Stem Cell Trial Positive in Batten Disease (STEM)
StemCells, Inc. (NASDAQ: STEM) has announced positive results from the first Phase I clinical trial of its proprietary HuCNS-SC product candidate of purified human neural stem cells, including demonstration of a favorable safety profile along with evidence of engraftment and long-term survival of the HuCNS-SC cells. This trial was in Neuronal ceroid lipofuscinosis, or Batten Disease, a fatal neurodegenerative disorder that afflicts infants and young children and is caused by genetic mutations. The Phase I trial was designed primarily to assess the safety of HuCNS-SC cells as a potential cell-based therapeutic. Six patients with advanced stages of infantile and late infantile neuronal ceroid lipofuscinosis were transplanted with HuCNS-SC cells and followed for 12 months.
Overall, the Phase I data demonstrated that high doses of HuCNS-SC cells, delivered by a direct transplantation procedure into multiple sites within the brain, followed by twelve months of immunosuppression, were well tolerated by all six patients enrolled in the trial.
The patients’ medical, neurological and neuropsychological conditions, following transplantation, appeared consistent with the normal course of the disease. The independent Data Monitoring Committee has also concurred with the Stem Cell’s safety assessment.
StemCells will present the final study report to the FDA and plans to pursue future clinical development of HuCNS-SC as a potential treatment for infantile and late infantile NCL. This was the first-ever FDA-authorized study of human neural stem cells as a potential therapeutic agent in humans. As many as one billion cells were transplanted into certain patients.
StemCells previously reported the loss of the second patient enrolled in the trial, who died from the natural progression of the disease approximately one year post-transplant. The family consented to an autopsy examination of the brain and the company was able to establish that the donor cells had engrafted and survived, despite severe brain atrophy related to the Batten Disease. By permitting the autopsy, the family allowed the researchers to learn very important details that will potentially benefit future patients.
Full data can be found at the StemCellsInc.com website.
JON C. OGG