A placebo-controlled trial in cerebral palsy might unlock some answers for parents

The parents of children with cerebral palsy (CP) rank high in number among the desperate calls that come to CIRM wanting to know about stem cell therapies offered on the internet. They don’t like to hear that we have very little information suggesting benefit from stem cells in these kids and that there is little reason to believe the types of cells being offered could grow new brain tissue to repair the abnormal brain development seen in CP.

They like hearing that there is some evidence that the type of stem cells being used might be able to tamp down any inflammatory process that is hampering brain function, and that it might be possible for these cells to trigger some sort of innate repair mechanism within the child. But the bottom line is that we really don’t know, and we certainly don’t know what is the best cell type to use and how, when and where to deliver the cells to get the maximum benefit if there is any.

So, it was heartening to see that a clinical trial registered with the Food and Drug Administration is enrolling patients in a study designed to answer some of these questions. Sponsored by Cord Blood Registry and conducted at the University of Texas Health Sciences Center at Houston the trial will compare two types of stem cells that came from the children themselves. They will compare stem cells from some children’s own stored cord blood with stem cells from other children’s bone marrow.

The research team plans to recruit 15 children into each group and 10 in each will receive the stem cells and five will receive a placebo injection, containing no cells. The parents will not know which injection their children received but at the end of one year, the parents of those who received the placebo will be told and given the option of a stem cell injection.

The Los Angeles Business Journal was one of many outlets that picked up the company’s press release. In it, the lead researcher, UT’s Charles Cox, explained the effect they hoped to see: “There is preclinical data indicating that the ongoing neuro-inflammatory response is a driver of further injury in cerebral palsy so the hope is to reduce this neuro-inflammation. Our goal is to break the cycle of inflammation and injury.” They plan to evaluate the children as six months, one year and 24 months. So, anxious parents may start to get a few answers in a year or so. CIRM convened a workshop on how stem cell science could impact CP and that cerebral palsy report is available online.

Don Gibbons