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Researchers announced Tuesday the beginning of a FDA-approved clinical trial that uses umbilical cord blood stem cells to ‘cure’ autism.
Dr. Michael Chez, director of pediatric neurology at Sutter Neuroscience Institute in Sacramento, Calif., said he and his colleagues have been processing the trial for more than a year now, and they have high hopes it will succeed.
“What we are looking at, is cases that don’t have an obvious genetic link,” Chez told FoxNews.com. “Patients that we presume something went wrong with their brains, which caused a change to autistic features.”
In other words, the trial’s patients will essentially have no reason to have autism – or at least no genetic markers for the disease. This means they must have presumably developed it through another factor, such as the environment or exposure to an infection.
Inspired by Rydr
Chez got the idea to ‘treat’ autism with cord blood stem cells when he observed the cells make a big difference for a little boy who had cerebral palsy.
Elisa Rudgers of Sacramento, Calif., had a normal pregnancy – but a difficult delivery. Her son, Rydr, was stuck for seven hours in the birthing canal, so doctors used a vacuum to remove him. At birth, he was not moving or breathing, and he had no lower brain function. Rydr spent six weeks in the neonatal intensive care unit, and was diagnosed with cerebral palsy at 11 months old..
Rydr’s parents chose to bank his cord blood at birth with CBR – a cord blood storage company based in Arizona that will provide Chez with participants for his trial.
Rydr received his first infusion at 15 months old. This was a child who couldn’t walk, talk or eat on his own – but he began crawling three months later.
After the second infusion, he began walking and talking, and after the third infusion, Rydr was also able to chew and swallow soft foods.
“I can verify he definitely got better,” Chez said.
Now, Chez wants to put Rydr’s experience to work for children with autism.
Autism is a developmental disorder that appears in a child’s first three years of life, according to the National Institutes of Health. One in 88 U.S. children have it, and it affects one in 54 boys. The condition impacts the brain’s normal development of social and communication skills – sometimes mildly, sometimes extremely.
More children will be diagnosed with autism this year than with AIDS, diabetes and cancer combined, according to AutismSpeaks.org.
“We want to see if there is any benefit to giving them an infusion to redirect the nervous system cells, which may have programmed themselves due to a secondary factor,” Chez said.
“This may work on autism on different mechanisms in theory by modifying either the immune system or modifying the nervous system by indirect or direct methods.”
Putting the cord blood to work
Chez will give 30 kids infusions of their own cord blood cells. Most of the children for the trial have already been lined up, and they have been screened to make sure they don’t have any other issues that may have caused their autism (for example, Fragile X syndrome, stroke, head injury or prematurity).
Using the child’s own cord blood will make the study safe and ethical – plus, the cells are younger and have not been exposed to environmental factors, like viruses or chemicals, which can alter the cell’s function and structure. By using the children’s own stem cells, their bodies cannot reject them.
However, in the future, a sibling’s cord blood could theoretically work – as long as the siblings shared the same blood type.
Researchers can develop further goals when and if they see results.
“The next level of research will be to determine how that works,” Chez said.
Everyone in the pilot study will get a dose of cord blood, however; the children will be divided into two groups. Group 1 will receive an initial infusion of cord blood, while Group 2 will receive a placebo saline infusion. The groups will be observed over the course of six months, and then the groups will reverse: Group 1 will receive a dose of saline and Group 2 will receive the cord blood cells.
Chez is hoping to see results of the study within six weeks. Rydr is now an average 4-year-old boy with some limitations and a cognitive ability at grade level.
Autism studies involving stem cells are nothing new – several have already occurred overseas. However, they weren’t necessarily controlled studies, or the stem cells may have come from an open pool of donors.
“The data is scattered with glimmers of potential benefit,” Chez said. “There are also patients who have gone to other countries and gotten so-called cord blood on their own; and we’re getting anecdotal Internet studies that we don’t want to be misinterpreted. We want to do this in a scientific way and make sure it’s not just these parents are so hopeful, they are seeing a placebo effect.”
If the results are that dramatic and turn the children’s symptoms around rapidly, then Chez feels the stem cells will be ‘talking’ to other cells within the body, causing those cells to become more functional than dysfunctional.
“In theory, it would take more than that time for the nervous system cells to grow,” he said. “It might be more than growing tissue. It may be a simple infusion, (and) it doesn’t have to travel to your brain.”
Heather Brown, vice-president of scientific and medical affairs at CBR, said it is exciting to partner with researchers and clinicians like Chez, “who are pursuing a scientifically-sound approach in evaluating new therapeutic uses for cord blood stem cells for conditions that currently have no cures.”