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.