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StemCells Announces Milestone in Batten Disease Clinical Trial

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StemCells, Inc. has announced that the Phase I clinical trial of its HuCNS-SC™ product candidate (purified human neural stem cells) has completed enrollment of the low-dose cohort and will proceed to the high-dose cohort.

This trial is designed to evaluate the safety and preliminary efficacy of the HuCNS-SC product candidate as a treatment for infantile and late infantile neuronal ceroid lipofuscinosis (NCL), also often referred to as Batten disease.

To date, three patients out of a planned total of six have been transplanted with HuCNS-SC cells. A review of the trial data to date, conducted by an independent Data Safety Monitoring Committee comprised of experts in pediatric neurosurgery, pediatric neurology, solid organ transplantation, and genetics, has identified no safety issues that would preclude advancing the trial to the next dose level.

“This is a significant milestone for the trial and StemCells, Inc. We are halfway through the planned enrollment in the first FDA-approved clinical investigation of purified human neural stem cells. The first patient enrolled in the trial has now reached the halfway point of the study and completed a number of important assessments. To date, all three patients have tolerated the transplantation and have returned home,” said Stephen Huhn, M.D., F.A.C.S., F.A.A.P., Vice President and Head of the Neural Program of StemCells, Inc.

“We are encouraged by the progress of the trial, but remain mindful of the difficult challenges involved with the development of novel therapeutics. We are also grateful for the participation of the families in this study and the commitment of the research staff at OHSU. Batten disease is a terrible affliction, and this trial is the first step on the path toward a treatment for this devastating disease and potentially other lysosomal storage disorders.”