StemCell Therapy

Scientific interest in stem cell therapies for autism began from one doctors observation.

In the late 1980s, Joanne Kurtzberg, a pediatrician specializing in blood cancers, worked with Hal Broxmeyer, who had been studying the stem cells present in blood from umbilical cords preserved at birth. Since the 1950s, doctors have treated blood cancers using transplants of bone marrow, which contains adult stem cells capable of generating blood cells. But as Broxmeyers group showed, cord-blood stem cells are more potent than adult stem cells at this task. Of particular value, they do not require a perfect match between donor and recipient.

Kurtzberg recognized the promise of cord blood for people of color, who often have trouble finding matching bone-marrow donors. So, in 1998, she founded the Carolinas Cord Blood Bank at Duke University and, in 2005, helped pass legislation to establish a national network of similar banks. Every year in the U.S., about 200,000 women donate cord blood from their babies to these public banks or to research labs; families also pay to store up to 7 million units of cord blood in private banks worldwide, though these samples rarely end up being used. In 2011, the FDA began requiring companies selling therapies developed from cord blood to submit data showing their safety and effectiveness.

In the late 1990s, Kurtzberg says she noticed something curious following cord blood transplants in children with certain metabolic conditions who also have autism traits. The transplants not only extended their lives but also seemed to prevent brain deterioration and improve brain function. Kurtzberg theorized that the stem cells in the cord blood might be engrafting in the brain and repairing damaged tissue. Other cells in the cord blood might also be sending chemical signals across the blood-brain barrier to lower inflammation, which is seen in some people with autism, she says.

To test the inflammation theory, Kurtzberg began collaborating with Geraldine Dawson, an autism researcher also at Duke. In 2014, they launched a clinical trial with $40 million in funding from the Marcus Foundation, a nonprofit based in Atlanta that supports a variety of causes. The foundations head, Home Depot co-founder Bernie Marcus, has called himself a real advocate for stem cells for autism, and he reportedly told the controversial leader of the Panama stem cell clinic the Perskins visited that he hoped to get approval from the FDA.

The Duke trial drew immediate criticism. In 2014, Arnold Kriegstein, a neural stem cell expert at the University of California, San Francisco, told Spectrum the study was a Hail Mary pass and that it was unlikely cord-blood stem cells could reverse autism-related changes to the brain during early development. None of their explanations hold water on why it would have any therapeutic value, Kriegstein now says. Paul Knoepfler, a researcher at the University of California, Davis who blogs about the stem cell industry, also expressed skepticism, noting that autisms causes are too diverse and mysterious for the therapy to make sense.

Still, the Duke team published encouraging results from the safety phase of the trial in 2017. Clinicians working with the researchers evaluated 22 of the participants, all between the ages of 2 and 5 years, and documented improvements in 13 of them six months after a single infusion. Without a control group, it is impossible to say whether the children would have improved anyway. A follow-up report found that those who improved showed increased connectivity in brain regions affected by autism, including the limbic system, but the researchers published no evidence to support their inflammation theory.

Because there were no adverse effects from the infusions, in November 2017 the FDA granted Kurtzberg special approval to provide cord blood infusions to certain children, including some with autism. Kurtzberg declined to say how much the treatments cost but confirmed that the parents from other countries are required to put down a $15,000 deposit; the money covers a 45-minute intravenous infusion of their childs own cord blood or that of a matched sibling. Kurtzberg says her team plans to treat about 1,000 children per year, and that they have a waiting list of more than a year. She expects to publish the results of a placebo-controlled phase of the trial sometime this year, comparing children who receive cord blood infusions with those who do not.

While the Duke trial pushes forward, others have sputtered. A 2018 clinical trial at the Sutter Institute of Medical Research in Sacramento, California, found minimal evidence of clinical effectiveness of cord blood infusions given to 29 autistic children. The National Institutes of Health clinical trials registry lists 11 other autism-related stem cell trials but 3 concluded years ago without registering any results, 3 are listed with an unknown status, and 2 have been withdrawn. Most are outside the U.S., and none randomized the participants to receive the therapy or placebo, which is considered the gold-standard approach in clinical trials. Some may even be pay-to-participate trials, which are little more than marketing ploys to lure customers, Turner says.

Kurtzberg acknowledges that her research has led to a boom in questionable stem cell providers but says her team members do their best to tell families to tread carefully. We dont want to promote a therapy thats not helpful, she says.

Excerpt from:
False hope for autism in the stem-cell underground ...

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