NIH Awards Stem Cell Grant for the Study of Autism

BusinessWire.com, August 11, 2009

The National Institutes of Health (NIH) have announced the awarding of a $3 million grant to Children's Hospital of Orange County (CHOC) in California to study stem cells in autistic children.

More specifically, ordinary fibroblasts (skin cells) will be derived from the autistic children, which will then be reprogrammed and de-differentiated into iPS (induced pluripotent stem) cells which in turn will be re-differentiated into neural cells. Scientists hope that properties of the neural cells will shed some light on the unique characteristics of autism, thereby ultimately leading to more efficacious forms of treatment for the disorder.

Funding from the NIH grant is to be distributed over 5 years and will result in the first repository of neural cells derived from living patients. Prior to the recent development of iPS technology, the only way to derived neural cells from someone was via autopsy after death. Now, however, any living person can easily donate an ordinary skin cell, which, from the intermediate stage of an iPS cell, can be reprogrammed into virtually any type of cell found within the human body.

The grant was awarded through the Eunice Kennedy Shriver National Institute of Child Health and Human Development. The specific recipient of the grant at CHOC is the National Human Neural Stem Cell Resource program, and the award constitutes the largest federally funded basic science research grant that any department within CHOC has ever received.

According to Dr. Philip Schwartz, principal investigator and founding director of the National Human Neural Stem Cell Resource at CHOC, "This is a completely novel approach to studying the neurobiology of autism and the first time we'll be able to do so with neural cells actually derived from large numbers of children living with the condition. We hope to confirm over the next several years that neural cells generated from these donated fibroblasts can provide a viable experimental model that will yield insights about the origins, diagnosis, and treatment of autism."

Autism spectrum disorders (ASD) constitute the fastest growing developmental disability in the United States, with approximately one child in every 150 being diagnosed with some form of ASD. According to the Autism Society of America, the rate of diagnosis increases between 10 and 17% every year. Although an indisputable, definitive etiology has not yet been proven, a certain amount of controversy continues to surround ASD, as a growing amount of evidence seems to implicate environmental causes as the initiating trigger of ASD, especially heavy metals such as the mercury that is traceable directly to thimerosal. Also known as sodium ethylmercurithiosalicylate (C9H9HgNaO2S), thimerosal was originally developed as an antiseptic and antifungal agent but continues to be routinely used as a preservative in childhood vaccines. The controversy surrounding thimerosal, however, and its possible link to autism, has resulted in the gradual phasing out of thimerosal in the U.S., the European Union and a few other countries. Nevertheless, by 2007 it was estimated that more than 5,000 families in the U.S. have filed claims in a federal vaccine court alleging that their children became autistic as a result of having been vaccinated with thimerosal-containing vaccines. Although most of the cases are still being adjudicated, the U.S. federal government did award damages in one case, to the family of a young girl with the pre-existing condition of mitochondrial enzyme deficiency, who subsequently developed autism after receiving a series of thimerosal-containing vaccines. Many parents regard this ruling as confirmation that thimerosal is indeed a cause of autism.

A number of prominent scientists have researched the autism-vaccine connection extensively, perhaps the most notable of whom was Bernard Rimland, Ph.D., of San Diego, who founded the Autism Society of America in 1965 and who also founded the Autism Research Institute (ARI) in 1967, which he directed until his death from cancer in 2006. Inspired by his autistic son, Mark, who was born in 1956 when autism was quite rare, Dr. Rimland dedicated the remainder of his life to finding a scientific explanation and cure for the disorder. After more than 40 years of research, he became increasingly convinced that the causes of autism can be traced directly to the increasing number of vaccines that are prescribed in childhood. Dr. Rimland was one of the first scientists to correlate the increasing number of autism diagnoses per year to the presence of mercury in vaccines, pointing out that in 1990 autism was diagnosed at a frequency of one child per every 10,000 (ten thousand) children, whereas by 2004 the frequency had risen to one child per every 150 children, which represents an increase of 6,670%. A prolific author and energetic lecturer on autism, Dr. Rimland garnered the attention of Hollywood who invited him to serve as primary technical advisor to the 1988 movie "Rain Man", in which Dustin Hoffman's character portrayed the disorder, thereby initiating a global awareness of autism at a time when it was not nearly as prevalent as it is today. Although Dr. Rimland was often at odds with the conventional medical establishment, to this day the ARI data bank, which Dr. Rimland created, remains the world's largest data bank on autism, containing over 40,000 detailed case histories of autistic children from more than 60 countries. Dr. Rimland's 1964 book, "Infantile Autism: The Syndrome and Its Implications for a Neural Theory of Behavior", remains a seminal, ground-breaking book in the field, and is credited by many with having changed the prevailing medical view of autism at that time from a disorder that was regarded as purely psychiatric in nature, to one that is now recognized as being distinctly biological.

Families of autistic individuals who are interested in donating cells to the CHOC for study under the NIH grant should contact CHOC directly for further information.