Transplanting stem cells from the bone marrow to the spinal canal — where they can reach the brain — improves motor function and muscle tone in people with cerebral palsy (CP) related to jaundice, an open-label study shows.
The study, “Improvement in gross motor function and muscle tone in children with cerebral palsy related to neonatal icterus: an open-label, uncontrolled clinical trial,” was published in the journal BMC Pediatrics.
Jaundice is a usually harmless condition affecting 60 to 70% of newborns worldwide, which makes a baby’s skin, eyes, and other tissues turn yellow. It is caused by the build-up in the blood of bilirubin — a yellow substance produced when red blood cells are broken down.
Treatment is usually only recommended if tests show very high levels of bilirubin in a baby’s blood. Such high bilirubin levels can cross the thin protective layer around the brain — the blood-brain barrier — and cause brain damage, potentially resulting in CP, deafness, and hearing loss.
The risk for cerebral palsy in newborns with jaundice is estimated to be 0.57 per 100,000 births.
Stem cells have the remarkable potential to develop into virtually any cell type in the body, and to replicate rapidly. These properties highlight their potential to replace cells that are lost, or to provide protective and survival signals to the remaining cells.
Previous studies have shown that transplantation of stem cells — derived from the umbilical cord or the bone marrow — improve motor function in CP patients. However, no investigations have been done specifically regarding people with CP related to jaundice.
To learn more, researchers in Vietnam evaluated the safety and effectiveness of transplanting stem cells from the bone marrow to the spinal canal in children with jaundice-related CP. The goal was to improve the children’s motor function and muscle tone.
The open-label, uncontrolled clinical trial (NCT03123562) included 25 children with bilateral spastic CP related to jaundice. The children — 15 boys and 10 girls — had a median age of 5.4 years, ranging from 2 to 15 years.
Participants had moderate-to-severe CP according to the Gross Motor Function Classification System (GMFCS). Among them, 14 (56%) were at a higher severity degree of GMFCS classification (level 5).
Changes in motor function and muscle tone were analyzed at three time points — at the beginning of the study, and at six and 12 months after treatment. Motor function was assessed with both 88-item and 66-item Gross Motor Function Measures (GMFM), while muscle tone was measured by the Modified Ashworth Scale.
Researchers collected stem cells from the bone marrow — known as bone marrow mononuclear cells (BMMCs) — of each patient. The cells were then injected directly into the spinal cord — from which it can reach the brain — of each child. The injections were done at the beginning of the study, and six months after the first transplant.
After stem cell treatment, all children received extensive rehabilitative therapy for one hour per day for 12 days. Parents were instructed on how to conduct rehabilitative therapy at home.
The results showed a significant improvement in gross motor function, and a significant decrease in muscle tone values at six and 12 months after stem cell transplant, compared with the beginning of the study.
The greatest change was observed in the median GMFM-88 score. It increased by 17.5 at six months, and by 34.9 at 12 months after treatment, compared with the score at the beginning of the study (18.3).
Factors such as age, gender, and severity of the disease had no impact on the results, according to the researchers.
Adverse events included vomiting (32%), local pain (16%), and mild fever without infection (4%). All adverse events were easily managed with standard medications. No severe adverse events were reported.
Researchers noted that these adverse events were less severe than those reported in previous trials involving the use of stem cells from umbilical cord blood delivered through the blood stream, confirming that stem cell transplant through the spinal canal is a minimally invasive, safe, and effective route.
The results suggest that BMMCs transplantation can be a safe and effective therapy to improve motor function and muscle tone in children with CP related to jaundice.
The team noted, however, that “these findings should be confirmed in larger, multicenter, placebo-controlled trials.”