August 11, 2009
CHOC Children’s announced today that its Research Institute has received a five-year, $3 million National Institutes of Health (NIH) Research Grant to generate, investigate, and store neural stem cells derived from skin cells (fibroblasts) donated by children with autism. The research program is designed to provide an important new tool for studying the impact of autism on the developing brain – in a completely harmless and non-invasive way – while assembling the first repository of neural cells from living patients as a resource for the entire autism research community. Awarded through the Eunice Kennedy Shriver National Institute of Child Health and Human Development, the grant recognizes CHOC Children’s unique stem cell culture, distribution and training capabilities and is the largest federally-funded, basic science research grant the hospital has ever received.
“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,” said Philip H. Schwartz, Ph.D., principal investigator on the NIH grant and founding director, National Human Neural Stem Cell Resource (NHNSCR) at CHOC Children’s. “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.”
For some time, researchers have believed that neural stem cells might provide a critical tool for the study of autism. However, until now, the only way to obtain these cells was to harvest them from the brains of people with autism after their deaths, a project that the NHNSCR has pursued in collaboration with the Autism Tissue Program of Autism Speaks for the past several years. Recent advances in stem cell research have allowed Dr. Schwartz and his colleagues to overcome this difficulty. It is now possible to convert easily-obtained human skin cells (fibroblasts) to induced pluripotent stem cells (iPSCs), which are then capable of differentiating into virtually any tissue cell type, including neural stem cells.
The NIH-funded research at CHOC Children’s will apply this new technology to fibroblasts derived from patients whose autism is clinically very well-characterized, as well as those derived from individuals without the disorder. This efficient, non-invasive method for generating autism neural stem cells from living individuals will allow research that has never been possible before. This includes studies of the detailed properties of the induced autism neuron; studies of sufficient statistical power to allow researchers to compare and contrast the effects of autism on the developing human brain; and studies of the influence of environmental factors on these processes. Importantly, all data generated by these investigations will be made widely available to the scientific community. The cells lines themselves will also be made available through the existing CHOC-funded stem cell repository, the NHNSCR, which Dr. Schwartz created in 2001.
“This project provides a new way for children with autism and their families to participate in the next era of research through a simple donation of skin cells,” said Dr. Schwartz.
As work on the grant progresses, Dr. Schwartz’s group at CHOC Children’s will coordinate patient participation and cell collection with a clinical team headed by Randi J. Hagerman, M.D., Medical Director of the U.C. Davis M.I.N.D. Institute.
The autism spectrum disorders comprise a set of developmental brain disorders affecting social interaction and communication that remain poorly understood yet today represent the fastest growing developmental disability diagnosis in the United States. In addition to its research collaboration with Autism Speaks, CHOC Children’s has emerged as a regional leader in the diagnosis and treatment of autism spectrum disorders. In April, 2009, The Family Autism Network was created as a collaboration between two neurodevelopmental programs at CHOC Children’s and UC Irvine and the Grandparent Autism Network to provide a one-stop resource for Orange County families impacted by autism.
About the NHNSCR at CHOC Children’s
The National Human Neural Stem Cell Resource (NHNSCR) was established to support national research in the field of neural stem cells by providing a reliable source for these cells to investigators nationwide. In addition to acting as a stem cell repository, the NHNSCR also hosts a periodic course on the culture of human pluripotent stem cells and adult stem cells that has trained more than 75 scientists from around the world in these techniques. The NHNSCR plans to train autism researchers in the use of the new cell lines created under the NIH grant. More information is available at www.nhnscr.org. Inquiries about cell donation should be sent to research@CHOC.org.
About CHOC Children's: Named one of the best children’s hospitals by U.S. News & World Report (2013-2014) and a 2013 Leapfrog Top Hospital, CHOC Children's is exclusively committed to the health and well-being of children through clinical expertise, advocacy, outreach and research that brings advanced treatment to pediatric patients. Affiliated with the University of California, Irvine, CHOC’s regional healthcare network includes two state-of-the-art hospitals in Orange and Mission Viejo, several primary and specialty care clinics, a pediatric residency program, and four centers of excellence - The CHOC Children’s Heart, Neuroscience, Orthopaedic and Hyundai Cancer Institutes. CHOC earned the Gold Level CAPE Award from the California Council of Excellence, the only children’s hospital in California to ever earn this distinction, and was awarded Magnet designation, the highest honor bestowed to hospitals for nursing excellence. Recognized for extraordinary commitment to high-quality critical care standards, CHOC’s Pediatric Intensive Care Unit (PICU) is the first in the United States to earn the Pediatric Beacon Award for Critical Care Excellence.
Denise Almazan, Director of Public Relations
phone: (714) 509-8680