FDA Approves Clinical Trial that Used Cord Blood to Treat Autism

The Sutter Neuroscience Institute in Sacramento, California has announced its collaboration with the Cord Blood Registry, the world’s largest stem cell bank in what promises to be the first FDA-approved clinical trial to assess the use of a child’s own cord blood stem cells to treat selected patients with autism. This placebo-controlled study is the first of its kind and will evaluate the efficacy of cord blood stem cells to help improve language and behavior in autistic children.

The Centers for Disease Control (CDC) places the frequency of autism in the US as one in 88, but for boys, the rate is even higher (1 in 54). Autism, today, is part of a series of conditions that are collectively defined as autism spectrum disorders (ASPs). ASPs include individuals with very different symptoms, and include everything from autistic disorder (also known as classic autism, Asperger syndrome and pervasive developmental disorder not otherwise specified (also known as atypical autism). These conditions are thought to have multiple risk factors that include genetic, environmental and immunological components.

With regard to this study, Michael Chez, M.D., director of Pediatric Neurology with the Sutter Neuroscience and principal study investigator of this clinical trial said: “This is the start of a new age of research in stem cell therapies for chronic diseases such as autism, and a natural step to determine whether patients receive some benefit from an infusion of their own cord blood stem cells. I will focus on a select portion of children diagnosed with autism who have no obvious cause for the condition, such as known genetic syndromes or brain injury.”

This clinical study will enroll 30 children between the ages of two and seven who have been diagnosed with autism, and meet all the criteria for inclusion in the study. Over the course of this clinical trial, all enrolled participants will receive two infusions over the course of 13 months. One of the infusions will contain the child’s own cord blood stem cells, and the other infusion will contain a placebo. The participants and the lead investigators will not know the content of each infusion. To ensure the highest quality and consistency in cord blood stem cell processing, storage and release for infusion, Cord Blood Registry is the only family stem cell bank that provides umbilical cord blood units from clients for the study.

A newborn’s umbilical cord blood contains several unique populations of stem cells. Scientists and physicians have been used for more than 20 years in medical practice to treat certain cancers, blood diseases and immune disorders. When patients undergo a stem cell transplant for such conditions, the umbilical cord blood stem cells effectively rebuild the blood and immune systems.

According to Dr. Chez, “A focus of my research has been the complex relationship between a child’s immune system and central nervous system. We have evidence to suggest that certain children with autism have dysfunctional immune systems that may be damaging or delaying the development of the nervous system. Cord blood stem cells may offer ways to modulate or repair the immune systems of these patients which would also improve language and some behavior in children who have no obvious reason to have become autistic. The study is similar to other FDA-approved clinical trials looking at cord blood stem cells as a therapy for cerebral palsy.”

Heather Brown, vice president of scientific & medical affairs at Cord Blood Registry, said: “It’s exciting to partner with thought-leading medical researchers and clinicians, like Dr. 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. Families who made the decision to bank their stem cells to cover the unknowns and what ifs in life are gaining access to this and other important clinical trials while playing an important role in the advancement of science.”

A co-investigator of the study is Michael Carroll, M.D., who is the medical director of the Blood and Marrow Transplantation and Hematological Malignancies Program at Sutter Medical Center, Sacramento. According to Dr. Carroll, “There is a vast amount of unchartered territory when it comes to how stem cell therapies may help patients living with these conditions. I’ve seen how stem cell therapy has changed my field of medicine and how I care for my blood cancer patients. I am eager to see how our work can open new doors for patients and families dealing with autism.”

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Professor of Biochemistry at Spring Arbor University (SAU) in Spring Arbor, MI. Have been at SAU since 1999. Author of The Stem Cell Epistles. Before that I was a postdoctoral research fellow at the University of Pennsylvania in Philadelphia, PA (1997-1999), and Sussex University, Falmer, UK (1994-1997). I studied Cell and Developmental Biology at UC Irvine (PhD 1994), and Microbiology at UC Davis (MA 1986, BS 1984).

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