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article imageAutism study at Duke draws skepticism from experts

By Mike Rossi     Jul 15, 2014 in Science
Durham - A bold, new study led by researchers at Duke University seeks to examine the applicability of umbilical cord stem cells for use in the treatment of autism.
The five-year, $40 million clinical trial—the first two years of which are being funded by a $15 million contribution from the Marcus Foundation—will take its direction from Joanne Kurtzberg, a doctor and professor of pediatrics at the university.
Researchers plan to study nearly 400 individuals, a combination of autistic infants and adults, to determine if either subject-banked or donated umbilical cord blood can help to reduce, or even reverse, the symptoms of autism.
Though no physicians or researchers criticize the noble intentions of the nascent program, some question the prudence of a $40 million investment given that so little is known about the therapeutic potential of stem cells on the brain, particularly with regard to a genetic disorder like autism.
“I wish we knew more about the pathogenesis of autism, which is a fancy word for saying what causes it, before we proceeded with these kinds of trials…Overall, I have very mixed feelings,” said Paul Knoepfler, a PhD and professor of Cell Biology and Human Anatomy at the University of California, Davis.
Arnold Kriegstein, the Director of Regenerative Medicine and Stem Cell Research at the University of California, San Francisco, also feels a trial of this magnitude is markedly premature. With purposeful directness, he stated “I think it would be marvelous if this trial worked, but it really seems more like a 'Hail Mary pass' than a rational therapy.”
Kurtzberg, however, intends to proceed with the trials as planned in spite of her critics, arguing that the program’s “enormous potential” outweighs the risk of starting major research without a solid foundation of empirical evidence.
The first stage of the trial, which got underway in June, features 20 children between the ages of two and five.
Doctors will apply a single dosage of the patient’s own, banked cord-blood and study the results over a non-specified timeline. The ultimate hope being that the cord-blood can diffuse through the brain’s protective barriers and develop into dedicated cells that will help restore normal function.
The study, which concludes in 2019, is currently enrolling qualified patients. Interested families can inquire further by contacting Duke at cordbloodtherapyinfo@dm.duke.edu or (844) 800-CORD or (844) 800-2673.
The full press release from Duke University is available here.
More about Autism, Stem cells, Duke, UCSF, Clinical trial
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