An experimental stem-cell treatment developed by StemCells of Palo Alto has shown no dangerous side effects after being injected into six children with a rare and as-yet always fatal brain disorder, the company said Monday.
The groundbreaking study begun in 2006 involves children suffering from Batten disease, a heretofore incurable malady that often causes its mostly young victims to suffer seizures and blindness before killing them.
The ailment results from a defective gene that fails to create an enzyme the brain needs to dispose of cellular waste. The waste piles up and kills healthy cells until the patients die, typically before they reach their teens. By injecting fetal stem cells into the children’s brains, researchers hope the cells will help the brains produce the missing enzyme.
In the study, approved by the U.S. Food and Drug Administration and involving children in advanced stages of the disease, the stem cells “were well tolerated by all six patients” and produced no ill effects, the company said in a prepared statement.
In January last year, a 9-year-old girl in the study died after becoming critically ill from an apparent viral infection, seizures and respiratory distress. But at the time, doctors who did the injections at Oregon Health & Science University’s Doernbecher Children’s Hospital and a committee of experts monitoring the study said they believed the girl’s death was due to the natural progression of the disease.
StemCells, which plans to continue to check on the six children’s health, said the youngsters’ advanced illnesses and limited number made it difficult to determine if the stem-cell treatment helped their conditions. To assess whether the cells can counter the disease, the company hopes to conduct another study with patients who have an earlier stage of Batten.
Marcus Kerner, whose son Daniel was the first of the six children to undergo the unique treatment at age 6, said his son has gradually deteriorated over the past 21/2 years.
“The disease is still progressing in him,” Kerner said. “The areas where he did not receive transplanted cells were the brain stem and the cerebellum. Those areas have continued to decline. But in the areas where he did receive the cells, we see subtle, continued abilities of empathy, smiling, awareness. And he did say over the weekend as he was taking a bath with his mom the word ‘dad.’ ” The last time the boy had said that was a year ago.
“Daniel is alive and he is still fighting,” Kerner added. “He still knows that he is very loved and that many people are praying for him. He has great days that are like brilliant sunshine, but this disease is always a storm for every child it touches.”
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