Translational Study on Stem Cell Therapy for Brain Infarction

Abstract

Regenerative medicine using stem cells for cerebral infarction is a promising therapy, and various clinical trials are being conducted worldwide. In the development of cell therapy products for cerebral infarction, a translational study from preclinical to early clinical stages, including first-in-human trials, is also important. The results of a large number of basic studies must be translated into clinical studies, a very important stage commonly referred to as the “valley of death,” to determine whether they can be applied clinically. Basic studies are needed to prove the efficacy of cell therapy in terms of neuroprotection, cell differentiation, and angiogenesis, according to the pathological condition of cerebral infarction, as well as to conduct non-clinical safety studies in accordance with Good Laboratory Practice to determine the formulation specifications. In the treatment of cerebral infarction, there are various treatment concepts, depending on the targeted stage, such as those expected to improve blood flow or have neuroprotective effects while ischemia-induced brain damage is incomplete, and those expected to restore brain function after completion of brain damage. Currently, the development of treatment products for cerebral infarction is mainly based on somatic stem cells; however, optimal cell source, route of administration, timing of administration, frequency of administration, and number of cells for cell therapy products in clinical trials vary. Therefore, this paper outlines translational studies in stroke regenerative medicine.