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Genetically Modified, Cord Blood Derived Natural Killer Cell Therapy for High Grade Pediatric Brain Tumors

Children’s Research Institute
Conrad Russell Cruz, MD, PhD
Grant Type: 
'A' Award Grants
Year Awarded: 
Type of Childhood Cancer: 
Brain Tumors, Glioma
Project Description: 


Certain brain cancers in children do not respond well to current treatment options. Newer therapies, like immunotherapy - which uses the body's own defenses (the immune system) as a "drug" for diseases - are particularly promising. However, these therapies are not readily applicable in the brain cancer setting. Brain cancers do not have any single identifying mark that the immune system can use to differentiate it from healthy organs, and the cancers themselves maintain an environment that is harmful to immune cells. Therefore to be useful, immunotherapies need to reliably differentiate brain cancer from the rest of the body and remain functional in hostile surroundings.

Project Goal

Among immune cells, a certain group called natural killer cells (NK cells) distinguish self from “nonself” as a means of detecting their potential targets. We believe that if we use "nonself" NK cells, we can potentially recognize tumors. On another level, NK cells also need a "go" signal before they kill their target, such signals are present in distressed cells like cancer but not in healthy cells. We propose using cord blood as a source of NK cells because of the ready availability of multiple cord blood banks around the world, and we further propose to arm these NK cells so that they can withstand the harmful effects of the cancer environment. This work will pave the way for the use of these novel cell therapies for brain cancer in children.

"Research support is critical to any beginning investigator, and I feel especially privileged that a foundation as respected and as inspiring as Alex’s Lemonade Stand has chosen my proposal as one if its award recipients. I have met successful scientists who have grown as investigators through the help of ALSF, and it is my hope that I follow their great examples."  – Russell Cruz