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Alex’s Lemonade Stand Foundation and The Babich Family Foundation Announce First Recipients of Familial RUNX1 Grant Program

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Alex’s Lemonade Stand Foundation and The Babich Family Foundation Announce First Recipients of Familial RUNX1 Grant Program

Funding supports research of Familial RUNX1 disorders and the predisposition of acute myeloid leukemia.

Alex’s Lemonade Stand Foundation (ALSF) and The Babich Family Foundation partnered together in 2016 to create new funding opportunities to accelerate research of a genetic familial platelet disorder with a predisposition to the development of acute myeloid leukemia (AML). ALSF and The Babich Family Foundation are excited to announce the recipients of the first Familial RUNX1 Grants: Leonard Zon, MD of Boston Children's Hospital Corporation, Alan Cantor, MD/PhD of Boston Children's Hospital Corporation, Eirini Papapetrou, MD/PhD of Icahn School of Medicine at Mount Sinai, Ravi Majeti, MD/PhD of Stanford University and Marshall Horwitz, MD/PhD of University of Washington Seattle.

The grantees will receive $250,000 over the course of two years to research prevention of the transition from pre-leukemia to leukemia for patients with the familial RUNX1 disorder. Leonard Zon will use a zebrafish model to study the process of leukemia initiation that is accompanied with expansion of an abnormal stem cell clone to better understand how the disease is established as well as to screen drug compounds to treat the progression. Alan Cantor will screen drugs in order to increase the activity of the wildtype, or remaining, functioning RUNX1 gene copy in order to prevent leukemia development. Eirini Papapetrou will develop a novel model (IPS cell based) of leukemia progression from familial RUNX1 mutations to study the changes that occur upon transformation in order to pinpoint possible targeted therapies. Ravi Majeti will use CRISPR to engineer RUNX1 mutations in both stem cells and its surrounding micro-environment to investigate how the mutation, under various scenarios, can impact disease development. Marshall Horwitz will utilize a number of novel therapies in order to attempt to stop leukemia development by having the wildtype, or remaining functioning RUNX1 gene copy, produce more proteins. 

Alex’s Lemonade Stand Foundation is committed to creating opportunities for new and innovative research into treatments and cures for childhood cancers. The RUNX1 Research Project is a research and advocacy venture committed to funding world-class, innovative and cross-disciplinary cancer research to find a cure for those affected by the RUNX1 familial platelet disorder with a predisposition to acute myeloid leukemia (FPD/AML). The Familial RUNX1 Grant program will accelerate research into inherited RUNX1 mutations or FPD/AML. Individuals with a mutation have a 50% likelihood during their life to develop AML. The current research of inherited RUNX1 mutations is very limited. Reported cases show people with inherited RUNX1 mutations can develop AML any time from age 2 to over 70.

“Through our partnership with The Babich Family Foundation, we’re excited to fund these promising research projects in an area that’s critical but underfunded,” said Jay Scott, co-executive director of ALSF. “We hope it will lead to better treatments for patients with RUNX1 who develop AML and ultimately all patients with AML, which is a deadly leukemia in children.”

“Our first grant cycle has funded five world-class leukemia research laboratories which is the first step in our goal of finding a cure for patients with the RUNX1 FPD/AML gene mutation,” said Timothy Babich of The Babich Family Foundation. “Alex's Lemonade Stand Foundation has been a wonderful partner of ours.”

For more information about RUNX1, visit www.runx1.com. For more information about ALSF’s various grant categories and successes, visit: www.ALSFgrants.org.