Osteosarcoma is the most common primary bone tumor in childhood. The survival rate remains dismal, mainly due to ineffective therapeutic approaches for the relapsed/metastatic patients. One major obstacle of treating osteosarcoma is lack of suitable preclinical models. Dr. He's studies have established the first cultured osteosarcoma tissue model (an organoid). Dr. He aims to establish the first biobank of osteosarcoma organoids from patients as an open resource for the field, and utilize this organoid biobank to evaluate a novel class of therapeutics targeting key signaling pathways in osteosarcoma cells. This study will provide a powerful platform for predicting clinical treatment responses and developing new therapeutics for treating osteosarcoma.

Based on progress to date, Dr. Lian was awarded new grants in 2018 and 2019 to fund additional years of this International Scholar grant. Based on progress to date, Dr. Lian was awarded a new grant in 2018 to fund an additional year of this International Scholar grant. MYCN-driven neuroblastoma accounts for about 30% of neuroblastomas and is associated with an extremely poor prognosis. Casein Kinase 2 (CK2) is an enzyme that is currently in clinical trials to treat multiple cancers. However, its efficacy on MYCN-driven neuroblastoma remains unknown. Dr. Lian's research aims to test if CK2 inhibition can serve as a new strategy to treat MYCN-driven neuroblastoma.

A portion of this grant was named for The Amanda Rozman Pediatric Cancer Research Fund, a St. Baldrick's Hero Fund created in memory of Amanda Rozman and honors her courageous battle with neuroblastoma by funding promising new treatments and clinical trials in the area of translational research.

Based on progress to date, Dr. Zhang was awarded new grants in 2017 and 2018 to fund additional years of this International Scholar award. Though cure rates have improved dramatically, geographic inequities in childhood acute lymphoblastic leukemia (ALL) therapy remain evident between developed vs. low-middle income countries. In China, about 7,700 newly-diagnosed children with ALL every year receive therapy thanks to the coverage provided by a special national health insurance scheme established in 2010. With the drastic increase in access to clinical care, the challenge is now shifting from remedying the inability to pay for ALL therapy to delivering better therapy and improving outcome. Dr. Zhang is working to bring improved therapy to China. This represents an unprecedented opportunity for translational research of childhood ALL in China, with potential impacts for a large number of patients.