Stem cell therapy for Cerebral Palsy

Over the past decade, there have been over three dozen clinical trials treating cerebral palsy with stem cells. Several of these trials had outcomes, published in peer-reviewed medical journals, which showed that cerebral palsy patients who received stem cells had a significant improvement compared to patients in control groups. It is hard to compare one trial against another, because a variety of stem cell types have been tested, and the trials differed in how and when they measured patient responses. Nonetheless, a 2023 meta analysis of multiple trials showed that there is statistically significant benefit from cord blood therapy for cerebral palsy.

Despite these successful reports, no one understands the “mechanism of action” by which the stem cells provide a benefit.  Both cord blood stem cells and cord tissue MSC are known to suppress inflammation. Researchers at Duke have argued that a trace component of the cells in cord blood may enable re-myelination of neurons in the brain. It is presumed that stem cell therapy probably also benefits other conditions that are similar to cerebral palsy, such as hypoxic ischemic encephalopathy. Bear in mind these benefits were proven for groups of patients and individual outcomes will vary. While some children have had dramatic improvements, for example Adriana or Asia, others have not improved at all. There are many open questions about stem cell therapy for cerebral palsy that have not yet been answered by the existing research. It is not clear what type of stem cells are the “best” for this therapy, and there is also some debate over the optimum method of administering the stem cells (intravenous drip versus intrathecal injection).

On the basis of the available evidence, Duke University has received permission from the FDA to offer expanded access to cord blood therapy for cerebral palsy and other pediatric brain injuries. Any family that has a child with an acquired (not hereditary) brain injury, and has that child’s cord blood or a sibling’s cord blood in storage, is eligible for this therapy. 

An important caveat for parents is that while stem cell therapy may improve a child’s cerebral palsy symptoms, it is not a cure all and does not replace other supports, such as physical and occupational therapy, tutoring, etc. Please remember that the website of Parent’s Guide to Cord Blood Foundation is not a substitute for medical advice from a physician.