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Study Presents Potential New Autism Treatment Option

LITTLE ROCK, AR (Jan. 24, 2012) - A new study published by researchers at Arkansas Children's Hospital Research Institute (ACHRI) shows promise for potential treatments for children with autism spectrum disorders. The research demonstrates that children with these disorders (ASD) are more likely to have a certain type of antibody, which could lay the groundwork for testing of that antibody and then treatment that could improve the lives of children with ASD significantly. The study is published this month in Molecular Psychiatry.

Richard E. Frye, MD, PhD, director of autism research at the Arkansas Children's Hospital Research Institute (ACHRI) and associate professor of Pediatrics at the University of Arkansas for Medical Sciences (UAMS), and Jill James, PhD, director of the Autism Metabolic Genomics Laboratory and professor of Pediatrics at UAMS, served as lead authors of the study, Cerebral folate receptor autoantibodies in autism spectrum disorder, that can be read in the journal's online edition at www.nature.com/mp/journal/vaop/ncurrent/pdf/mp2011175a.pdf.

The study measured serum folate receptor autoantibodies (FRAs) concentrations in 93 children with ASD. A high prevalence rate (75.3 percent) of FRAs was found in the children participating in the study. Children with FRAs were treated with oral leucovorin calcium, and treatment response was measured and compared with a wait-list control group. Compared with controls, significantly higher improvement ratings were observed in treated children over a mean period of 4 months in the areas of verbal communication, receptive and expressive language, attention and stereotypical behavior.

"Our study demonstrates a high prevalence of an autoantibody that has the potential to cause neurological disability in children with autism spectrum disorder. This study is significant for at least three reasons: First, this autoantibody can be easily measured, so it is possible to easily identify this subgroup of autistic children. Second, our study demonstrates that a significant portion of children in this subgroup respond positively to treatment with a particular type of folate. Third, it is very possible that the autoantibody we have studied has a causative role in the development of autistic symptoms, so identifying this autoantibody early in life and intervening at that time could minimize or even prevent the development of autistic symptoms," explains Frye. "While more research needs to be done to confirm our results, we are excited about the possibility that a simple safe intervention could improve the lives of a significant number of children with autism spectrum disorder."

Edward Quadros, PhD, Department of Medicine, State University of New York - Downstate Medical Center, member of the study's research team, said of the study, "The identification of folate receptor autoantibodies in a majority of autism spectrum disorders provides for the first time a marker and a specific treatment for this condition. Considering the role of folates in fetal development, neural tube defects and cerebral folate deficiency syndrome, the detection of these autoantibodies provides a mechanism by which the pathological manifestations of these neuro-developmental disorders could occur due to blocking of folate transport to the developing brain. Identification of this autoimmune disorder and prompt intervention with pharmacological doses of folate could prevent many of the neuropathological manifestations. Researchers at SUNY Downstate were the first to identify these autoantibodies in neural tube defect pregnancy and in cerebral folate deficiency syndrome and have developed techniques for the determination of these autoantobodies in blood and are providing this test to physicians worldwide."

The study was supported, in part, by funding from the Autism Research Institute and the Jane Botsford Johnson Foundation. The study was produced by a research team that also included:

  • JM Sequeira, PhD, Department of Medicine, State University of New York - Downstate Medical Center, Brooklyn, NY
  • EV Quadros,PhD, Department of Medicine, State University of New York - Downstate Medical Center, Brooklyn, NY
  • DA Rossignol, MD, International Child Development Resource Center, Melbourne, FL

ACHRI provides a research environment on the ACH campus to meet the needs of the UAMS faculty. Research scientists at ACHRI conduct clinical, basic science, and health services research for the purpose of treating illnesses, preventing disease and improving the health of children everywhere. The laboratories of Drs. Frye and James are supported, in part, through funding from ACHRI and the Arkansas Biosciences Institute.

Arkansas Children's Hospital is the only pediatric medical center in Arkansas and one of the largest in the United States serving children. The campus spans 29 city blocks and houses 316 beds, a staff of approximately 500 physicians, 80 residents in pediatrics and pediatric specialties and more than 4,000 employees. The private, nonprofit healthcare facility boasts an internationally renowned reputation for medical breakthroughs and intensive treatments, unique surgical procedures and forward-thinking medical research - all dedicated to fulfilling our mission of enhancing, sustaining and restoring children's health and development. ACH recently ranked No. 75 on FORTUNE 100 Best Companies to Work For®. For more information, visit www.archildrens.org.

UAMS is the state's only comprehensive academic health center, with colleges of Medicine, Nursing, Pharmacy, Health Related Professions and Public Health; a graduate school; a hospital; a statewide network of regional centers; and seven institutes: the Winthrop P. Rockefeller Cancer Institute, the Jackson T. Stephens Spine & Neurosciences Institute, the Myeloma Institute for Research and Therapy, the Harvey & Bernice Jones Eye Institute, the Psychiatric Research Institute, the Donald W. Reynolds Institute on Aging and the Translational Research Institute. Named best Little Rock metropolitan area hospital by U.S. News & World Report, it is the only adult Level 1 trauma center in the state. UAMS has more than 2,800 students and 775 medical residents. It is the state's largest public employer with more than 10,000 employees, including about 1,000 physicians and other professionals who provide care to patients at UAMS, Arkansas Children's Hospital, the VA Medical Center and UAMS' Area Health Education Centers throughout the state. Visit www.uams.edu or www.uamshealth.com.

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