Biodistribution studies for cell therapy products: Current status and issues

Area of Science:

• Biomedical Science
• Pharmacology
• Regenerative Medicine

Background:

• Biodistribution (BD) studies are vital for assessing cell therapy products (CTPs) safety and efficacy.
• Regulatory requirements, implementation, and analytical methods for BD studies are critical for CTP development.

Purpose of the Study:

• To survey international and Japanese trends in BD studies for CTPs.
• To provide a resource for implementing effective BD studies in CTP development.

Main Methods:

• Comparative analysis of global and Japanese regulatory guidelines for BD studies.
• Case study review of BD studies using databases in cell therapy.
• Examination of quantitative polymerase chain reaction (qPCR) applications in non-clinical BD studies.
• Survey of imaging techniques for non-clinical and clinical BD studies.

Main Results:

• Identified key regulatory differences and similarities in BD study requirements.
• Highlighted current practices and challenges in cell therapy BD studies through database analysis.
• Assessed the utility and limitations of qPCR for non-clinical CTP biodistribution.
• Cataloged various imaging modalities employed for CTP biodistribution assessment.

Conclusions:

• This review offers a comprehensive overview of BD study methodologies and regulatory landscapes.
• The findings will aid researchers and developers in designing and executing robust BD studies for CTPs.
• Facilitates informed decision-making for CTP regulatory submissions and clinical translation.