FL118, drug candidate discovered at Roswell Park, granted FDA Orphan Drug status for pancreatic cancer

Feb. 27, 2024
Derived from tree bark, FL118 is based on a compound used in traditional Chinese medicine.

The U.S. Food and Drug Administration (FDA) has awarded Orphan Drug Designation to Canget BioTekpharma LLC for FL118, a drug candidate developed at Roswell Park Comprehensive Cancer Center, as a possible treatment for pancreatic cancer.

FL118, also known by its chemical name, 10,11-methylenedioxy-20(S)-camptothecin (10,11-MD-CPT), is one of only 206 pharmaceutical agents granted Orphan Drug Designation for the treatment of pancreatic cancer since the FDA initiated the incentive program in 1984. 

Discovered by a team of scientists led by Fengzhi Li, PhD, Associate Professor of Oncology in the Department of Pharmacology and Therapeutics at Roswell Park, FL118 has been shown in preclinical studies to eliminate both pancreatic and colorectal tumor cells by binding to DDX5, a powerful cancer-causing protein. It’s a small molecule derived from camptothecin, a component found in the bark and stem of a tree native to China — and used as a traditional Chinese remedy for centuries.

Research recently published by Dr. Li and colleagues in the American Chemical Society’s Journal of Medicinal Chemistry documents the synthesis, identification and characterization of new analogs of FL118 shown to have enhanced comparative antitumor activity. Read more in “From the Forest: Researchers at Roswell Park are Utilizing a Novel Derivative of Camptothecin, an Antitumor Agent Found in Chinese Tree Bark,” on Roswell Park’s CancerTalk blog.

Roswell Park Comprehensive Cancer Center release on Newswise