Improving natural product research translation: from source to clinical trial

Barbara C. Sorkin; Adam J. Kuszak; Gregory Bloss; Naomi K. Fukagawa; F. Hoffmann; Mahtab Jafari; Bruce Barrett; Paula N. Brown; Frederic D. Bushman; Steven J. Casper; Floyd H. Chilton; Christopher S. Coffey; Mario G. Ferruzzi; D. Craig Hopp; Mairead Kiely; Daniël Lakens; John B. MacMilan; David O. Meltzer; Marco Pahor; Jeffrey Paul; Kathleen Pritchett-Corning; Sara K. Quinney; Barbara Rehermann; Kenneth D.R. Setchell; Nisha S. Sipes; Jacqueline M. Stephens; D. Lansing Taylor; Hervé Tiriac; M.A. Walters; Dan Xi; Giovanna Zappalá; G.F. Pauli

doi:10.1096/fj.201902143R

Improving natural product research translation: from source to clinical trial

Barbara C. Sorkin (Corresponding author), Adam J. Kuszak, Gregory Bloss, Naomi K. Fukagawa, F. Hoffmann, Mahtab Jafari, Bruce Barrett, Paula N. Brown, Frederic D. Bushman, Steven J. Casper, Floyd H. Chilton, Christopher S. Coffey, Mario G. Ferruzzi, D. Craig Hopp, Mairead Kiely, Daniël Lakens, John B. MacMilan, David O. Meltzer, Marco Pahor, Jeffrey PaulKathleen Pritchett-Corning, Sara K. Quinney, Barbara Rehermann, Kenneth D.R. Setchell, Nisha S. Sipes, Jacqueline M. Stephens, D. Lansing Taylor, Hervé Tiriac, M.A. Walters, Dan Xi, Giovanna Zappalá, G.F. Pauli

Human Technology Interaction

Research output: Contribution to journal › Article › Academic › peer-review

57 Citations (Scopus)

Abstract

While great interest in health effects of natural product (NP) including dietary supplements and foods 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. Natural product clinical trials 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.

Original language	English
Pages (from-to)	41-65
Number of pages	25
Journal	The FASEB Journal
Volume	34
Issue number	1
DOIs	https://doi.org/10.1096/fj.201902143R
Publication status	Published - Jan 2020

Funding

The authors thank Paul M. Coates, Joseph M. Betz, and F. Ellis McKenzie for inspiration and support, Wen Chen for help with the initial concept, Wendy P. O’Meara for insightful editing, Sharon Ross and Harold Seifried of the NIH National Cancer Institute for invaluable input into the workshop, Miriam Sander for workshop transcription and notes, Cara Lynch, James Hubley, Katy Fuller, and Joyce Merkel for outstanding technical support. This project was supported by staff and funding from the NIH’s Office of Dietary Supplements, National Cancer Institute, National Center for Complementary and Integrative Health, National Institute on Aging, National Institute on Alcohol Abuse and Alcoholism, and National Institute of Environmental Health Sciences. Support was also provided by staff from the Food and Drug Administration and the US Department of Agriculture. The authors thank Paul M. Coates, Joseph M. Betz, and F. Ellis McKenzie for inspiration and support, Wen Chen for help with the initial concept, Wendy P. O?Meara for insightful editing, Sharon Ross and Harold Seifried of the NIH National Cancer Institute for invaluable input into the workshop, Miriam Sander for workshop transcription and notes, Cara Lynch, James Hubley, Katy Fuller, and Joyce Merkel for outstanding technical support. This project was supported by staff and funding from the NIH?s Office of Dietary Supplements, National Cancer Institute, National Center for Complementary and Integrative Health, National Institute on Aging, National Institute on Alcohol Abuse and Alcoholism, and National Institute of Environmental Health Sciences. Support was also provided by staff from the Food and Drug Administration and the US Department of Agriculture.

