StemCells (STEM) is seeking permission from the FDA to start a 2nd clinical trial of its proprietary HuCNS-SC human neural stem cells in neuronal ceroid lipofuscinosis (NYSE:NCL), which is also often referred to as Batten disease.
NCL is a fatal neuro-degenerative disorder that afflicts infants and young children. Batten Disease is caused by genetic mutations, and children who inherit the defective gene are unable to produce enough of an enzyme that processes cellular waste substances that accumulate in a part of cells known as the lysosome. Without the enzyme, the cellular waste builds up, and eventually the cells cannot function and die. Children with NCL appear healthy when born, but as their brain cells die, they begin to suffer seizures and progressively lose motor skills, sight and mental capacity. Eventually, they become blind, bedridden and unable to communicate or function independently. The infantile and late infantile forms of NCL are caused by different genetic mutations. There currently is NO effective treatment for the disease.
StemCells’ lead product candidate, HuCNS-SC cells are a highly purified composition of human neural stem cells that are expanded and stored as banks of cells.
- 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.
STEM had completed a phase-I clinical trial in neuronal ceroid lipofuscinosis (NCL) in 1/09 and reported the results to the US FDA in 9/09. The 1st NCL trial was focused primarily on safety; data showed that the cells, the immunosuppression regimen and the procedure were well tolerated. STEM studies have shown that HuCNS-SC cells produce the enzyme missing in neuronal ceroid lipofuscinosis (NCL).
- STEM plans to enroll 6 patients with infantile and late infantile neuronal ceroid lipofuscinosis. Under the proposed protocol, all patients would be transplanted with HuCNS-SC cells and immunosuppressed for 9 months,
- The patients would also be evaluated and assessed at regular intervals over the course of 12 months following transplantation. A separate 4 year observational study would be initiated at the conclusion of this trial.