Treatment Guide

New Study Will Test Theory That Enzyme Contributes to Autism

The debate rages on both the causes and “cures” for autism. There is the mercury-vaccination contingent, the gluten-free, casein-free diet supporters, those that believe genetics play a role, and the list goes on. There may very well be multiple etiologies for this developmental disorder, and research continues throughout the world to determine, definitively and finally, what that is.

One of the newest clinical trials is just beginning across the country, at fifteen institutions, including the University of California at San Francisco. Funded by Curemark, a New York-based drug company, this Phase III clinical trial for CM-AT autism treatment, has been granted fast track status by the FDA. Researchers will be testing whether certain children with autism can benefit from regular doses of an enzyme to help them digest proteins, which may in turn improve their brain function and ease some symptoms of autism.

The trial is not without its naysayers. There is very little research to support the premise that a missing enzyme is a factor in the cause of autism. There are those studies which have shown that a small subset of children diagnosed with autism have enzyme deficiencies, but there is debate as to whether it is a causation or a symptom of the disorder. But with a new case of autism being diagnosed every 20 minutes in America, it is imperative that research such as this be conducted. Sometimes, with a bit of persistence and creative thinking, you do find that needle in a haystack.

The trial will involve 170 children, ages three to eight, over a 90-day period. Half of the participants will be a control group and receive a placebo, while the other half will receive three enzyme treatments per day (a tasteless powder sprinkled over food). At the end of the 90 days, parents can remove their children entirely from the project or choose to continue for one year on the enzyme regimen.

“The treatment is enormously simple, but finding it out wasn’t simple at all,” said Joan Fallon, chief executive of Curemark. “Is it theoretical? Yes. But we hope the trials will give some benefit to a subgroup of children. And we hope our trials will make other researchers look at the physiology of the disorder.”

Curemark has identified a series of biomarkers that determine which children with autism and Pervasive Developmental Disorder (PDD) may have digestive deficiencies underlying or as a major component of their disease.  Research by Dr. Fallon showed enzyme deficiencies in children with autism, resulting in an inability to digest protein.  The inability to digest protein affects the production of amino acids, the building blocks of chemicals essential for brain function.

Autism is a neurological and biological disorder which typically affects children ages 18 months to 5 years of age. It knows no racial, ethnic or social boundaries. A child’s chances of having autism are not determined by their family’s lifestyle, education or income.

Author Information: Susan Brady
Susan Brady, the editor of The World Is a Kitchen, is a woman with a passion for food. When not living the life of a typical suburban soccer mom, she spends long hours in the kitchen testing recipes from around the world, and travels to faraway places to learn new cuisines. You can reach Susan by e-mail.

About Susan Brady


  1. I wonder why no one is looking into the purine metabolic disorder angle? My father had gout, and when I did a google search to see if there was any connection, I came across the Purine Research website, which I admit is awfully poor and lacking in good information. I also found somewhat of an explanation on the Great Plains Laboratory site. I’m very interesting in this because as I got older, I became very sensitive to any legume. But we have soy allergies and peanut allergies already. Guar gum is derived from a legume also, and this might explain why some of us celiacs are sensitive to it. I have gotten to the point where I have to treat legumes in the same manner as I treat gluten. I absolutely do wonder if legumes are also a trigger in autoimmune disease, and the few disorders that are recognized are just the tip of the iceberg, along the same lines as celiac disease was considered rare in the U.S. in the pre – Fasano days?

  2. The Purine Research website has not been updated significantly in years, but I found this information on PUMPA, a British site:

    Why purine metabolic patients?

    When a defective gene causes gaps to appear in this recycling process, these chemicals are not metabolised properly, and adults or children can suffer from any one of twenty-eight hereditary disorders, possibly some more as yet unknown. Symptoms can include gout, anaemia, autism, epilepsy, delayed development, deafness, compulsive self-biting, kidney failure or stones, or loss of immunity (similar to AIDS).


    I don’t mean to hijack your column. But that problem with immunity…I wonder if that makes it easy for gluten intolerance to piggyback on to a purine metabolic disorder. The article goes on to talk about babies so immune deficient that they must be hospitalized in order to have a bone marrow transplant.

    And again, with all the soy, soybean oil and gum thickeners in our food these days, people who have the genetic predisposition are getting much greater exposure than they should. I suggest this might explain why some celiacs have a greater problem adjusting. Legumes do cause terrible inflammation and diarrhea when I get an accidental exposure. Thank you for your patience. Rebecca

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