Health News: Scientific Profiles First Human Neural Stem Cell Transplant in the Country

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Phase I trial of HuCNS-SC ™ to treat Stem Cell Batten Disease advances.

Scientific American's science news web site,, today posted the findings of the first ever human neural stem cell clinical trial being conducted in the United States. This is the first trial to use a purified composition of human neural stem cells as a potential therapeutic agent in humans.

StemCells Inc., (NASDAQ: STEM) a clinical-stage company focused on the discovery, development and commercialization of cell-based therapeutics to treat diseases of the nervous system, liver and pancreas, is the biotech innovator behind this Phase I clinical trial. StemCells is the first firm to directly identify and isolate human neural stem cells from normal brain tissue. The company's programs seek to repair or repopulate neural, liver or other tissue that has been damaged or lost as a result of disease or injury.

The first transplantation of Stem Cells, Inc's proprietary human neural stem cell product, HuCNS-SC took place on November 15, 2006 at the Oregon Health & Science University's (OHSU) Doernbecher Children's Hospital. The transplant is part of Stem Cells Inc's planned Phase I clinical trial designed to evaluate the safety and preliminary efficacy of neural stem cells, HuCNS-SC, as treatment for infantile and late infantile neuronal ceroid lipofuscinosis (NCL). This medical condition is often referred to as Batten disease, a rare and fatal neurodegenerative condition afflicting infants and children. The clinical trial is open label with two dose cohorts, and is expected to enroll six patients during the next 12 months.

Batten disease is named after the British pediatrician who first described the juvenile form of neuronal ceroid lipofuscinosis (NCL) in 1903. The name is now commonly used to encompass three forms of NCL, infantile, late infantile and juvenile onset. All forms have the same basic cause; lack of a lysosomal enzyme and have a similar progression and outcome. The different forms of NCL have traditionally been classified by age of onset, but today the disease is more precisely classifiable in terms of mutations in the specific gene and enzyme causing the disease. Children with Batten disease suffer seizures, progressive loss of motor skills, sight and mental capacity, eventually becoming blind, bedridden and unable to communicate. Today, Batten disease is always fatal.

Neural stem cells are harvested from the post-natal, post mortem, human and animal brains for stem cell research. Investigation on stem cell research will aid the science and medical community's understanding of the nervous system and may assist in the development and treatments for currently incurable brain diseases and injuries. Stem cell research through clinical trial investigation allows for the study of these neural stem cells as transplantable tissue for the repair of injury such as that sustained during traumatic brain injury, inherited generic disorders or stroke for the repair of pathological processes such as those seen in neurogenetic diseases or neurodegenerative conditions such as Parkinson's or Alzheimer's disease.

At the end of this year, Scientific American's web site,, will be launching a new Health Channel on its navigation bar. This channel will enhance the web's site ongoing and breaking science and technology section in addition to showcase a series of topics covering well-being, fitness and prevention.

About Scientific American

Scientific (, which debuted in 1996, has grown into a dynamic online resource that provides coverage of the latest in science and technology as well as health news articles from current and past issues, podcasts, website exclusives, daily science news, blogs and weekly polls. It has become a leading online science health news and technology destination with more than 1,700,000 visitors per month.

For more than 155 years, Scientific American, one of the world's most enduring and revered science and technology magazines, has chronicled for its readers major and technology innovations and discoveries using expert accounts and assorted journalistic features. The magazine publishes 15 foreign language editions with a total circulation of more than 1,000,000 worldwide.


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