Targeted Therapy With Gefitinib in Patients With USP8-mutated Cushing's Disease
Status:
Unknown status
Trial end date:
1969-12-31
Target enrollment:
Participant gender:
Summary
The USP8 gene and its downstream target, epidermal growth factor receptor (EGFR), is a
potential therapeutic target of Cushing disease. The EGFR inhibitor, Gefitinib, has been
shown to reduce the production of ACTH both in vitro and in vivo, especially in USP8-mutated
corticotrophin adenomas. The investigators hypothesize that Gefitinib will suppress pituitary
corticotroph tumor ACTH production and normalize urinary free cortisol levels in patients
with USP8-mutated Cushing's disease. Gefitinib is an FDA approved drug used to treat
non-small cell lung cancer. However, in this study, the drug will be used to treat
corticotrophin adenoma.