ISSCR Launches Resource to Propel PSC-Derived Therapies Forward

The International Society for Stem Cell Research (ISSCR) has unveiled a significant new resource aimed at expediting 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 and interactive document is designed to facilitate the transition of PSC-derived therapies into clinical trials and eventual commercial use.

Developed by an assembly of leading international experts in the field of cell therapy, the resource outlines fundamental principles and decision points crucial for creating safe, effective, and regulatory compliant PSC-derived products. The document is organized into seven sections, addressing a range of essential topics, including PSC line selection, raw material utilization, and regulatory considerations, as well as preclinical studies and clinical trials.

Collaboration to Enhance Stem Cell Research

The creation of this resource demonstrates the impact of collective expertise within the stem cell community. According to Hideyuki Okano, ISSCR President, “I am deeply grateful to the ISSCR Best Practices Task Force for their vision and dedication in producing this blueprint for advancing new cellular therapies.” He emphasized the ongoing evolution of clinical applications for pluripotent stem cells and the commitment to sharing best practices with the community through regular updates.

The document is particularly relevant for researchers and innovators in the field, as Jacqueline Barry, co-chair of the Best Practices Steering Committee and Chief Clinical Officer at Cell and Gene Therapy Catapult in the UK, noted: “Focused research is the foundation of innovation, but without clear translational guidance, promising therapies can stall before reaching patients for reasons that could have been anticipated and prevented.” She highlighted that the best practices distilled from the collective experience of global experts can significantly reduce development time and costs while accelerating the approval of new therapies.

Comprehensive Guidelines for Global Standards

The ISSCR Best Practices document underscores the critical importance of selecting appropriate PSC starting materials and adhering to regulatory requirements across different jurisdictions. It also emphasizes maintaining high quality throughout all stages of development, from cell banking and ancillary materials to drug product manufacturing. Presented in a visually engaging format, the resource includes links to essential regulatory information relevant to various jurisdictions and will be updated annually to stay aligned with the evolving landscape of stem cell therapy development.

The document specifically addresses regulatory timelines and operational considerations for nine jurisdictions, including Australia, Brazil, Canada, China, the European Union, Japan, South Korea, the United Kingdom, and the United States. This consolidated information provides the scientific community with vital steps necessary for progressing in cell therapy development.

According to 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, “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 expressed hope that the anticipated effects of these best practices will enhance the speed and rigor of the translational process, ultimately increasing both the quantity and quality of stem cell-based therapies accessible to patients worldwide.

The release of this comprehensive guide reflects a substantial advance in the stem cell research field, reinforcing the commitment to developing transformative therapies that have the potential to improve patient outcomes across various medical conditions.