Alex's Lemonade Stand Foundation for Childhood Cancer

Background

The stimulator of interferon genes (STING) pathway is known to be critical for generating immune responses to tumors. But the role of STING activation has not yet been investigated in neuroblastoma (NB). Therefore, there is a unique opportunity to investigate the efficacy of STING-activating therapeutics as a novel treatment for NB.

Background

Malignant rhabdoid tumor, or MRT, is a rare but devastating childhood cancer. Most children diagnosed with MRT are under the age of two, and most will die from the disease despite intensive surgical, radiological, and chemotherapeutic interventions. A sad reality of MRT is that it is such a rare cancer that drug companies have little interest in developing new ways to treat the disease, meaning that a dismal prognosis is all we can hope to offer the two dozen children who are diagnosed with MRT in the United States every year.

The Vanderbilt Childhood Cancer program is a part of both the Monroe Carell Jr. Children's Hospital at Vanderbilt and the Vanderbilt-Ingram Cancer Center, an NCI-designated Comprehensive Cancer Center each of which were recognized for their excellence in the most recent U.S. News & World Report rankings [1, 2].

Rhabdomyosarcoma (RMS), an aggressive childhood cancer, is the most common soft tissue malignancy in children, accounting for 5-10% of all pediatric tumors. The most common form of RMS is the embryonal subtype (eRMS), representing nearly two-thirds of RMSs. RMS is thought to be initiated from muscle stem cells because the tumor expresses skeletal muscle markers. During the analysis of transgenic mice expressing activated Shh signaling in the brain, we serendipitously discovered that these mice exhibited RMS with 100% incidence.