Investigating the use of sibling cord blood to treat cerebral palsy

What is cerebral palsy?

Cerebral palsy refers to a group of disorders that affect movement, balance, and posture. It is caused by abnormal brain development or damage to the developing brain, and symptoms can vary widely between affected individuals. Severely affected people might not be able to walk at all or require special equipment to walk, while someone who is mildy affected might just walk a little awkwardly.

Cerebral palsy patients often also have other related conditions such as intellectual disability, seizures, problems with vision, hearing, or speech, joint problems, or changes in the spine.

The majority of cerebral palsy cases (85–90%) are congenital, meaning that they were caused by something during pregnancy or birth, with a small number thought to be due to lack of oxygen at birth. However, the underlying cause of the abnormal brain development or damage to the developing brain in most cerebral palsy cases is unknown.

Previous studies with stem cells

As of today, there is no cure for cerebral palsy. Scientists are exploring potential treatment options and the use of umbilical cord stem cells is one option. Cord blood offers a rich source of stem cells, which have unlimited potential and can be easily collected using non-invasive methods.

Previous clinical trials have shown improved whole brain connectivity and motor function in affected children that were given infusions of their own stem cells (collected from their own cord blood at birth). These “self” stem cells are known as autologous stem cells and are a perfect match; hence removing the risks and complications of cell rejection.

Are “non-self” stem cells an option?

Many children suffering from cerebral palsy do not have their own stem cells saved from birth, so a recent study aimed to investigate the safety of using “non-self” stem cells (allogenic stem cells). This would open up the use of stem cells to treat a much wider proportion of people affected by cerebral palsy.

A recent phase I clinical trial included 15 children with moderate-to-severe cerebral palsy. Each of these children received infusions from a sibling and were followed up for two years post-treatment. The researchers did not observe any negative effects linked to the cord blood infusions, suggesting that sibling samples or other matching stem cell samples are a potential treatment option.

Next steps

During this study, the researchers also noted some improvement in motor function in treated children. However, they could not conclusively attribute these improvements to the stem cells treatment because the trial was only looking to establish the safety of using umbilical stem cells from siblings.

Now that the safety of donor stem cells has been shown in this small clinical trial, future trails can now investigate the effectiveness of using donor stem cells to treat cerebral palsy.

References:

Sun JM, Song AW, Case LE, Mikati MA, Gustafson KE, Simmons R, Goldstein R, Petry J, McLaughlin C, Waters-Pick B, Chen LW, Wease S, Blackwell B, Worley G, Troy J, Kurtzberg J. Effect of Autologous Cord Blood Infusion on Motor Function and Brain Connectivity in Young Children with Cerebral Palsy: A Randomized, Placebo-Controlled Trial. Stem Cells Transl Med. 2017 Dec;6(12):2071-2078. doi: 10.1002/sctm.17-0102. Epub 2017 Oct 28. PMID: 29080265; PMCID: PMC5702515.

Sun JM, Case LE, Mikati MA, M Jasien J, McLaughlin C, Waters-Pick B, Worley G, Troy J, Kurtzberg J. Sibling umbilical cord blood infusion is safe in young children with cerebral palsy. Stem Cells Transl Med. 2021 Sep;10(9):1258-1265. doi: 10.1002/sctm.20-0470. Epub 2021 Jun 4. PMID: 34085782; PMCID: PMC8380440.