Principal Investigator: Elizabeth Maher, MD, PhD, University of Texas Southwestern Medical Center, Dallas, Texas

Pilocytic astrocytomas (PA), the most common childhood brain tumor, is classified into 2 groups, those that are resected and do not recur (non-progressors), and those that recur early and need further treatment, progressors. There is little that distinguishes these patients at diagnosis, a fact which leads to uncertainty regarding the need for further treatment on the part of the physician and uneasiness and worry about the long-term outcome for their children on the part of the parent.

A large part of pediatric PA patients has been followed at UT Southwestern Medical Center over the past 25 years and a database of clinical information linked to a tissue repository containing tumor samples from most of the patients has been amassed. Over the past year we have initiated a pilot study of 32 patients (16 progressors, 16 nonprogressors) to generate comprehensive molecular profiles of their tumors with the goal of identifying specific distinguishing genetic altercations in these groups.

Exciting preliminary data shows distinct differences, lending confidence to the assertion that the studies proposed here will identify novel markers that can predict progressive disease and, equally important, identify novel targets for new drug development for this important childhood disease.