Public HealthStemCells, Inc. To Present Phase I Clinical Trial Results At The 12th International Congress On NCL
StemCells, Inc. (NASDAQ:STEM) announced plans to present data from its Phase I clinical trial of its proprietary HuCNS-SC® product candidate (purified human neural stem cells) at the 12th International Congress on Neuronal Ceroid Lipofuscinoses (NCL) being held June 3 - 6, 2009 in Hamburg, Germany. Stephen Huhn, MD, FACS, FAAP, vice president and head of the Company"s CNS program, is scheduled to present the data on Saturday, June 6.
The six-patient Phase I trial, conducted at Oregon Health & Science University Doernbecher Children"s Hospital, was designed primarily to assess the safety associated with transplanting HuCNS-SC cells as a treatment for infantile and late infantile NCL, often referred to as Batten disease.
About Neuronal Ceroid Lipofuscinosis (Batten Disease)
Neuronal ceroid lipofuscinosis is a fatal neurodegenerative disorder that afflicts infants and young children. The disorder 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, these children become blind, bedridden and unable to communicate or function on their own. There currently is no cure for the disease. The infantile and late infantile forms of NCL are caused by different genetic mutations. As the names imply, the two forms begin to afflict patients at different stages of infancy, but both have similar disease progression and outcomes.
About HuCNS-SC® Cells
StemCells" lead product candidate, HuCNS-SC cells, is a purified composition of normal human neural stem cells that are expanded and stored as banks of cells. The Company"s preclinical research has shown that HuCNS-SC cells can be directly transplanted; they engraft, migrate, differentiate into neurons and glial cells; and they survive for as long as one year with no sign of tumor formation or adverse effects. These findings show that HuCNS-SC cells, when transplanted, act like normal stem cells, suggesting the possibility of a continual replenishment of normal human neural cells.
StemCells, Inc.