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Title:Mechanism of action, potency and efficacy : considerations for cell therapies
Authors:ID Simon Jr., Carl G. (Author)
ID Bozenhardt, Erich H. (Author)
ID Celluzzi, Christina M. (Author)
ID Dobnik, David (Author)
ID Grant, Melanie L. (Author)
ID Lakshmipathy, Uma (Author)
ID Nebel, Thiana (Author)
Files:URL URL - Source URL, visit https://doi.org/10.1186/s12967-024-05179-7
 
.pdf PDF - Presentation file, download (4,26 MB)
MD5: 98DD6E0119EEFC3B0509163C71948613
 
Language:English
Typology:1.01 - Original Scientific Article
Organization:Logo NIB - National Institute of Biology
Abstract:One of the most challenging aspects of developing advanced cell therapy products (CTPs) is defining the mechanism of action (MOA), potency and efficacy of the product. This perspective examines these concepts and presents helpful ways to think about them through the lens of metrology. A logical framework for thinking about MOA, potency and efficacy is presented that is consistent with the existing regulatory guidelines, but also accommodates what has been learned from the 27 US FDA-approved CTPs. Available information regarding MOA, potency and efficacy for the 27 FDA-approved CTPs is reviewed to provide background and perspective. Potency process and efficacy process charts are introduced to clarify and illustrate the relationships between six key concepts: MOA, potency, potency test, efficacy, efficacy endpoint and efficacy endpoint test. Careful consideration of the meaning of these terms makes it easier to discuss the challenges of correlating potency test results with clinical outcomes and to understand how the relationships between the concepts can be misunderstood during development and clinical trials. Examples of how a product can be “potent but not efficacious” or “not potent but efficacious” are presented. Two example applications of the framework compare how MOA is assessed in cell cultures, animal models and human clinical trials and reveals the challenge of establishing MOA in humans. Lastly, important considerations for the development of potency tests for a CTP are discussed. These perspectives can help product developers set appropriate expectations for understanding a product’s MOA and potency, avoid unrealistic assumptions and improve communication among team members during the development of CTPs.
Keywords:cell therapy product, efficacy endpoint test, mechanism of action, potency test, metrology
Publication status:Published
Publication version:Version of Record
Publication date:02.05.2024
Year of publishing:2024
Number of pages:str. [1]-17
Numbering:Vol. 22, art. no. ǂ416
PID:20.500.12556/DiRROS-19009 New window
UDC:576
ISSN on article:1479-5876
DOI:10.1186/s12967-024-05179-7 New window
COBISS.SI-ID:196774659 New window
Note:Soavtorji: Erich H. Bozenhardt, Christina M. Celluzzi, David Dobnik, Melanie L. Grant, Uma Lakshmipathy, Thiana Nebel, Linda Peltier, Anthony Ratcliffe, James L. Sherley, Glyn N. Stacey, Rouzbeh R. Taghizadeh, Eddie H. P. Tan & Sandrine Vessillier; Nasl. z nasl. zaslona; Opis vira z dne 27. 5. 2024;
Publication date in DiRROS:27.05.2024
Views:121
Downloads:122
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Record is a part of a journal

Title:Journal of translational medicine
Shortened title:J. transl. med.
Publisher:BioMed Central
ISSN:1479-5876
COBISS.SI-ID:513978393 New window

Licences

License:CC BY 4.0, Creative Commons Attribution 4.0 International
Link:http://creativecommons.org/licenses/by/4.0/
Description:This is the standard Creative Commons license that gives others maximum freedom to do what they want with the work as long as they credit the author.

Secondary language

Language:Slovenian
Title:Mechanism of action, potency and efficacy: considerations for cell therapies
Keywords:celična terapija, učinkovitost, mehanizem delovanja, test moči, meroslovje


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