Oregon researchers are set to begin the first clinical trial in the nation using fetal stem cells to treat infants and children suffering from a rare and fatal brain disease.
The stem cells will be injected into the brain in an effort to halt the progress of the genetic disorder called Batten disease that leaves its victims blind, speechless and paralyzed before they die.
Researchers note the cells are taken from fetal tissue — not from developing embryos.
StemCells Inc. of Palo Alto, Calif., announced last October that it received clearance from the Food and Drug Administration to begin a human trial for its proprietary fetal stem cell product.
Oregon Health & Science University, which will conduct the clinical trial, emphasized it will be used only to determine whether the product is safe.
"While the preclinical research in the laboratory and in animals is promising, it is important to note that this is a safety trial and, to our knowledge, purified neural stem cell transplantation has never been done before," said Dr. Robert Steiner, vice chairman of pediatric research at OHSU and the lead investigator in the study.
Fully formed brain cells have been transplanted to treat Parkinson’s disease patients and stroke victims but this is the first trial involving immature neural cells still capable of transforming themselves into different kinds of brain cells.
Once patients are found for the trial, a pediatric surgeon will drill holes in the child’s skull to insert a needle to inject the fetal stem cells directly into the brain, Steiner said.
No biopsies or invasive testing will be done following the injections, he said.
"But we will be following the patients clinically — their motor skills, development, language skills — to see if the use of these cells might actually have an impact on the course of the disease," Steiner said.
Batten disease is named for the British pediatrician who first described it in 1903. Research has since determined it is caused by mutations in the genes responsible for teaching the body how to make certain enzymes that affect brain function.
Without these enzymes, material builds up inside brain cells that cause a progressive decline in mental and motor function, blindness, seizures and early death.
Steiner said doctors estimate less than one in 100,000 children are affected.
One potential candidate for the trial is Daniel Kerner of Orange County, Calif., the 6-year-old son of Joanna and Marcus Kerner, who were featured last month on the CBS news magazine "60 Minutes."
