April is World Autism Awareness Month, and as such, it’s a terrific opportunity to “shine a light on autism” and spread awareness of the disease and some of the advances being made in treating it.
There are approximately 2 million people in the United States with autism spectrum disorder (ASD), a group of complex disorders of brain development. These disorders are characterized, in differing degrees, by difficulties in social interaction, verbal and nonverbal communication and repetitive behaviors. Currently, there are treatment and education approaches that can address some of the challenges associated with ASD, but there is no cure. But there is hope as new stem cell treatments are having a demonstrable positive impact on treating patients with autism.
In a clinical trial completed in August 2013, 37 children with autism were divided into three groups: 14 subjects received cord blood mononuclear cells (CBMNC) transplantation and rehabilitation therapy; nine subjects received both CBMNC and umbilical cord–derived mesenchymal stem cell (UCMSC) transplantation and rehabilitation therapy; and 14 subjects received only rehabilitation therapy. The investigators hypothesized that infusion of a patient's own umbilical cord blood (UCB) can offer neural protection and repair in the brain and reduction of inflammation associated with this disorder.
The results went a long way in confirming the original hypothesis. The results showed that transplantation of CBMNCs demonstrated efficacy compared to the control group; however, the combination of CBMNCs and UCMSCs showed larger therapeutic effects than the CBMNC transplantation alone.
Geraldine Dawson, Ph.D. & Joanne Kurtzberg, M.D.
In June 2014, Duke Medicine was awarded $15 million to support an innovative research program to explore the use of umbilical cord blood cells to treat autism, stroke, cerebral palsy and related brain disorders. The project was headed up by Joanne Kurtzberg, M.D., chief scientific and medical officer of Duke’s Robertson Cell and Translational Therapy Program, and Geraldine Dawson, Ph.D., director of the Duke Center for Autism and Brain Development and Professor of Psychiatry and Behavioral Sciences.
The project comprised a series of clinical trials using umbilical cord blood cells to treat a total of 390 children and adults with autism, 100 children with cerebral palsy and 90 adults with stroke. Based on previous research, Kurtzberg and Dawson hypothesize that cord blood may promote repair of dysfunctional or damaged areas of the brain.
The initial phase of the program begun in June 2014. It was a preliminary trial involving 25 pediatric subjects with autism. It used their own banked cord blood for intravenous transfusion. The study measured more than a dozen results including changes in repetitive behavior, sensory experience, expressive one-word picture vocabulary, and attention to social stimuli.
Promising Initial Results
The initial results showed great promise: among 25 children ages 2–5, more than two-thirds appeared to show improvements in speech, socialization, and eye contact as reported by parents and assessed by researchers. We previously shared early news of these findings, including videos of some of the improvements seen in participating autistic children. One such child in those video had an IQ that went from 54 to 82 and a language score that rose from 62 to 97.
In addition, CNN did a video of one family's story, detailing the results one couple saw with their daughter Gracie.
More To Come
The researchers at Duke are now leading a larger, controlled Phase II clinical trial to determine whether the initial suggestion of benefit to children with autism spectrum disorder can be replicated. They hope to develop cell-based therapies that can potentially restore brain function in people with the disorders for which there currently are no cures. If successful, the study could identify therapies for further evaluation in clinical trials to potentially decrease disabilities and improve the quality of life for millions of children and adults. The trial is expected to be complete in 2019.
At Cryo-Cell International, we are lighting it up blue to support autism research and to show people with ASD that they are not alone. We hope and support the continuation of research using umbilical cord blood and cord tissue stem cells to treat not only autism but other neurological conditions as well. So don your blue this month.