Clinical Trials on Cell Therapy for Perinatal Brain Injury: Challenges and Opportunities

We performed a review of the literature and identified 9 published studies that conducted clinical trials to identify the safety, feasibility, and efficacy of cell therapy and 16 ongoing clinical trials. Most studies showed an improvement of motor function compared to the control groups, and the improvement appeared in the relatively early stage after transplantation. However, the mechanism by which stem cells help to recover neurological cells remains unclear. Many of the studies or trials were in phase 1 or phase 2; thus, it is too early to conclude the effectiveness of cell therapy. Therefore, further clinical trials at an advanced stage and experimental research using animal models are needed to elucidate the mechanism and efficacy. Conversely, the pharmaceutical agencies that review new drugs and medical devices have installed various systems and policies to accelerate the development of new drugs. For instance, the Pharmaceuticals and Medical Devices Agency (PMDA) has established a special department for cellular and tissue-based products; furthermore, the PMDA has launched a conditional time-limited marketing authorization system. This system makes it possible to rapidly provide new drugs to patients. In addition, the PMDA has installed an accelerated assessment system to facilitate the development of drugs for diseases without route-based treatment.