Academia and industry efforts offer renewed hope for rare pediatric brain tumor therapy

Diffuse intrinsic pontine glioma (DIPG) is an extremely rare pediatric brain tumor with a dismal prognosis. On average, less than 50% of patients are expected to be alive a year after diagnosis.

GlobalData notes that the attrition rate of drugs for gliomas, which include DIPG, is high compared to other cancers, and there is only a 6% chance of a glioma drug transitioning from Phase II clinical trials to an approved therapy. The data and analytics company notes that this environment has led to there being no US Food and Drug Administration or the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) approved drugs for DIPG. However, innovative approaches to generate pipeline candidates, including the use of artificial intelligence (AI), may give new therapies a chance of approval.

“DIPG is a true orphan indication for which accelerated development programs and special designations are greatly needed. It could be a big opportunity, too. Rare disease drugs routinely command a $250,000-$350,000 per-year price tag in the US, which, in the case of DIPG, could easily result in global peak sales in the $70 million-$95 million range,” noted GlobalData’s senior oncology analyst Sakis Palioras.