitability may be directed to Katrin Ericson at KEricson@Runx1-FPD.org. Other program questions may be directed to Margaret Poore at M.Poore@AlexsLemonade.org, and the guidelines also list Grants@AlexsLemonade.org and 866-333-1213 as general Alex's Lemonade Stand Foundation grant contacts. Proposal Central account questions should be directed to pcsupport@altum.com.

Meet every guideline and eligibility criterion because noncompliant applications are administratively rejected; emphasize a high-quality research proposal under the NIH 9-point scoring system; demonstrate meaningful collaboration between research groups; align the work with prevention of hematologic malignancies in RUNX1-FPD; show feasible bench-to-bedside translation; provide clear milestones, expected outcomes, risks, and contingency plans; describe concrete resource-sharing methods and timing; avoid prohibited human embryonic stem-cell and non-human-primate research.