In order to develop a better understanding of the neurobiology of autism, Children's Hospital of Orange County (CHOC), in conjunction with the Autism Speaks' Autism Tissue Program, is utilizing a new technology to obtain neural progenitor cells from postmortem tissue. These cells will mature into various types of brain cells in the laboratory, allowing study of the processes thought to take place in the maturing brain.

Philip Schwartz, the lead researcher and director of the National Human Neural Stem Cell Resource Center at CHOC, recently developed the new technology with his team. Studying brain cells from individuals with autism will allow researchers unprecedented ability to define the biochemical and genetic mechanisms of brain cell development and maturation. The unique ability for researchers to observe these neural progenitor cells maturing and developing into different brain cell types makes them a useful tool for studying this neurodevelopmental disorder. Schwartz has already been successful in establishing living cell cultures from postmortem brains tissue of donors with Rett and Fragile X Syndrome.

“When examining maturing brain cells, if we understand what happened and when, and how and where the cells started to change, then we will have the answer to the underlying cause, such as excessive protein levels, gene mutations or environmental triggers,'' Schwartz said. “Only then can we begin to look at developing a treatment. Our ultimate goal will be to be able to repair or prevent the damage.”

In addition to investigating genetic and environmental susceptibility, neural cells being established at CHOC also have the potential to change the way drugs are developed. Instead of drug trials using animals or people, new medications could be tested on these cells in a dish. This has the potential to help scientists develop safer drugs much more quickly. Currently, there are no pharmacological agents which treat the major symptoms of autism, forcing doctors to use drugs which are not intended for use in children and adults with this disorder. The cells created from postmortem brain tissue would provide a unique opportunity to examine specific drug treatment strategies on living human cells.

The new cell technology will now be applied to brain tissue donated to the Autism Tissue Program. Established in 1998, the Autism Tissue Program, or ATP helps investigators studying autism by coordinating brain tissue donation and ensuring that this precious resource is distributed to the best scientific projects. Unfortunately, no living cell cultures have so far been established for scientists to study. Because the cells that are obtained through this project will be available for other researchers to examine, ATP and CHOC will expand the resource through this cutting-edge technology.

This resource is not possible without the precious gift of brain tissue made by individuals and families who have chosen to donate brain tissue to fight autism. The establishment of these neural cell cultures requires fresh brain tissue. ATP is actively educating parent groups, individuals, and professionals about the need to donate brain tissue, since brain tissue donation is not generally included in organ and tissue donor programs. For more information, please visit the National Human Neural Stem Cell Resource website at www.nhnscr.org, the Autism Tissue Program web site at www.memoriesofhope.org, or call toll-free (877) 333-0999.