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Groundbreaking Autism Clinical Trial Regarding Cord Blood Stem Cells

  • Published: 2012-08-25 (Revised/Updated 2018-03-15) : Author: Sutter Neuroscience Institute
  • Synopsis: First FDA approved clinical trial to evaluate use of childs own cord blood stem cells to treat patients with autism.
Stem Cells

Distinguished from other cell types by two important characteristics. First, they are unspecialized cells capable of renewing themselves through cell division, sometimes after long periods of inactivity. Second, under certain physiologic or experimental conditions, they can be induced to become tissue or organ-specific cells with special functions. Stem cells have the remarkable potential to develop into many different cell types in the body during early life and growth. In addition, in many tissues they serve as a sort of internal repair system, dividing essentially without limit to replenish other cells as long as the person or animal is still alive. When a stem cell divides, each new cell has the potential either to remain a stem cell or become another type of cell with a more specialized function, such as a muscle cell, a red blood cell, or a brain cell.

Main Document

"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."

Autism and Cord Blood Stem Cells: FDA Gives Green Light for Groundbreaking Clinical Trial. Sutter Neuroscience Institute, a recognized Center of Excellence, and CBR (Cord Blood Registry), the world's largest stem cell bank, are launching the first FDA- approved clinical trial to assess the use of a child's own cord blood stem cells to treat select patients with autism. This first-of-its-kind placebo controlled study will evaluate the ability of an infusion of cord blood stem cells to help improve language and behavior. The study is in conjunction with the Sutter Institute for Medical Research.

According to the Centers for Disease Control and Prevention, autism spectrum disorders impact one in 88 children in the U.S., and one in 54 boys.1 The condition is thought to have multiple risk factors including genetic, environmental and immunological components.

"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," said Michael Chez, M.D., director of Pediatric Neurology with the Sutter Neuroscience and principal study investigator. "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."

The study will enroll 30 children between the ages of two and seven, with a diagnosis of autism who meet the inclusion criteria for the study. Enrolled participants will receive two infusions - one of the child's own cord blood stem cells and one of a placebo - over the course of 13 months. Both the participants and the lead investigators will be blinded from knowing the content of each infusion. To ensure the highest quality and consistency in cord blood stem cell processing, storage and release for infusion, CBR is the only family stem cell bank providing units from clients for the study. For information on study, visit

Study Rationale

A newborn's umbilical cord blood contains a unique population of stem cells that 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 these conditions, the stem cells effectively rebuild the blood and immune systems.

"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," continued Dr. Chez. "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."

"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," said Heather Brown, vice president of scientific & medical affairs at CBR. "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."

The co-investigator of the study is Michael Carroll, M.D., medical director of the Blood and Marrow Transplantation and Hematological Malignancies Program at Sutter Medical Center, Sacramento.

"There is a vast amount of unchartered territory when it comes to how stem cell therapies may help patients living with these conditions," said Dr. Carroll. "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."

1 Centers for Disease Control and Prevention, Autism Spectrum Disorder, Data and Statistics,, accessed May 2012

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