StemCells Completes Cell Transplanting into First Cohort of Phase II Study

Palo Alto, Calif.-based StemCells Inc., a stem cell company developing cell-based therapeutics, has completed transplanting the six patients comprising the first cohort of its phase II Pathway Study.

The first cohort is an open-label dose escalation arm to determine the cell dose to be used for the second cohort of the study. The second cohort of the study is a single-blind arm in 40 patients that will assess efficacy of the company’s proprietary HuCNS-SC (purified human neural stem cells) platform technology for the treatment of cervical spinal cord injury (SCI).

“We are breaking new ground with this study,” said Stephen Huhn, M.D., FACS, FAAP, vice president, CNS clinical research and chief medical officer at StemCells. “This is the first controlled study to use neural stem cells with the intent to demonstrate improvement in motor function following spinal cord injury. In this first cohort, we have achieved the highest number of neural stem cells administered into the injured human spinal cord in medical history. We expect to release six month interim data on the first cohort later this year.

“Interest in this study from both physicians and patients has been very encouraging, resulting in the rapid enrollment of this cohort. The company is using web-based recruitment and, in the first six months, we have seen over 1,500 inquiries and 500 completed questionnaires and over 100 subjects have been reviewed for possible eligibility. Our experience with the first cohort reflects great clinical momentum, as well as the excitement within the spinal cord injury community for potential novel treatments. Detecting evidence of motor improvement in this phase II study would provide proof-of-concept for this approach in spinal cord injury and would ultimately lead to a therapy that dramatically enhances the quality of life for those afflicted with spinal cord injuries.”

The Pathway phase II study, titled “Study of Human Central Nervous System (CNS) Stem Cell Transplantation in Cervical Spinal Cord Injury,” will evaluate the safety and efficacy of transplanting the company’s proprietary human neural stem cells into patients with traumatic injury in the cervical region of the spinal cord. Conducted as a randomized, controlled, single-blind study, the trial will measure efficacy by assessing motor function according to the International Standards for Neurological Classification of Spinal Cord Injury (ISNCSCI). The primary efficacy outcome will focus on change in upper extremity strength as measured in the hands, arms and shoulders. The trial will enroll approximately 52 subjects and follow the patients for 12 months post-transplant.

StemCells has demonstrated human safety data from completed and ongoing clinical studies in which its proprietary HuCNS-SC cells have been transplanted directly into all three components of the central nervous system: the brain, the spinal cord and the eye. StemCells clinicians and scientists believe that HuCNS-SC cells may have broad therapeutic application for many diseases and disorders of the central nervous syste,. Because the transplanted HuCNS-SC cells have been shown to engraft and survive long-term, there is the possibility of a durable clinical effect following a single transplantation, the company reports. The HuCNS-SC platform technology is a highly purified composition of human neural stem cells (tissue-derived or “adult” stem cells). Manufactured under cGMP (current good manufacturing practices) standards, the Company’s HuCNS-SC cells are purified, expanded in culture, cryopreserved, and then stored as banks of cells, ready to be made into individual patient doses when needed.