Stem Cells and Autism

Umbilical Cord Blood stem cells have long been used to help treat multiple diseases. Dr. Kurtzberg has been at the forefront of this research.  The spectrum news report below notes: 

In 1990 Dr. Kurtzberg noticed cord blood transplants in patients with autism extended their lives, prevented brain deterioration, and improved 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.

More research is needed to use adult or umbilical cord blood stem cells routinely in clinical care, but the future is exciting about this potential “natural” treatment option. 


Joanne Kurtzberg, MD

Professor, Department of Pediatrics; Chief Scientific Officer, Robertson Clinical and Translational Cell Therapy Program
Dr. Joanne Kurtzberg is an internationally renowned expert in pediatric hematology/oncology, pediatric blood and marrow transplantation, umbilical cord blood banking and transplantation, and novel applications of cord blood in the emerging fields of cellular therapies and regenerative medicine. Dr. Kurtzberg pioneered the use of umbilical cord blood as an alternative stem cell source for unrelated hematopoietic stem cell transplantation (HSCT). Over the last two decades Dr. Kurtzberg has established an internationally known pediatric transplant program at Duke which treats children with cancer, blood disorders, immune deficiencies, hemoglobinopathies and inherited metabolic diseases. In 2010, Kurtzberg established the Julian Robertson Cell and Translational Therapy Program (CT2) at Duke. CT2 focuses on translational studies from bench to bedside with a focus on bringing cellular therapies in regenerative medicine to the clinic. Recent areas of investigation in CT2 include the use of autologous cord blood in children with autism spectrum disorder, neonatal brain injury and cerebral palsy, as well as preclinical studies manufacturing oligodendrocyte-like cells from cord blood to treat patients with acquired and genetic brain diseases. Studies of autologous bone marrow ALDHbright cells in adults with stroke and radiation induced brain injury are also underway.
Dr. Kurtzberg established one of the largest unrelated donor cord blood bank, the Carolinas Cord Blood Bank, in the world at Duke in 1998. The bank has a current inventory of >40,000 units and has provided cord blood units to over 2,200 patients undergoing unrelated donor HSCT over the past 10 years. Dr. Kurtzberg’s lab has developed novel assays to predict cord blood potency from segments attached to cryopreserved cord blood units, and is performing translational research testing cord blood expansion, cellular targeted therapies and tissue repair and regeneration. In 2012, under the direction of Dr. Kurtzberg, the Carolinas Cord Blood Bank received FDA approval for DuCord, a stem cell product derived from umbilical cord blood, for use in transplants between unrelated donors and recipients. Dr. Kurtzberg currently holds several INDs for investigational clinical trials.
Dr. Kurtzberg has published over 300 peer-reviewed papers, multiple chapters and scientific reviews. She is a member of the American Society of Hematology, the American Association of Blood and Marrow Transplantation, the American Society of Pediatric Hematology/Oncology, the International Society of Cellular Therapies, the Pediatric Blood and Marrow Transplant Consortium (PBMTC), and other organizations. She serves on the Board of the Foundation of Accreditation of Cellular Therapies, co-chairs the National Marrow Donor Program’s Cord Blood Advisory Group and is a member of the Advisory Council of Blood Stem Cell Transplantation to Health and Human Services. Dr. Kurtzberg was awarded a Lifetime Achievement Award from the PBMTC in 2012.

From hope to hype:

Scientific interest in stem cell therapies for autism began from one doctor’s 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 Broxmeyer’s 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 foundation’s 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 autism’s 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 child’s 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 trialat 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 don’t want to promote a therapy that’s not helpful,” she says.
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