Neuroblastoma is the most common tumor of early childhood outside of the brain. Despite efforts to improve chemotherapy and radiation treatment, few patients with advanced diseases are cured or even achieve long-term survival. The completion of the human genome project now offers new opportunities to understand neuroblastoma at the genetic level. Such understanding in turn could lead to the development of novel therapies.

The first goal of cancer genetics research is to understand why children develop cancer and to identify if specific families or children have an even higher risk of developing cancer. This information impacts the child’s cancer care and determines who else in the family may need increased cancer surveillance to try and detect cancer at its earliest stage. Many cancer susceptibility genes also play a major role in cancer in the general population. Thus, the larger goal of cancer genetics research is to improve our understanding of cancer cells and to identify new targets for treatment.

Project Update - Breakthrough Therapy Status by the FDA,

One of ALSF’s early Innovation Grantees, Dr. Matthias Gromeier, has had his clinical trial designated as a breakthrough therapy by the FDA.  Dr. Gromeier and his team used a modified version of the polio virus  to attack glioblastoma, a deadly form of brain cancer.  Breakthrough status will allow hundreds of patients access to this treatment immediately.  His project was featured on 60 Minutes in May 2016. 

Update - 6/14:
A paper based on this research was published in Oncogene:

DeWitt J, Ochoa, V, Urschitz J, Elston M, Moisyadi S, and Nishi R. 2014. Constitutively active TrkB confers an aggressive transformed phenotype in a normal neural crest derived cell line. Oncogene 33(8):977-85.

Project Goal
This study determined the molecular characteristics and clinical significance of amplification of the 13q31 chromosomal region in alveolar rhabdomyosarcoma (ARMS), an aggressive pediatric cancer with frequent PAX3-FOXO1 and PAX7-FOXO1 gene fusions.