Date: 2016-06-16
Type of information: Presentation of results at a congress
phase: 1-2
Announcement: presentation of results at the International Symposium on Pediatric Neuro-Oncology (ISPNO)
Company: Hadassah Medical Organization (Israel) Medivation (USA - CA)
Product: pidilizumab
Action
mechanism: monoclonal antibody/immune checkpoint inhibitor. Pidilizumab is a humanized monoclonal antibody which belongs to a class of anticancer therapies that target the immune system. Cancer cells evade destruction by suppressing immune T-lymphocytes through activation of the PD-1 (programmed death-1) pathway. Pidilizumab binds the PD-1 protein on T lymphocytes and facilitates the T-cells' ability to target and destroy cancer cells.
Disease: diffuse intrinsic pontine glioma (DIPG)
Therapeutic area: Cancer - Oncology - Rare diseases
Country: Israel
Trial
details: Diffuse intrinsic pontine glioma is a rare and aggressive pediatric cancer which is responsible for the highest brain tumor mortality in children. Approximately 300-400 pediatric brain stem tumors are diagnosed per year in the United States, approximately 75%-80% of which are DIPGs. Children with DIPG experience a median overall survival between 9-12 months and a two-year survival rate of less than 10%. The disease is incurable with currently used treatments. One of the mechanisms through which tumor cells escape immune detection is activation of the inhibitory lymphocyte receptor PD-1 PD-1/PD-1 ligand interaction promotes tolerance of the immune system to a specific antigen. Lately, studies reported high levels of PD-1 expression in tumor infiltrating lymphocytes ,and strong expression of PD-ligand within multiple tumor types including high grade glioma. This clinical trial will assess assessing the anti PD-1 monoclonal antibody, pidilizumab in this population with extremely poor prognosis. (NCT01952769)
Latest
news: * On June 16, 2016, Medivation announced results from a Phase I/II study of pidilizumab that demonstrated potential clinical benefit in pediatric patients with diffuse intrinsic pontine glioma (DIPG). The study, which was exploratory in nature, was designed to assess the safety and tolerability of pidilizumab, as well as key clinical outcomes, such as event-free and overall survival, in this pediatric population. Preliminary data were presented in an oral symposium at the International Symposium on Pediatric Neuro-Oncology (ISPNO) by the study's lead investigator Iris Fried, M.D., Attending Physician, Pediatric Hemato-oncology, Hadassah Medical Center, Jerusalem, Israel. Data from nine pediatric patients with DIPG who were treated with pidilizumab following completion of standard radiation therapy were presented. The median age of the study population was 6.5 years (range: 3-19 years): eight patients had intermediate risk features and one patient had high risk features. The reported mean event-free and overall survival estimates were 12 and 15.6 months, respectively. Three patients with DIPG remained progression-free at 16.3, 22, and 24 months following diagnosis, with one patient experiencing a partial response. Adverse events of any grade reported in at least one treatment cycle include neutropenia, fatigue, loss of appetite, hypertension, nausea, and lymphopenia; only neutropenia and hypertension were reported as grade 3 adverse events. The study continues to enroll patients.
