Resistance and Develop Improved Treatments for Relapsed/refractory Pediatric ALL
Childhood leukemia is one of the great success stories of the "War on Cancer." Over the last 4 decades, the outcomes for these children have risen to a current estimated 90% cure rate. Despite this, children with leukemia who relapse or do not respond to the standard treatments have little chance of survival. Complicating our search for improved treatments for these children is the fact that no method exists to predict the success of a new cancer drug prior to costly and potentially dangerous clinical trials. We have developed a method that allows us to study how a drug works in human cancer cells, by growing these cancer cells in mice. Several lines of evidence suggest this method may be able to predict how a drug will act in human patients. Using this method, we have demonstrated that a new drug, CGM097, is very effective at treating most kinds of acute lymphoblastic leukemia-the most common type of leukemia found in children. Even more important, this drug is able to improve survival from acute leukemias that were derived from children who relapsed on standard treatments.
To make this drug work even better, we will perform experiments to determine what changes occur in the leukemia cell to make it resistant to CGM097. We will identify other cancer drugs that can prevent these changes from occurring and work in combination with CGM097 to lengthen lifespan of children with leukemia who would otherwise die of this disease.
"Funding from Alex's Lemonade Stand Foundation will allow me to investigate new drugs to treat relapsed or therapy-resistant leukemia in children. I will focus on finding ways to combat difficult to treat cancers and identifying new ways to make existing treatments more effective. The experience gained through these studies will provide me with essential training to embark on a career as an independent cancer researcher." - Elizabeth Townsend, PhD