Preclinical Studies of a Somatic Stem Cell Therapy: Pathway to Clinical Approval

摘要

In 2005, StemCells, Inc. received clearance from the U.S. Food and Drug Administration (FDA) to begin a Phase I safety and preliminary efficacy trial for the Company's proprietary human neural stem cell product -HuCNS-SC~(TM) - to treat Batten disease. This was the first-ever FDA-approved clinical trial to use a purified composition of human neural stem cells as a potential therapeutic agent in humans. Prior to approval, extensive efforts were put forth to characterize our product and demonstrate efficacy and safety in preclinical animal models.