Alex's Lemonade Stand Foundation for Childhood Cancer

Ependymoma is a lethal pediatric brain tumor that is still treated by surgery and radiation. When tumors recur, which is often, patients have limited treatment options and often receive additional radiation further worsening neurologic side effects. Novel, effective and less-toxic therapies are desperately needed for these patients. Ependymomas that arise in the forebrain are frequently driven by an oncogene that joins two proteins, C11ORF95 and RELA (denoted as C11ORF95-RELA fusion).

Childhood cancers are caused by genetic mutations that lead to changes in proteins that are essential for normal human development. The protein MYCN is the cause of many of the most aggressive pediatric cancers, including high-risk neuroblastoma and medulloblastoma. One of the holy grails of pediatric cancer drug development is finding a way to directly target MYCN in patient tumors, but this has been elusive. To address this major unmet need, we have developed an international team of scientists with complementary expertise to attack this problem with innovative new technologies.

Diffuse midline glioma (DMG) is an uncurable pediatric brain tumor with extremely limited treatment options. The Krenciute Lab at St. Jude is working to develop new types of treatments known as immunotherapy for children with DMGs, in which the patient’s own immune system is harnessed to fight the cancer. Specifically, we are interested in developing chimeric antigen receptor (CAR) T cells, a form of immunotherapy where a patient’s T cells are re-engineered to better target and attack the cancer cell.

Children with solid tumors have poor overall survival. Further, the treatments used for these diseases are very toxic. One particularly difficult to treat pediatric solid tumor is called neuroblastoma (NB). Children with neuroblastoma are treated with very toxic therapies, and even so, many of them will not be cured. In addition, while large-scale studies have identified the specific proteins that NB cells depend on for growth, these proteins are especially hard to target with traditional drugs. This limits our ability to treat NB effectively.