RARE Grant Program – Research Accelerating RUNX1 Exploration

Funding Agency:
Alexs Lemonade Stand Foundation

The RUNX1 Research Program and Alex’s Lemonade Stand Foundation are delighted to continue their partnership this year and are excited to launch the Research Accelerating RUNX1 Exploration (RARE) Grant.  

The Research Accelerating RUNX1 Exploration Grant is a two-year award designed to fund research that will lead to the development of therapies for patients with RUNX1-FPD that will either intercept the transition from a precancer state (clonal hematopoiesis) to MDS/AML or prevention of cancer (before clonal hematopoiesis).  

The purpose of the grant is to fund research that will accelerate the discovery of druggable oncogenic pathways and deliver cancer interception and cancer prevention therapeutics for RUNX1-FPD children and family members. 

Application Due Date: December 4, 2023

Agency Website

Areas of Interest

The following areas of research are of priority and are not ranked according to their order:  

Drug Repurposing and Clinical Translation • Support for preclinical experiments and correlative clinical studies designed to test regulatory-approved agents for the rescue of RUNX1-FPD hematopoietic function and interception of high-risk clonal hematopoiesis.  

RUNX1 Biology • Characterize the normal function of RUNX1 and the function of germline RUNX1 mutant proteins.  High interest in defining the role of RUNX1 in DNA damage repair in HSCs and the potential role germline RUNX1 mutations may play in acquisition of somatic mutations. • Identify regulators of RUNX1 expression and function to discover novel methods of correcting RUNX1 function within the hematopoietic system.  

Discovery • Investigate feasibility of targeting RNA splicing for disease therapy in patients with splicesite mutations or nonsense suppression therapies for patients with nonsense mutations. • Develop therapeutic strategies to raise normal RUNX1 activity or RUNX1 protein levels to improve hematopoietic function and reduce HM risk. • Identify druggable mechanisms, with a focus on drug repurposing, that drive clonal hematopoiesis, clonal expansion and ultimately malignant transformation in RUNX1-FPD patients. 

Eligibility Requirements

• Applicant institutions may be based in the U.S. or outside of the U.S. Applicants need not be United States citizens. Funds must be granted to non-profit institutions or organizations.

• Applicants must have an MD, PhD, or MD/PhD (DO, MBBS or equivalent).  

• Applicants may have research grants from other funding sources during the award period, but there must be clear documentation of mechanisms to avoid scientific and budgetary overlap.

• Applicants must have research experience working in, and a deep understanding of, normal or malignant hematopoiesis and/or immunology. 



Amount Description

A Research Accelerating RUNX1 Exploration Grant is $250,000 over 2 years (maximum $125,000 per year may be requested). 

Funding Type





Medical - Basic Science
Medical - Clinical Science
Medical - Translational

External Deadline

December 4, 2023