Stemcell Therapy Technology News

BIOTECH COMPANY DEVELOPED BRAIN DISEASE TREATMENT
By Steve Johnson
Mercury News


One of six children with a brain disease who were given a stem-cell treatment developed by StemCells of Palo Alto in a groundbreaking study, has died apparently from her disease, the company said Friday.

The unidentified 9-year-old girl, who died earlier this week, had been given the nerve stem cells derived from fetal tissue in January last year to treat neuronal ceroid lipofuscinosis, also known as Batten disease, the company said in a prepared statement. The disease is a rare and always fatal condition that affects children.

The girl, who received the treatment at Oregon Health & Science University's Doernbecher Children's Hospital, became critically ill from an apparent viral infection, seizures and respiratory distress, and was hospitalized nearly two weeks ago, the statement said.

Based upon an initial review by Doernbecher doctors, a committee of experts monitoring the study and StemCells' medical specialists, the girl's death was believed due to the natural progression of her disease rather than from the stem cells she received, the company said.

Children with Batten disease - which is caused by genetic mutations - suffer seizures and progressive loss of motor skills, sight and mental capacity, before eventually becoming blind, bedridden and unable to communicate. The stem-cell treatment was the first ever tried with children suffering from the malady.

StemCells initially asked the U.S. Food and Drug Administration in December

2004 for permission to conduct the unprecedented test on children. After asking the company for more information about how the surgery would be done and whether it risked causing cancer, the federal agency gave its permission Oct. 20, 2005.

Batten disease is caused by a defective gene that fails to create an enzyme the brain needs. By injecting the fetal stem cells into the brains of the six children, researchers involved in the study said they hoped the cells would help the brains produce the missing enzyme.

StemCells' statement added that the company is continuing to investigate the death and has been in contact with the U.S. Food and Drug Administration about the matter.

"These patients are in the very late stage of the disease and that is one of the criteria of enrollment" in the study, which is designed to determine if the treatment has any unsafe side effects, said StemCells Chief Executive Officer, Martin McGlynn.

"Coming into the study they have no prospect of survival," he added. The fact that one of them has died is a reminder of "how important it is to find a therapy or even a cure for this devastating disease."

All of the children who received the treatment were under the age of 13, said McGlynn, who noted that the surviving youngsters will be monitored by medical officials until the early part of 2009, when the study is expected to be completed.


From:http://www.mercurynews.com/peninsula/ci_8018506?nclick_check=1