Alex's Lemonade Stand Foundation for Childhood Cancer

Treatment options for children with fusion-driven Alveolar Rhabdomyosarcoma (aRMS) are very limited, and there are no approved new targeted therapy approaches for these children. In fact, there have been no substantial improvements in treatments for these children in over two decades. I have identified CDK8 as a candidate therapeutic target in aRMS and interaction partner of PAX3-FOXO1, the abnormal gene involved in the initiation and maintenance of this disease.

Mentor Name: Pratiti Bandopadhayay

Brain tumors are the leading cause of cancer related morbidity and death in children. Therapeutic approaches that target the mutations that make tumors grow stand to increase the effectiveness of our treatments. The goal of this project is to develop strategies that target mutations in a gene called FGFR1 which we have found to occur across pediatric gliomas.

The goal of this project is to test a novel strategy for disrupting the effects of MYCN amplification, which is seen in over 50% of high risk neuroblastoma patients. Although the association of MYCN amplification with a poor outcome is well established, attempts to directly target MYCN have been challenging. Therefore, we propose to tackle one of the consequences of amplified MYCN that enables abnormal cell growth, that of increased protein expression (translation). Proteins are the “doers” in the cell and originate from RNA.

Mentor Name: Steve DuBois

Mentor Name: Pratiti Bandopadhayay

During my summer internship, I will study how the different effects each histone mutation has on DNA repair. To do this, we will use model systems that the lab has developed to introduce histone mutations into normal cells. I will use a range of molecular biology methods to study the effect of these mutations on normal cells and the subsequent changes in normal cellular processes, including different DNA repair responses.