Autism Drug Trial Underway

November 8, 2016

By: Austin Ramsey
Source: DisabilityScoop

NEWBURGH, Ind. — Researchers are hopeful that a new clinical drug trial based on research linking diet and brain development could yield a treatment that addresses core symptoms of autism.

Dr. Michelle Galen of Deaconess Clinic is one of more than two dozen doctors nationwide participating in the Phase III U.S. Food and Drug Administration fast-track trial. Curemark, a biotech company based in New York, has developed a potential treatment for autism based on its founder’s discovery that many children on the spectrum are deficient in an important enzyme used to digest protein into its building blocks.

Galen explained that many children with autism exhibit impaired protein digestion and, as a result, may self-restrict their diets. The inability to digest protein affects the availability of essential amino acids in the body. Those chemicals play a critical role in the expression of several genes important to neurological function and serve as precursors to key neurotransmitters important for brain development.

Curemark’s trial drug, CM-AT, is a proprietary enzyme in powder form. It’s sprinkled on food and formulated to activate in the small intestine, where it may enhance protein digestion, thus increasing the availability of amino acids.

“This investigation takes advantage of the relationship between your brain and your gut,” Galen said. “There aren’t any drugs that treat the core symptoms of autism, except antipsychotic medications. This treatment may get at what causes autism.”

The U.S. Centers for Disease Control and Prevention estimates that 1 in 68 children in the country have autism spectrum disorder, an umbrella term for a group of developmental disorders that can involve varying degrees of language and social impairments, and repetitive behaviors.

The CM-AT trial, Galen said, is a 14-week open-label extension study for children ages 3 to 8. Kids must first participate in an initial screening to ensure that they fall within the spectrum and so that doctors can note their outward symptoms. Galen said she invites parents to bring their children in for screening, even if they have not yet received an autism diagnosis. There are no restrictions about where on the spectrum a child must fall. High-functioning children with autism are participating, Galen added.

The National Institutes of Health estimates that almost 180 children are currently in the drug trial nationwide. So far, Galen said about five children are participating in the trial in Newburgh.

Galen said the drug is sprinkled over a child’s food so that it has a minimal impact on the child’s schedule. Interrupting the daily routine of a child with autism can be harmful to their health and cause emotional or psychological duress, she said.

Researchers, Galen said, are actually looking for symptomatic improvements with this trial, a development that she called “groundbreaking” in the field of autism research and treatment.

“We are looking for changes in behavior,” she said.