Experimental Treatment for Diffuse Intrinsic Pontine Glioma Earns Rare Pediatric Disease Designation

According to a story from BioSpace, Ziopharma Oncology, Inc. has recently announced that its investigational therapy Ad-RTS-hIL-12 with veledimex (also called Controlled IL-12) has been given Rare Pediatric Disease designation from the US Food and Drug Administration (FDA). This drug is being developed as a potential treatment for a rare brain tumor called diffuse intrinsic pontine glioma (DIPG).

About Diffuse Intrinsic Pontine Glioma

Diffuse intrinsic pontine glioma is a very rare brain tumor that is known for its poor response for treatment. It appears in the pons, the central area of the brain stem. Its locations makes the tumor inoperable via surgery. Unfortunately, the cause of this tumor remains unknown, and there are few known definitive risk factors; mutations of H3K27M are implicated in brain tumors in children. Symptoms of diffuse intrinsic pontine glioma include vision loss, difficulty swallowing, difficulty breathing, and problems with speech. The normal treatment approach for this tumor is radiation therapy for a period of six weeks; surgery is rarely possible, and the effectiveness of chemotherapy is unclear. Like other brain tumors, this one is difficult to treat as many drugs cannot cross the blood-brain barrier. The tumor almost always relapses after treatment, and five year survival rate is less than one percent. To learn more about diffuse intrinsic pontine glioma, click here.

About Rare Pediatric Disease Designation

Rare Pediatric Disease designation is reserved for drugs that are being developed to treat rare diseases that mostly affect those age 18 or younger. A rare disease is defined as any that affects less than 200,000 patients in the US. This designation is typically only for diseases that are considered serious or life-threatening. The recipient of this designation is eligible for a Priority Review voucher if the drug in question is approved. This can be used to shorten the review period for a future product or can be put up for sale or traded to a different company.

About Controlled IL-12

Controlled IL-12 is an experimental gene therapy that is intended to modulate the production of IL-12, which plays a central role in regulating immune system activity. A body of previous data has established its potential impacts on tumors and it is currently being investigated in a phase 1/2 clinical trial.

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