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Two clinical trials for autism

Posted Mar 18 2010 2:47pm

I am, and will continue to be, highly critical of therapies used to treat autistics (typically children) which are potentially dangerous and which are hyped beyond any actual proof. Since many often misinterpret this to say that I am against any treatment of autistics, I thought I would discuss two clinical trials caught my eye this week, one completed and one starting. These are studies which I am happy to see made.

What separates these from other studies and, worse, the
1) They can give answers to the anecdotal reports that these work
2) The drugs are known, so the safety concerns (including adverse reactions) can be weighed by parents before hand. The side effects are reversible and, for the most part, the side effects are minor.
3) They are being studied under controlled conditions, so results have the potential to give useful information.

Digestive Enzyme Supplementation for Autism Spectrum
Disorders: A Double-Blind Randomized Controlled Trial

Sujeeva A. Munasinghe, Carolyn Oliff, Judith Finn, John A. Wray

Just came out in the Journal of Autism and Developmental Disabilities.

The abstract from this paper states:

Abstract To examine the effects of a digestive enzyme supplement in improving expressive language, behaviour and other symptoms in children with Autism Spectrum Disorder. Randomized, double-blind placebo-controlled trial using crossover design over 6 months for 43 children, aged 3–8 years. Outcome measurement tools included monthly Global Behaviour Rating Scales, Additional Rating Scales of other symptoms by parents and therapists, and monthly completion of the Rescorla Language Development Survey. Compared with placebo, treatment with enzyme was not associated with clinically significant improvement in behaviour, food variety, gastrointestinal symptoms, sleep quality, engagement with therapist, or the Language Development Survey Vocabulary or Sentence Complexity Scores. A small statistically significant improvement on enzyme therapy was seen for the food variety scores. No clinically significant effect improvement of autism symptoms with enzyme use was shown with this trial, however, possible effects on improvement in food variety warrants further detailed investigation.

The basis for using digestive enzymes is the so-called “leaky gut” theory, or “opiod excess” theory, whereby the digestive tract is permeable (leaky gut) and chemicals leak into the bloodstream which cause autistic behavior (opiod excess). It is not a theory which I give much weight, but it is commonly accepted and the use of digestive enzymes is not uncommon in the alternative medical autism community.

The authors found, not surprisingly in my opinion, that digestive enzymes were not effective. The sample size was relatively small (43), but the study was a “crossover” type, so that all the children spent some of the study time on the enzyme and some time on placebo.

The second study was announced this week in a press release
Autism Research Study Announced By Children’s Health Council

Dr. Glen Elliott of Children’s Health Council leads clinical research for local patients

The press release is quoted below:

Autism is an exceptionally complex illness. Autism is a developmental disorder with impairments in social interaction and communication, in addition to restricted, repetitive behaviors. Once considered quite rare, 1 in every 150 children in the United States is now diagnosed with the illness, making it a common developmental disability.

Diagnosis of Autism typically occurs at about 3 years of age. The treatment of the condition is complex, as there is no single known cause or cure. Early childhood is the period during which the symptoms of Autism are clearly observable and children are experiencing rapid developmental changes. Researchers continue to look for more effective behavioral, educational and medical treatments to improve the lives of children with Autism. Therefore, a key to overcoming some of the challenges associated with the condition is early diagnosis and intervention.

A clinical research study is now underway in the Bay Area for children between the ages of 3 and 6 with Autistic Disorder. The goal of the study is to evaluate the safety and effectiveness of an investigational medication for children with this condition.

Dr. Glen Elliott of Children’s Health Council (CHC) is conducting this clinical research study. CHC believes all children deserve the opportunity to reach their full emotional, educational and developmental potential. CHC seeks participants for this clinical study, based on the following: the children must be at least 3 years old and less than 7 years old; candidates for the study must meet the criteria for Autistic Disorder; and, she or he must have an IQ or developmental quotient (DQ) of at least 50.

Parents must give informed consent for their child to participate in this clinical research study, and must also be willing and able to comply with all study requirements. All clinical study-related care will be provided at no cost, including physical exams, psychological testing, and study medication. Compensation may be available to eligible parents or guardians. Health insurance is not required to participate.

If your child or a child you know has Autism, additional information about this clinical research study and how to participate is available at (800) 314-2597 and at .

If one follows the links, one finds that the study is for Sapropterin. The trial is on .

The Children’s Health Council is an established, well respected, private clinic and school based near Stanford University in California. The lead researcher is Dr. Glen Elliott , formerly of the University of California San Francisco. Sapropterin (Kuvan) is a drug used for the treatment of some forms of Phenylketonuria (PKU), a rare but severe developmental disorder. Most children in developed countries are tested for PKU at birth. A discussion of the use of Sapropterin for PKU is here .

As an aside, PKU results from a genetic disorder. I often hear autism parents say things like, “Doctors tell us that autism is genetic and, thus, untreatable”. Genetic doesn’t mean untreatable, and here is a classic example.

Back to the clinical trial. I don’t understand the proposed mechanism by which Sapropterin is supposed to work. What I do know is that there are at least two studies on the use of Sapropterin in treating autistic children (I will try to link to them soon). The studies were small and not conclusive, but the thought is that Sapropterin helps communication. There are at least a couple of doctors already using Sapropterin in the U.S., and I expect we would hear more about this if it weren’t for the fact that the drug is incredibly expensive. As in, about $50,000 per year. If I understand it correctly, Sapropterin was invented as much as 20 years ago, but has only recently been applied to PKU and that under an “orphan drug” patent. Since clinical trials have already been held on Sapropterin for PKU, one can find the side effects . Additional side effects include seizures in seizure prone patients.

Any drug, any therapy carries with it the potential for an adverse reaction. This is especially true in a clinical trial where the effects of the drug on the population studied are untested. The need for caution is compounded when the study subjects are children, and doubly compounded when the subjects are disabled children.

I do not take the idea of clinical trials on autistic children lightly. However, I will again list the reasons why these particular studies were interesting to me:

1) The drugs are relatively safe
2) the risks, especially for Sapropterin, are documented
3) the studies are controlled, so the contributions of the children involved are not wasted as with some of the flimsy studies we see periodically
4) the studies can answer questions about drugs already in use.

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