Stem Cell Therapy for Autism
Autism is a developmental condition that affects a person’s brain development, including communication and social skills. Symptoms of this disorder often appear during the first three years of life. Many different types of autism exist, which is why the many levels are grouped under Autism Spectrum Disorders (ASD). The prevalence rate is 1 in 15,000, but some research reports indicate these conditions are more common. According to the Center for Disease Control CDC), 1 in 88 live births result in ASD, which is a 78% increase from 2002 to 2008.
Because ASD is on the rise, researchers are exploring treatments with stem cells. Mesenchymal stem cells (MSCs) act through varying mechanisms. Stem cells stimulate plastic response in host damaged tissues, restore synaptic transmitter release by offering local reinnervations, secrete growth factors, integrate into new nerve and synaptic networks, and reestablish functional efferent and afferent connections. MSCs also secrete interleukins and other substances that offer benefit to the treatment of autism. MSCs also have a strong immunosuppressive capacity and inhibit pro-inflammatory cytokine production.
A research safety trial out of Duke University involved treating children with autism with IV stem cells. Each child received 1-2 billion stem cells through an IV. After a 6-month evaluation, some children were speaking better, having meaningful communication, and had less repetitive behaviors than when they started the study. Because this was only a safety trial, researchers started a larger trial involving 165 children ages 2 to 8 years. Another study from this organization found that children treated with umbilical cord blood stem cells had increased communication, decreased sensory sensitivity, decreased social withdrawal, and less repetitive behaviors following treatment.
Studies involving MSCs and Autism
In post-mortem brains of ASD patients, researchers have found much evidence of abnormal functioning of the cerebellum. ASD subjects also have fewer Purkinje cells in the brain compared to their unaffected counterparts. Autism is known to be associated with dysregulation in growth and plasticity of dendritic spine morphology, and scientists are exploring restoration of brain function through stem cell replacement. In one study, SCs were able to promote synaptic plasticity and full functional recovery of cerebellar Purkinje cells. In addition, other studies suggest that granulocyte colony-stimulating factor was able to mobilize MSCs into peripheral blood.
In one mice study, MSCs were integrated into damaged brain tissue. Researchers noted that the MSCs migrated to the sites of injury and began active tissue repair. Recently, a controlled clinical trial was used to assess efficacy and safety of human cord blood stem cell transplantation in children with autism. The autistic children were followed for 24 weeks, and the treatment had no side effects or associated adverse events. Based on findings, stem cells caused improvements in emotional, visual, and intellectual responses, adaption to change, body use, fear/nervousness, non-verbal communication, and activity level, as recorded by the Childhood Autism Rating Scale.
A long-term evaluation study conducted recently found that intrathecally transplanted autologous bone marrow stem cells improved quality of life for a 14-year-old boy with autism. The cells were also found to be safe, and showed immediate improvement only after a week regarding eye contact and attention, as well as fine motor activities. Over a year’s time, significant improvements were noted in impulse control, social interaction, emotions, tracing, and hyperactivity. In addition, transplanted stem cells were found to ameliorate neural hypoperfusion, which is related to hypoxia, neurotransmitter dysregulation, and abnormal metabolites.
A study involving 32 people with autism was conducted in an open-label, proof-of-concept fashion. The researchers used autologous bone marrow-derived stem cells. The injected intrathecally the stem cells, which were found to be safe. Clinical improvements were noted following stem cell therapy involving socialization, emotional responsiveness, behavior, and communication. In addition, the authors found that the stem cells restored function to ASD patients by way of immunomodulation, neural circult reconstruction, and neural plasticity.
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Ichim TE, Solano F, Glenn, E, et al. (2007). Stem cell therapy for autism. J Transl Med, 27(5), 30.
Siniscalco D, Sapone A, Cirillo A, et al. (2012). Autism Spectrum Disorders: Is Mesenchymal Stem Cell Personalized Therapy the Future? J Biomed Biotech.