A phase 1 trial of a treatment for DIPG, or diffuse intrinsic pontine glioma, looks promising. DIPG is an aggressive type of childhood cancer that forms in the brainstem. With a median survival rate of 8-11 months, DIPG is one of the deadliest childhood brain cancers.
The trial, which was led by Mark Souweidane, MAC member, and Zhiping Zhou, a former IronMatt grant recipient, was partially funded by IronMatt. Results were published in the June 2018 issue of Lancet Oncology.
In his grant application, Dr. Zhou wrote:
“Diffuse intrinsic pontine glioma (DIPG), the deadliest of childhood brain cancers, is located in the brain stem, which is the center that controls basic life functions such as breathing, heart beat, blood pressure, swallowing, etc. DIPG has no known cure. Nearly no children with this cancer will survive beyond 1-2 years following diagnosis with current standard therapy.
“Recently, two key signals driving tumor growth in this disease have been identified. Experimental therapies targeting these two overactive signals will be tested in this proposed study.
“To overcome the obstacle that drugs do not get into the tumor, we will deliver the drugs directly into the tumor using a technique called convection enhanced delivery (CED). By using combinations of drugs, we expect to slow or even stop growth of the tumor.
“Additional therapeutic targets will also be screened for using a modern technology called microarray analysis. We expect new therapeutic targets will be found. These efforts will eventually improve the clinical outcomes of patients with DIPG.”
Twenty-eight children were enrolled in the trial, with 25 evaluable for the primary endpoint. No treatment-related grade 4 adverse events or deaths occurred.
Results showed that convection-enhanced delivery in the brainstem of children with diffuse intrinsic pontine glioma who have previously received radiation therapy seems to be a rational and safe therapeutic strategy.
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