OMRF brain tumor drug gets FDA nod
A “game changing” drug for treating two different types of cancer has been given the green light by the U.S. Food and Drug Administration.
OKN- 007, an investigation drug discovered at the Oklahoma Medical Research Foundation, was awarded “Rare Pediatric Disease Designation” by the FDA for treatment against diffuse intrinsic pontine glioma and “Orphan Drug Designation” for treatment of malignant glioma.
Diffuse intrinsic pontine gl io ma is a fast-growing pediatric cancer that starts in the brain stem and currently has no effective treatments, according to Dr. R heal Towner, an OMRF scientist.
It is one of several sub-categories of malignant gliomas, deadly cancers of the brain and spinal cord.
According to the National Brain Tumor Society, approximately 2 6, 000 Americans will be diagnosed with primary malignant brain tumors this year. Of those, DIPG.org reports that up to 300 will be cases of DIPG.
“It's a huge game changer,” said Towner, who discovered OKN-007 along with fellow OMRF scientist Robert Floyd, Ph.D. “We are showing that it's much more effective than any current standard of care and that it'll be a major player for therapeutic options. For DI PG there were no good options, now there is some promise.”
In trials of the drugs in mice, Towner said scientists were able to demonstrate that the drug would increase animal survival, decrease tumor size, decrease tumor vascularization back to normal and reduce aggressive mutation.
Oblato acquired all rights to OKN-007 from OMRF in 2018 and will develop the drug.
The company is currently testing the investigational drugi na Phase 2 c li nical study of 56 patients suffering from recurrent gl io blast om a, the most aggressive form of glioma, and will begin clinical trials in DIPG patients in 2021.
“OMRF and Oblato are committed to a single goal: helping patients overcome these life-threatening illnesses,” said OMRF Director of Technology Ventures Andrew Westmuckett, Ph.D. “Our hope is that OKN-007 can transform the therapeutic landscape.”