ISSCR Launches Resource to Fast-Track Pluripotent Stem Cell Therapies

The International Society for Stem Cell Research (ISSCR) has unveiled a new resource titled Best Practices for the Development of Pluripotent Stem Cell-Derived Cellular Therapies. This comprehensive, interactive guide aims to expedite the transition of human pluripotent stem cell (PSC)-derived therapies into clinical trials and commercial application.

Developed by a team of leading international experts in cell therapy, this document identifies essential principles and decision points for creating safe, effective, and regulatory-compliant PSC-derived products. The guide is divided into seven sections, covering a range of topics from PSC line selection to regulatory considerations, preclinical studies, and clinical trials.

Hideyuki Okano, President of the ISSCR, expressed his gratitude towards the Best Practices Task Force for their dedication in creating this vital resource. “While the clinical applications of pluripotent stem cells will continue to evolve, this initiative ensures that the best practices available today are shared broadly with our community now and in the future through regular updates,” he stated.

Streamlining Development Processes

The Best Practices document highlights the necessity of selecting appropriate PSC starting materials and meeting regulatory standards across various jurisdictions. It emphasizes maintaining quality at every development stage, from cell banking to drug product manufacturing.

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.” She added that the document encapsulates the collective expertise of global professionals, providing actionable best practices that can shorten development time, reduce costs, and facilitate the approval of new therapies.

The resource is designed in an accessible visual format and includes links to jurisdictionally relevant regulatory information. It will be updated annually to keep pace with the rapidly changing landscape of stem cell therapy.

Global Impact and Accessibility

Nine regions, including Australia, Brazil, Canada, the European Union, Japan, South Korea, the United Kingdom, and the United States, are integrated into the resource. This approach consolidates crucial steps for the scientific community, guiding researchers and stakeholders through the complexities of cell therapy development.

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, remarked, “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 emphasized the potential of the Best Practices to enhance the speed and rigor of the translational process, ultimately increasing the number and quality of stem cell-based therapies reaching patients globally.

The ISSCR’s new initiative represents a significant step forward in the field of stem cell research, promoting collaboration and innovation while addressing the critical need for efficient pathways to clinical application.