How Rally’s Support Helped Target a Rare and Aggressive Childhood Leukemia

Cheng-Kui Qu, M.D., Ph.D., a researcher at Emory University, received seed funding from Rally Foundation from 2018-2022. That early and consistent support allowed his lab to make meaningful progress toward developing new treatments for juvenile myelomonocytic leukemia (JMML), a rare and aggressive childhood blood cancer that is difficult to cure. 

In 2018, Rally’s funding helped Dr. Qu and his team explore whether a clinically used antipsychotic drug called pimozide might have a therapeutic effect against JMML.  

While pimozide is typically used to treat neurological disorders by blocking dopamine receptors in the brain, researchers are learning it may also affect cancer cell survival pathways. In the lab, pimozide has shown potential to disrupt cancer growth by interfering with key proteins that leukemia cells rely on.   

This research wasn’t about using a psychiatric drug to treat cancer directly, but about exploring how it could be repurposed to attack leukemic stem cells, the root cells that drive JMML and make it hard to treat. 

The preliminary data Dr. Qu and his team gathered with Rally Foundation’s help directly led to a prestigious National Institutes of Health (NIH) R21 grant, providing funding to continue exploring how to eliminate these tumor precursor cells. 

Then in 2022, thanks to another round of early funding, the team investigated a surprising new angle: using an antibiotic commonly used in dental antiseptics called alexidine dihydrochloride to eliminate JMML precursor cells. These encouraging results laid the groundwork for an NIH R01 proposal, which the team plans to submit in 2026. 

“Without Rally’s early investment, this work simply wouldn’t have been possible,” said Dr. Qu. 

Rally’s support helped bridge the gap between early-stage ideas and major federal funding. It also helped bring hope for better treatment strategies for one of the rarest and toughest childhood leukemias. 

This is why early-stage research funding matters.

This is the power of philanthropic seed investing.

This is Rally.