By Amy Norton. A study evaluating umbilical cord blood as a treatment for autism has found clues of potential benefits for some children, but the researchers note that much more work is needed to get firmer responses. Read: WhatsApp: How to put music in your states?
The study of 180 children found that a single infusion of umbilical cord blood did not improve social or communication skills in the overall group. But there were positive signs in the subgroup of children who did not have intellectual disabilities: Their communication skills improved, on average, over the next six months.
Autism. Photo: The New York Times.
The findings, published in the May 19 issue of the Journal of Pediatrics, do not support the use of umbilical cord blood outside of a clinical trial, the researchers emphasized.
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“This just gives us some hopeful data, and we are planning additional studies,” said senior researcher Dr. Joanne Kurtzberg, professor of pediatrics at Duke University School of Medicine in Durham, North Carolina.
The underlying principle of the treatment is based in part on previous laboratory research that suggested that certain cells in the umbilical cord blood, called monocytes, may moderate a type of brain inflammation seen in people with autism.
It is not clear that inflammation is the cause of autism symptoms. But there is “fairly strong evidence” of a relationship to the social communication problems that characterize the disorder, according to Kurtzberg.
Previous small studies found no adverse side effects from giving autistic children an infusion of blood from their own umbilical cord (collected at birth), or from a donation from an umbilical cord blood bank.
But whether or not it has any effect is another topic, and a difficult subject to study, Kurtzberg noted.
A basic problem is that autism cases are complex and diverse, and they vary greatly from child to child.
Some children have milder problems with socialization and communication, while others are deeply affected: they speak little, if they speak, and engage in repetitive and obsessive behaviors. Some children with autism have intellectual disabilities, while others have average or above-average IQs.
In the most recent trial, most children had some degree of intellectual disability, a higher number than the researchers expected to enroll. It also turned out to have a great placebo effect, Kurtzberg said. That is, parents of children who actually received a placebo instead of umbilical cord blood, however, frequently reported improvements in their children’s behavior.
The researchers also studied a wide age range, from 2 to 7 years. And in young children, Kurtzberg said, it is difficult to know whether any progress in social or communication skills is due to therapy or natural development.
“In the younger children, there was a significant placebo effect,” he said.
The Duke researchers had begun another study involving only children ages 4-8. But this has a downside, since it is generally believed that the earlier an intervention for autism can be performed, the better, Kurtzberg said.
Dr. Andrew Adesman is chief of developmental and behavioral pediatrics at Cohen Pediatric Medical Center in New Hyde Park, New York.
He congratulated the researchers for stating what their findings do not prove.
“The results of this study are disappointing because there appears to be no benefit for children with [autismo] lower functioning, and perhaps modest benefits for children with [autismo] in those with higher functioning, “said Adesman.
This more recent finding, he added, “would certainly have to be replicated in a very well-controlled follow-up study.”
The trial involved 180 children with autism who were randomized to one of three groups: 56 received an infusion of blood from their own umbilical cord, 63 an infusion from a genetically matched donor, and 61 received a placebo infusion.
In total, 12 children had an allergic reaction to the infusion: four were severe, with symptoms such as difficulty breathing and facial inflammation.
Regarding the benefits of treatment, overall there were no clear benefits. But when the researchers focused on children without intellectual disabilities, they found that those who had received the cord blood showed improvements in communication skills, on average.
“Some who were not verbal became verbal, and were able to express needs, like ‘I want milk,'” Kurtzberg said. “Some went from having minimal verbal communication to being able to have a conversation.”
But the results are based on a small number of children. Going forward, Adesman added, it would be helpful to have a study involving multiple research centers, and a larger group of children without intellectual disabilities.