RE: Second Orphan Drug Designation request26 Apr 2023 10:54
Motivation for this request was generated from two landmark pre-clinical studies completed by Dr. Mona Al-Gizawiy, PhD in the laboratory of Dr. Kathleen Schmainda, PhD at MCW. These studies demonstrated similar remarkable results of GaM in atypical teratoid/rhabdoid tumor (ATRT) and GBM in children as it did in the pre-clinical study for adult GBM. Current treatment protocols for pediatric brain tumors subject children to the same toxic, invasive, and harsh treatment protocols used to treat adult brain tumors.