A first-of-its-kind double-blinded, crossover study of children with autism who were treated with their own cord blood showed the treatment to be safe and suggests improvement in socialization.
The study, which was published this week in the medical journal Stem Cells Translational Medicine, involved 30 children and was conducted by Michael Chez, M.D., the primary investigator who designed the study that was carried out through the Sutter Neuroscience Institute of Sacramento and the Sutter Institute for Medical Research, with research grant funding by Cord Blood Registry® (CBR®). It was the first randomized, double-blinded, placebo-controlled trial performed in the United States to assess the feasibility of treating autistic children by using their own newborn cord blood harvested from their banked umbilical cord. The study can be viewed here.
One of the most exciting outcomes of the study, said Dr. Chez, who is with Sutter Neuroscience Institute, involved successful treatment and blinding of parents and investigators, and safety and tolerability of the treatment in this autistic patient population. Trends toward improvements in areas such as socialization and language were observed.
“The Vineland Adaptive Behavior Scale for Socialization, one of the tests commonly used to measure real-world functional abilities, showed significant improvement after 12 weeks of cord blood treatment over placebo,” Dr. Chez said. However, when correcting for possible statistical chance when looking at many outcome variables, this observed significance no longer reached significance, probably due to variables such as only 30 patients in this pilot research study.
The study also mirrored a recent non-blinded, open-label Duke University trial that showed similar Vineland Socialization score improvements while also demonstrating that the use of autologous cord blood is safe for the children through intravenous infusions.
Parents contacted about the study were pleased with the improvements they saw in their children.
Jennifer Lundberg of Orlando, Fla., said she knew almost immediately when Hayden received the cord blood infusion as opposed to the saline.
“We had been trying for more than four years to have him potty-trained, and within two weeks he started to self-potty. We never thought this was possible,” she said. “He also now has this global understanding of language that he didn’t have before. For example, I would ask him to open up the air-conditioning vents, and he all of a sudden knew what to do. He doesn’t have the same outbursts that he used to have. It is truly amazing. If we could do it again, we definitely would do it again.”
Another mother who asked not to be identified said her young son also improved. “We saw improvements in his overall development – in his language and social interaction,” she said. “It definitely helped.”
CBR, which funded the study, has long been committed to supporting research on the use of cord blood to treat a variety of diseases from cerebral palsy and autism to hearing loss and Type I diabetes.
“The outcome of Dr. Chez’s study, in addition to other recent research advances looking at the use of cord blood for children with autism, are encouraging,” said Heather Brown, vice president of scientific and medical affairs, CBR. “As researchers continue to explore cord blood’s role in regenerative medicine, CBR remains dedicated to investing in clinical trials to better understand this potential and investigating ways we can increase cord blood’s utility.”
Sutter Neuroscience Institute and Sutter Institute for Medical Research are affiliates of Sutter Health, Northern California’s largest health provider. Sutter Health was named one of the nation’s top five large health systems in 2016 thanks to its integrated network of physicians, employees and volunteers—more than 60,000 people strong. Rooted in Sutter Health’s not-for-profit mission, these team members partner to deliver exceptional care that feels personal. From physician offices to hospitals to outpatient care centers and home services, they proudly support and learn from the more than 3 million people in their care—nearly 1 percent of the U.S. population, in one of the most diverse and innovative regions in the world.