A new study is offering hope for families impacted by Autism. Twenty-five children took part in the first-of-its-kind study at Duke University in Durham, North Carolina. The goal was to see whether a transfusion of their own umbilical cord blood containing rare stem cells could help treat their autism. The results were impressive. More than two-thirds of the children reported improvements. A larger second trial is underway and researchers hope it will lead to long-term treatment for children with autism.
Skeptics say there are too many uncertainties to get excited. Even Duke researchers acknowledge as much. The initial trial, published Wednesday in the journal Stem Cells Translational Medicine, was a safety study. It was not a controlled, double-blind study with definitive proof of positive results. This study was open-label, which means everyone knew that the therapy was being administered.
For some participants, the changes have been monumental. Are the changes a result of the cord blood transfusion stimulating their brains? Or did their brains just mature as they got older? Could it be that their parents were subconsciously determined to magnify the improvements, given all that these families had been through?
Those are questions still being asked. What they do know is that the transformations appeared to begin about six months after transfusion and have continued since.
A freezer deep inside the Carolinas Cord Blood Bank at Duke University Medical Center, known as a thermogenesis freezer, stores up to 3,640 units of cord blood — left over from babies’ umbilical cords and placenta — at minus 196 degrees Celsius.
Each unit is designated by labels with specially designed adhesive to withstand extremely cold temperatures for decades. There are 14 cord blood freezers in all. It is the cord blood in those freezers — stored or donated by parents in case a serious illness develops — that’s at the cutting edge of this research.
Dr. Joanne Kurtzberg, who heads the Robertson Clinical and Translational Cell Therapy Program, has teamed up with Dr. Geraldine Dawson, director of the Duke Center for Autism and Brain Development. Both saw a great need for medical advances to help treat children with autism. An estimated one in every 68 children in America has some form of autism spectrum disorder, according to the Centers for Disease Control and Prevention. About 30% never learn to speak, and many children even with early behavioral interventions still struggle to adapt. There also are no FDA-approved medications that improve the core symptoms of autism.
Over the past two decades, Kurtzberg had seen children with inherited metabolic disorders be treated with cord blood after receiving high doses of chemotherapy.
“We’ve been able to show that with some of these diseases, a cord transplant rescues them from death and also improves their neurological outcome,” she says.
She began wondering if cord blood could help other children.
About a decade ago, her laboratory began clinical tests of children with cerebral palsy whose parents had banked their cord blood. Again, they saw positive results. And in some of those children who had autistic tendencies, they saw autistic symptoms improve. Another spark went off. What if they tested cord blood specifically for autism?
The safety trial began a little over a year and a half ago. Not only did it find cord blood to be safe, but 70% of the 25 children, age 2 to 6, had behavioral improvements as described by their parents and tracked by the Duke researchers. The research is largely funded by a $40 million donation from the Marcus Foundation, a nonprofit created by Home Depot co-founder Bernie Marcus.
The children traveled to Duke three times over the course of a year. They underwent a series of evaluations such as autism assessments, MRIs and EEGs to track their brain activity. On the first trip, the children received the cord blood infusion along with the intense evaluations. Each child received 1 billion to 2 billion cells, given through an IV in their arms or legs. At six months and then a year later, the children returned for more tests and observations.
“Some children, who were not speaking very much, had big increases in their vocabulary and their functional speech,” Kurtzberg says. “Many children were able to attend to play and have meaningful communication in a way that they weren’t before. Some children had less repetitive behaviors than they did when they came into the study.”
Adds Dawson, “The study was very encouraging. We did see positive results. However, it did not have a comparison group, which is very important in establishing whether a treatment is actually effective.”
Both researchers can’t stress that enough: that although they’re cautiously optimistic about the results, they want the science to play out. They are now in the midst of the definitive trial on whether cord blood can treat autism — a double-blind, placebo-controlled trial involving 165 autistic children, ranging in age from 2 to 8. The FDA has oversight of the study. During the phase II study, the children on their first visit receive a cord blood infusion — either their own or from a donor — or they get a placebo. They also undergo a battery of assessment tests and brain monitoring. On their second visit six months later, the children receive a second infusion with whatever preparation they did not receive the first time and undergo more evaluations. The order of the infusions is not known. Researchers will monitor them for the next year for any sign of behavioral improvements.
It’s known as a crossover trial, in which each subject gets the treatment and the placebo but in a different order. Researchers say it would have been nearly impossible to find participants if parents knew that their children might not receive an infusion.
“If we can show that receiving an infusion of cord blood is more effective for improving social behavior than the placebo,” Dawson says, “then this will be game-changing.”
Kurtzberg adds, “We’ll be extraordinarily encouraged if the second trial shows that the cells benefit children when the placebo does not. We will consider that a breakthrough.”
Kurtzberg has a hypothesis about what may be happening. Certain immune cells within the cord blood are crossing the blood-brain barrier and altering brain connectivity while also suppressing inflammation, which may exist with autism.
“I feel more confident now because of our (cerebral palsy) study, which preceded this study and does show benefits,” Kurtzberg says.
Families raising children with autism are in desperation. They long for their children to have a shot at a normal life. They want to see their children succeed. If the current study leads to similar success, it could change the lives of autistic children and their families everywhere.