Diffuse intrinsic pontine glioma (DIPG) is an incurable disease with a median 
overall survival of 10 months. Immune modulating antibodies have recently 
emerged as a highly promising treatment modality in multiple cancer types. We 
present results from the first study to evaluate the immune modulating antibody 
MDV9300 (pidilizumab) in pediatric patients with DIPG. All patients aged 3 years 
and older, diagnosed with DIPG between February 2014 and June 2015 in Israel, 
were offered to participate in the study. Enrolled patients were started on 
biweekly 6 mg/kg MDV9300 after radiation completion. Treatment was continued 
until disease progression on imaging. Patients were followed biweekly for the 
occurrence of neurological deficit toxicities and side effects. Secondary 
endpoints were event free survival and overall survival. Of 13 children 
diagnosed with DIPG during the study period, nine were enrolled in the study. 
The patients underwent radiotherapy and none had chemotherapy. A total of 83 
cycles of MDV9300 (range 2-16) were applied. The main side effects were 
neutropenia (CTCAE grade 1-3), mild to moderate fatigue, and acute elevation of 
blood pressure. Four patients died within 1 year of the diagnosis, another three 
died within 2 years and two children are still alive nearly 30 months from 
diagnosis, with stable disease. The median event free survival is 9.3 months 
(range 6.8-24) and the median overall survival is 15.6 months (range 6.9-28). 
Preliminary results demonstrate that MDV9300 treatment is safe and may be 
effective in the treatment of children with DIPG.