A girl enrolled in a stem-cell trial for a fatal disease has died. In January, the nine-year-old received a brain transplant of neural stem cells derived from fetal tissue. She was one of six children in the trial for Batten disease, in which children rarely live into their teens. An independent group monitoring the trial decided that the death was due to the disease not the experimental treatment and said the trial could continue.
This starkly contrasts with the hope expressed by the father of one little boy enrolled in the trial just over a year ago: “He was a little boy who was basically waiting to die, now he’s waiting to get better,” Marcus Kerner told the Associated Press in an article entitled
“Coming into the study they have no prospect of survival,” said StemCells Chief Executive Officer, Martin McGlynn quoted in the San Jose Mercury News. “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.
A press release from the company expressed sympathy for the family and said the trial of the other children, who have all already received transplants of cells, would continue.
The clinical investigators involved in the trial who were quoted in this and other articles refused to hype the technique and even downplayed it, but should remind those involved in clinical trials of the danger of the therapeutic misconception, something that Nature Reports Stem Cells has covered in a commentary by Mildred Cho and David Magnus.
In Batten disease, the brain essentially poisons itself because it cannot clear away fats and proteins. The result is a slow, painful death accompanied by crippling debilities, including blindness. Since the disease is by mutations in the gene coding for an enzyme necessary to remove accumulating compounds, one hope is that injecting cells with a working version of the enzyme will ameliorate the disease. Such suffering will make parents and loved ones desperate.