Research and Development of Atractylodes lancea (Thunb) DC. as a Promising Candidate for Cholangiocarcinoma Chemotherapeutics.

Treatment and control of cholangiocarcinoma (CCA): the bile duct cancer is limited by the lack of effective chemotherapeutic drugs and alternative drugs are needed, particularly those from natural sources. This article reviews steps of research and development of Atractylodes lancea (Thunb) DC. (AL) as potential candidate for CCA chemotherapy, with adoption of the reverse pharmacology approach. Major steps include (1) reviewing of existing information on its phytochemistry and pharmacological properties, (2) screening of its activities against CCA, (3) standardization of AL, (4) nonclinical studies to evaluate anti-CCA activities, (5) phytochemistry and standardization of AL extract, (6) development of oral pharmaceutical formulation of standardized AL extract, and (7) toxicity testing of oral pharmaceutical formulation of standardized AL extract. Results from a series of our study confirm anti-CCA potential and safety profiles of both the crude extract and the finished product (oral pharmaceutical formulation of the standardized AL extract). Phases I and II clinical trials of the product to confirm tolerability and efficacy in healthy subjects and patients with advanced stage CCA will be carried out soon.