Improving Natural Product Research Translation: from Source to Clinical Trial

Authors: SORKIN, BARBARA - National Institutes Of Health (NIH); KUSZAK, ADAM - National Institutes Of Health (NIH); Fukagawa, Naomi; HOFFMAN, FREDDIE ANN - National Cancer Institute (NCI, NIH); JAFARI, MAHTAB - University Of California; BARRETT, BRUCE - University Of Wisconsin; BROWN, PAULA - British Columbia Institute Of Technology; BUSHMAN, FREDRIC - University Of Pennsylvania; CASPER, STEVEN - Food And Drug Administration(FDA); CHILTON, FLOYD - University Of Arizona; COFEY, CHRISTOPHER - University Of Iowa; FERRUZZI, MARIO - North Carolina State University; HOPP, D. CRAIG - National Institute Of Health (INSA); KIELY, MAIREAD - University College Cork; LAKENS, DANIEL - Eindhoven University Of Technology; MACMILAN, JOHN - University Of California; MELTZER, DAVID - University Of Chicago; PAHOR, MARCO - University Of Florida; JEFFREY, PAUL - Drexel University; PRITCHETT-CORNING, KATHLEEN - Harvard University; QUINNEY, SARA - Indiana University; REHERMANN, BARBARA - National Institute Of Diabetes And Digestive And Kidney Diseases; SETCHELL, KENNETH - Cincinnati Children'S Research Hospital; SIPES, NISHA - National Institutes Of Health (NIH); STEPHENS, JACQUELINE - Louisiana State University; TAYLOR, D. LANSING - University Of Pittsburgh; TIRIAC, HERVE - University Of California; WATERS, MICHAEL - University Of Minnesota; XI, DAN - National Cancer Institute (NCI, NIH); ZAPPALA, GIOVANNA - National Institute On Aging (NIA, NIH); PAULI, GUIDO - University Of Illinois

Submitted to: Journal of Federation of American Societies for Experimental Biology
Publication Type: Peer Reviewed Journal
Publication Acceptance Date: 10/21/2019
Publication Date: N/A
Citation: N/A

Interpretive Summary: While great interest in health effects of natural product (NP) foods and dietary supplements persists, promising preclinical NP research is not consistently translating into actionable clinical trial (CT) outcomes. Generally considered the gold standard for assessing safety and efficacy, CTs, especially phase III CTs, are costly and require rigorous planning to optimize the value of the information obtained. More effective bridging from NP research to CT was the goal of a September, 2018 transdisciplinary workshop. Participants emphasized that replicability and likelihood of successful translation depend on rigor in experimental design, interpretation, and reporting across the continuum of NP research. Discussions spanned good practices for: NP characterization and quality control; use and interpretation of models (computational through in vivo) with strong clinical predictive validity; controls for experimental artefacts, especially for in vitro interrogation of bioactivity and mechanisms of action; rigorous assessment and interpretation of prior research; transparency in all reporting; and prioritization of research questions. NPCTs prioritized based on rigorous, convergent supporting data and current public health needs are most likely to be informative and ultimately affect public health. Thoughtful, coordinated implementation of these practices should enhance the knowledge gained from future NP research.

Technical Abstract: While great interest in health effects of natural product (NP) foods and dietary supplements persists, promising preclinical NP research is not consistently translating into actionable clinical trial (CT) outcomes. Generally considered the gold standard for assessing safety and efficacy, CTs, especially phase III CTs, are costly and require rigorous planning to optimize the value of the information obtained. More effective bridging from NP research to CT was the goal of a September, 2018 transdisciplinary workshop. Participants emphasized that replicability and likelihood of successful translation depend on rigor in experimental design, interpretation, and reporting across the continuum of NP research. Discussions spanned good practices for: NP characterization and quality control; use and interpretation of models (computational through in vivo) with strong clinical predictive validity; controls for experimental artefacts, especially for in vitro interrogation of bioactivity and mechanisms of action; rigorous assessment and interpretation of prior research; transparency in all reporting; and prioritization of research questions. NPCTs prioritized based on rigorous, convergent supporting data and current public health needs are most likely to be informative and ultimately affect public health. Thoughtful, coordinated implementation of these practices should enhance the knowledge gained from future NP research.