New drug for TGCT elevates patientreported outcomes in trials

Tynosynovial giant cell tumor, or TGCT, is a rare disease, affecting three to four people per 100,000 per year. It’s also considered an “orphan disease,” due to the high cost of developing treatments for the small population that might benefit. But when the discovery of a signaling protein called CSF1 - a pathway for tumor growth investigated as a potential cancer treatment – showed promise for treating TGCT, it created the possibility for a new oral therapy for this painful inflammatory condition that was once only treated surgically.