Children and infants with a rare neurodegenerative disorder, will undergo a Phase I clinical trial using stem cells at Doernbecher Children’s Hospital, Oregon Health & Science University.

This trial will test if:

1. HuCNS-SC(TM), a proprietary human central nervous stem cell product developed by Stem Cells, Inc. is safe or not.

2. It can slow the progression of two forms of neuronal ceroid lipofuscinosis (NCL).

NCL is caused due to mutations or changes in the genes responsible for producing certain enzymes. Without these enzymes or proteins, material builds up inside brain neurons and other brain cells, causing a rapidly progressive decline in mental and motor function, blindness, seizures and early death.

Dr Robert D Steiner, vice chairman of pediatric research at Doernbecher Children’s Hospital, said that neural stem cell transplantation has never been done before and stem cells will provide an important therapeutic advancement for the children diagnosed with NCL.

Co-investigator Nathan Selden commented that the effort of delivering stem cells directly into the human brain is found safe and effective it will be tagged as one of the potential treatment in helping thousands of patients suffering from degenerative brain diseases.

Up to six children from Oregon or around the country will undergo HuCNS-SC transplantation at Doernbecher. Previous studies on mice have shown that HuCNS-SC increases the amount of the missing enzyme, reduces the amount of abnormal material in the brain and prevents the death of some brain cells.

Stem Cells, Inc. received clearance from the U.S. Food and Drug Administration to initiate a Phase 1 clinical trial of HuCNS-SC in October 2005. The company believes this will be the first trial using a purified composition of neural stem cells as a potential therapeutic agent in humans.

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