Accelerating Cell Therapy for Stroke in Japan: Regulatory Framework and Guidelines on Development of Cell-Based Products

In recent years, the clinical application of regenerative medicine using stem cells has been heralded as a new approach to the treatment of cerebral infarction.1 In Japan, various clinical trials on cell-based products are being initiated in patients with the disease.2,3 However, the full-scale clinical use of such products remains a major challenge. Unsolved questions include how nonclinical studies should be conducted and what type of designs should be used in clinical trials. Similar questions have remained in the United States. Researchers in the academia, industry leaders, and regulatory representatives organized the Stem Cell Therapeutics as an Emerging Paradigm in Stroke (STEPS) program, which proposed basic approaches to quality assurance, nonclinical studies, and clinical trials in the development of cell-based products for the treatment of cerebral infarction.4–6In Japan, the government has published guidelines for ensuring the quality and safety of individual cell-based products that are used as raw materials. However, no specific guidelines have been established for cell-based products for the treatment of cerebral infarction. Although the STEPS has proposed helpful basic approaches to the development of such regenerative medicine products, guidelines are needed that are based on Japanese regulations, taking into consideration the clinical setting of cerebral infarction treatment in the country.7 Such guidelines would be useful to facilitate appropriate development of the products. For these reasons, we prepared the Guidelines on Development of Cell-based Products for the Treatment of Cerebral Infarction. The guidelines comprised consensus about quality assurance, nonclinical studies, cell delivery methods, and clinical trials on products that were discussed in relevant individual working groups, while considering disease-specific issues and the existing Japanese guidelines on ensuring quality and safety of cell-based products for individual cell types.The original guidelines in Japanese language were framed by Working Group for …