ISSCR Launches New Resource to Enhance PSC-Derived Therapies

The International Society for Stem Cell Research (ISSCR) has unveiled a new resource aimed at accelerating the development of therapies derived from human pluripotent stem cells (PSCs). Titled “Best Practices for the Development of Pluripotent Stem Cell-Derived Cellular Therapies,” this comprehensive guide is designed to facilitate the transition of PSC-derived therapies into clinical trials and commercial use.

Developed by a team of leading international experts in cell therapy, the document provides essential principles and decision points necessary for creating safe, effective, and regulatory-compliant PSC-derived products. Organized into seven sections, it covers a wide range of topics, including PSC line selection, raw material use, regulatory considerations, preclinical studies, and clinical trials.

Hideyuki Okano, President of ISSCR, expressed gratitude toward the Best Practices Task Force for their dedication to this initiative. He stated, “This blueprint for advancing new cellular therapies ensures that the best practices available today are shared broadly with our community now and in the future through regular updates.”

The guide aims to address the critical gap in translational guidance that has historically stalled promising therapies before they reach patients. Jacqueline Barry, co-chair of the Best Practices Steering Committee and Chief Clinical Officer at Cell and Gene Therapy Catapult in the UK, remarked, “Focused research is the foundation of innovation, but without clear translational guidance, promising therapies can stall.”

The ISSCR document emphasizes several key areas, including the selection of appropriate PSC starting materials and adherence to regulatory requirements across various jurisdictions. Moreover, it stresses the importance of ensuring quality throughout the entire process, from cell banking and ancillary materials to the manufacturing of drug products. Presented in an accessible visual format, the resource includes links to critical regulatory information relevant to different jurisdictions and will be updated annually to reflect the evolving landscape of stem cell therapies.

Global Collaboration and Regulatory Considerations

The resource encompasses insights from nine jurisdictions, including Australia, Brazil, Canada, China, the European Union, Japan, South Korea, the United Kingdom, and the United States. This broad perspective provides the scientific community with consolidated access to vital steps necessary for the development of cell therapies.

Kapil Bharti, co-chair of the Best Practices Steering Committee and scientific director at the National Institutes of Health’s National Eye Institute in the USA, noted the document’s significance for various stakeholders. “This resource is a much-needed tool for academic researchers, biotech innovators, regulators, and industry stakeholders preparing to transition from proof-of-concept studies to first-in-human trials,” he stated.

Through this initiative, the ISSCR aims to enhance the speed and rigor of the translational process, ultimately increasing both the quantity and quality of stem cell-based therapies available to patients worldwide. The release of this resource marks a significant step in fostering collaboration and innovation within the field of stem cell research.