For parents of young children, getting an autism diagnosis can be crushing. Why my child? What challenges lie ahead? Will he or she be able to lead a normal life? The Centers for Disease Control estimates that 1 out of every 88 children suffers from an autism spectrum disorder, and for their parents, the questions are daunting. Worsening the blow is the fact that there is currently no available medication to treat the disorder’s core symptoms.

That’s why ConnectMe, an ongoing global clinical trial being held at Arkansas Children’s Hospital, is so important, according to Dr. Richard Frye, director of autism research at Arkansas Children’s Hospital. The ConnectMe clinical research program will test the safety and effectiveness of an investigational drug, memantine hydrochloride, on autism’s core symptoms: impaired communication, impaired social interactions and restricted interests and repetitive behaviors. ConnectMe is the largest-ever global clinical research program for autistic children ages 6 to 12, with more than 180 sites across the country.

According to Frye, who will lead the study locally, participants will undergo three phases of involvement. First, approximately 192 children ages 6-12 will be given a dosage of the drug, and they, their parents and their doctor will know it has been administered. If participants have a positive response (improved social function, language, etc.), they will move into phase two of the study, in which the medication is taken away from some participants. (Participants will not know whether their medication has been replaced with a placebo or not.) If participants lose efficacy due to withdrawal of their medication, they will move into phase three of the program and will be placed back on the medication.

According to Frye, the ConnectMe trial is unique because all participants will have the opportunity to receive the medication before moving into the “placebo controlled withdrawal” phase. Also, parents and participants get to determine up front if they have had a positive reaction to the medication before moving into phase two.

Frye explains that research suggests the brain of children with autism is hyperexcitable. Normal brains have a balance of being both excited and inhibited; whereas, the autistic brain is too excited or not inhibited enough. “This medication is designed to decrease excitability in the brain so that the brain can function better,” Frye says about memantine, the drug used in the study.

Frye also says he is hopeful about the study’s findings based on the results of similar, preliminary studies. In addition to other trials with autistic children, the drug has been used to treat elderly patients with Alzheimer’s disease and has proven to be “safe and effective,” Frye says. “Theoretically it should help children with autism,” he says. “I’m hoping that either way, we’ll have some good information. That’s what’s really important for us, to get that objective information.”

Children ages 6-12 may be eligible to participate in the study if they have autism, Asperger’s Disorder or Pervasive Developmental Disorder Not Otherwise Specified (PDD-NOS); they are verbally fluent or use some phrase speech; they were not born prematurely; they do not have a major psychiatric disorder; they do not have a seizure disorder; and do not have an IQ in the mentally retarded or mildly retarded ranges.

Frye says the most important thing for parents of children with autism spectrum disorders is to make sure the child is in therapy and appropriate classroom settings. “Medication is no substitute for therapy,” he says. “If they are not progressing socially, then it’s important to determine if there are other things that can be done to make them progress more, and this is one of those,” he says about the ConnectMe trial.

ConnectMe participants will be on the medication for a significant length of time, until the study ends in 2014. Frye says that if the results are positive and clear, Forest Laboratories, Inc., the company hosting the study, may apply to the FDA to market the drug, and the drug could be on the market sometime shortly thereafter.

Frye also points out that he and his team at ACH run a number of other treatment trials, and that children who aren’t eligible for the ConnectMe trial could be eligible for another ACH study. The hospital is currently building a center for autism translational research, where they can investigate multiple ways to improve the lives of those with autism as well as host treatment trials. “We definitely want to bring in all the latest cutting edge treatments that are available,” Frye said.

For more information on the ConnectMe research program or to find out how to apply, visit ConnectMeProgram.com.