Rare Disease Report

Glioblastoma Treatment Gets FDA Orphan Drug Designation

AUGUST 18, 2017
Mathew Shanley
Curtana Pharmaceuticals recently announced the U.S. Food and Drug Administration (FDA) has granted CT-179 an Orphan Drug Designation to treat gliomas, including glioblastoma in adults and brain tumors in children.

In July, glioblastoma made headlines following an announcement from U.S. Senator John McCain, who was diagnosed with the antagonistic and fatal cancer type.

Glioblastoma is the most aggressive cancer type, and typically begins in the brain. While the cause of many cases is unclear, risk factors include genetic disorders like neurofibromatosis and prior chemotherapy or radiation. Initial signs include headaches, personality changes, nausea, and symptoms closely resembling that of a stroke.

"We are pleased that the FDA has recognized the potential for CT-179 to provide a significant therapeutic benefit to patients with (glioblastoma)," said Dr. Gregory Stein, the company’s CEO, in a statement. "GBM (glioblastoma multiforme) is a devastating disease with a generally very poor prognosis even with the best available care. CT-179, projected to enter the clinic in the first half of 2018, has been shown to significantly prolong survival in relevant animal models, especially when combined with the standard of care treatments, temozolomide and radiation therapy."

CT-179 is a highly potent inhibitor of Olig2, which is highly expressed in all diffuse gliomas and is found in almost all glioblastoma cells that are positive for the tumorigenic CD133 cancer stem cell marker. When tested in animal models, it was proven that the drug intelligently avoids normal cells and substantially reduces cancer cell migration into normal brain tissue.

To be taken orally, CT-179 readily crosses the blood-brain barrier, and achieves significantly high concentrations in the brain, considerably prolonging survival. When combined with standard of care temozolomide and radiation, the drug noticeably prevents tumor growth in comparison to either treatment.

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