Long-term High-quality Survival with Single-agent Mifepristone Treatment Despite Advanced Cancer

Abstract

These cases helped influence the US Food and Drug Association in granting an investigator-initiated investigational new drug study on advanced non-small cell lung cancer.

Mifepristone Extends Both Length and Quality of Life in a Patient With Advanced Non-small Cell Lung Cancer that Has Progressed Despite Chemotherapy and a Check-point Inhibitor

“…Most previous anecdotal cases were treated with a 200 mg dosage of mifepristone (1)(2)(3)(4). The drug was very well tolerated consistent with extensive data from patients that used it for the treatment of meningiomas (26).…”

“…Thus, it seems highly likely that the complete remission for 5.5 years (and probably longer had she not had a myocardial infarction) was related to the mifepristone treatment, rather than to spontaneous remission. This case shows that mifepristone can correct the syndrome of inappropriate antidiuretic hormone by causing tumor regression, since no other treatment for this condition was given (4).…”

“…The chest x-ray and CT scan showed multiple lung nodules. She refused surgical biopsy, but the clinical presentation suggested small cell lung cancer (4). She had no previous treatment for her lung cancer, i.e., neither surgery, radiation therapy, chemotherapy, or immunotherapy (4).…”

“…The progesterone receptor modulator, mifepristone, has been found to improve quality of life, and extend the length of life when used as a single agent treatment in patients with a variety of advanced metastatic cancers that have progressed despite standard therapy, or were not candidates for chemo or immunotherapy, even when the tumors were devoid of the classic nuclear progesterone receptor (1)(2)(3)(4).…”

“…Based on anecdotal evidence showing considerable palliation and increased longevity when used in treating a variety of cancers, and on controlled studies in spontaneous murine cancers, the United States Food and Drug Administration (FDA) approved an investigator-initiated investigative new drug approval (IND), to treat up to 40 patients with stage IIIB or IV non-small cell lung cancer, that had progressed despite a minimum of two chemotherapy or immunotherapy regimens ("A phase II study of treatment with oral mifepristone as salvage therapy in patients with advanced or metastatic non-small cell lung cancer who have failed two or more previous chemotherapy regimens") (www.clinicaltrials.gov) (1)(2)(3)(4)(12)(13)(14). The study was approved by the Chesapeake Institutional Review Board (approval number Pr000011306).…”