Date: April 21, 2010
Summary:
In an official company news release, Stem Cells, Inc., a biotechnology company in the field of stem cell research, announced plans to advance to a second clinical trial using purified human neural stem cells to treat Batten disease:
StemCells, Inc., a biotechnology company in the field of stem cell research and regenerative medicine, announced today that it has submitted a protocol to the FDA for initiation of a second clinical trial of its proprietary HuCNS-SC® human neural stem cells in neuronal ceroid lipofuscinosis (NCL), which is also often referred to as Batten disease. NCL is a genetic disorder characterized by the absence of a critical enzyme, which leads to the loss of neurons and the eventual death of the patient. The Company completed a Phase I clinical trial in NCL in January 2009 and reported the results to the FDA in September 2009.
The proposed new trial is designed to further assess the safety of HuCNS-SC cells in NCL, while also examining the ability of the cells to affect the progression of the disease. The Company plans to enroll six patients with infantile and late infantile NCL. Because intervention prior to the final stages of the disease will likely be key to providing a therapeutic benefit, the Company plans to enroll patients with less brain atrophy than those enrolled in its first trial. Under the proposed protocol, all patients would be transplanted with HuCNS-SC cells and immunosuppressed for nine months. The patients would also be evaluated and assessed at regular intervals over the course of 12 months following transplantation. As the Company intends to follow the effects of this therapy long-term, a separate four-year observational study would be initiated at the conclusion of this trial. Upon FDA authorization of the trial protocol, the Company will proceed with site selection and seek the necessary Institutional Review Board approval to initiate the trial.