Funders	Funder number
National Institutes of Health
U.S. Food and Drug Administration (USDA)
National Institute on Aging	P30AG028740
National Cancer Institute
U.S. Department of Agriculture

Keywords

clinical predictive validity
dietary supplements
model systems
rigor and replicability
value of information

UN SDGs

This output contributes to the following UN Sustainable Development Goals (SDGs)

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10.1096/fj.201902143R

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Sorkin, B. C., Kuszak, A. J., Bloss, G., Fukagawa, N. K., Hoffmann, F., Jafari, M., Barrett, B., Brown, P. N., Bushman, F. D., Casper, S. J., Chilton, F. H., Coffey, C. S., Ferruzzi, M. G., Hopp, D. C., Kiely, M., Lakens, D., MacMilan, J. B., Meltzer, D. O., Pahor, M., ... Pauli, G. F. (2020). Improving natural product research translation: from source to clinical trial. The FASEB Journal, 34(1), 41-65. https://doi.org/10.1096/fj.201902143R

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abstract = "While great interest in health effects of natural product (NP) including dietary supplements and foods 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. Natural product clinical trials 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.",

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author = "Sorkin, {Barbara C.} and Kuszak, {Adam J.} and Gregory Bloss and Fukagawa, {Naomi K.} and F. Hoffmann and Mahtab Jafari and Bruce Barrett and Brown, {Paula N.} and Bushman, {Frederic D.} and Casper, {Steven J.} and Chilton, {Floyd H.} and Coffey, {Christopher S.} and Ferruzzi, {Mario G.} and Hopp, {D. Craig} and Mairead Kiely and Dani{\"e}l Lakens and MacMilan, {John B.} and Meltzer, {David O.} and Marco Pahor and Jeffrey Paul and Kathleen Pritchett-Corning and Quinney, {Sara K.} and Barbara Rehermann and Setchell, {Kenneth D.R.} and Sipes, {Nisha S.} and Stephens, {Jacqueline M.} and Taylor, {D. Lansing} and Herv{\'e} Tiriac and M.A. Walters and Dan Xi and Giovanna Zappal{\'a} and G.F. Pauli",

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Sorkin, BC, Kuszak, AJ, Bloss, G, Fukagawa, NK, Hoffmann, F, Jafari, M, Barrett, B, Brown, PN, Bushman, FD, Casper, SJ, Chilton, FH, Coffey, CS, Ferruzzi, MG, Hopp, DC, Kiely, M, Lakens, D, MacMilan, JB, Meltzer, DO, Pahor, M, Paul, J, Pritchett-Corning, K, Quinney, SK, Rehermann, B, Setchell, KDR, Sipes, NS, Stephens, JM, Taylor, DL, Tiriac, H, Walters, MA, Xi, D, Zappalá, G & Pauli, GF 2020, 'Improving natural product research translation: from source to clinical trial', The FASEB Journal, vol. 34, no. 1, pp. 41-65. https://doi.org/10.1096/fj.201902143R

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AU - Sorkin, Barbara C.

AU - Kuszak, Adam J.

AU - Bloss, Gregory

AU - Fukagawa, Naomi K.

AU - Hoffmann, F.

AU - Jafari, Mahtab

AU - Barrett, Bruce

AU - Brown, Paula N.

AU - Bushman, Frederic D.

AU - Casper, Steven J.

AU - Chilton, Floyd H.

AU - Coffey, Christopher S.

AU - Ferruzzi, Mario G.

AU - Hopp, D. Craig

AU - Kiely, Mairead

AU - Lakens, Daniël

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AU - Meltzer, David O.

AU - Pahor, Marco

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AU - Quinney, Sara K.

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AU - Setchell, Kenneth D.R.

AU - Sipes, Nisha S.

AU - Stephens, Jacqueline M.

AU - Taylor, D. Lansing

AU - Tiriac, Hervé

AU - Walters, M.A.

AU - Xi, Dan

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AU - Pauli, G.F.

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Improving natural product research translation: from source to clinical trial

Abstract

Funding

Keywords

UN SDGs

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