Autism Biomedical Information Network



What about "It can't hurt to try" a new treatment?


Overview

"What is wrong with an empiric trial of a new treatment?"
Insistence on evidence
C.R.A.P. detector
A theory of disease and its treatment has to "make sense"
"Lorenzo's Oil syndrome"
What do families really need?

"What is wrong with an empiric trial of a new treatment for autism" if it "can't hurt?"

This is a question that I've heard recently in relation to secretin and this is my reply. I believe that a parent should insist on a treatment that is both safe and effective. But, what if a treatment is believed to be safe but it is not known if it is effective? What's wrong with "well, it can't hurt to try it?" Unfortunately this attitude leaves one open to being exploited by the various snake oil peddlers out there and wasting several hundreds or thousands of dollars. I would not subject my own child to a treatment that is not evidence-based or, in the absence of evidence, makes no biological or physiological sense; i.e., does not have a ring of plausibility within the range of phenomena that are likely to occur in biological systems. After all, not everything is possible. What may at first seem possible is constrained by the limits of plausibility, especially in the instance of as complex a phenomenon as autism. In view of this complexity (see commentary, What is autism?) I cannot begin to imagine that something as simplistic as a single injection of secretin; or vitamins; or hyperbaric oxygen; etc., can re-arrange "the wiring" of the brain (if the wiring of critical modules subserving social cognition was even there in the first place). In fact, in the instance of a single injection of secretin one would have to invoke an improbable sustained effect on cell biology (translational processing and protein synthesis; or neurotransmitter synthesis) persisting well beyond the usual time course of action of a hormone.

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Insistence on evidence

This perspective is based on plausibility-constrained thinking and insistence on evidence. Although there is certainly room for empiricism in medical practice, it needs to be balanced by a healthy skepticism until confirmatory studies are done. The road to the pre-eminent state of U.S. medicine is littered with empiric, "aconceptual" therapies that did not provide benefit and fell by the wayside. Only a few have serendipitously held up over time. A good example is corticosteroid treatment (such as prednisone) for idiopathic nephrotic syndrome. Such therapy was initially tried for all the wrong reasons, given the imperfect understanding of this disease at that time (some 40 odd years ago). To this day we still do not know "how it works" in the treatment of nephrotic syndrome. But this is an exception.

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C.R.A.P. detector

When a proposed empiric treatment doesn't "make sense" it sets off my C.R.A.P detector. This is a term I heard used many years ago by Dr. Harriet Dustan (once a prominent cardiology researcher at the Cleveland Clinic Foundation). More recently I have seen it translated as "Circular Reasoning or Anti-Intellectual Pomposity" by GR Norman and DL Streiner in "PDQ Statistics" (1986, published by BC Decker Inc., Toronto, p. 13).

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A theory of disease has to "make sense"

Many of us, who later went into medicine, were once kids who tried to figure out "how things work." Even then there was a little bit of the "scientist" in us. Later, during our study of medicine, we learned about mechanisms of disease (and health). Some of us are, in part, theoretical biologists. Good theory is based on much antecedent work by others and an understanding of how biological systems "work." So, for me, a theory of disease and its treatment has to "make sense." Since, short of a lobotomy, I can't do much about my own rational nature, using treatment "because it can't hurt" goes against my grain. It also opens up the possibilty of exploitation. Families are desperate, vulnerable, hoping against hope. The autism culture is rife with treatments that are expensive and have lured vulnerable families to distant places for hyperbaric oxygen treatment, "detoxication", chelation, swimming with dolphins, etc. The promise of a quick cure belies the complexity of the mind-brain entity and of the subtle neuropathology of autism.

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"Lorenzo's Oil syndrome"

It can be argued that even if a treatment doesn't "make sense" and is not likely to work, that it helps parents to be "kept busy doing something." That it gives them hope. That they should "press all the buttons." However, this only serves to perpetuate denial and keep parents from coming to terms with the reality that there is no quick cure for autism. Some parents react by distrusting medical science. Hence the "Lorenzo's oil syndrome" when parents, so distrustful of professionals, seek out alternative therapies (recall the film about a degenerative brain disorder in young children starring Nick Nolte and Susan Sarandon). These therapies may even have a placebo effect in the sense of enforcing interaction between parent and child (such as "floor time" as advocated by Stanley Greenspan). However, although any interaction is better than none at all, a family system can become unhinged by the daily stress. Other children in the family don't get the attention they need.

At the present state of knowledge family resources should be focused on intensive behavior treatment for young children rather than wasted on unproven treatments.

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What do families really need?

What families really need are supports, respite care, and a case manager to provide continuity of care and who is "there" for the crises that frequently arise at home. And who is "there" to constantly prod the schools to deliver one-on-one truly individualized educational services and have a way of monitoring whether or not educational goals are being met. Until the fundamental neurobiological basis of autism is understood, the only things we can offer are intensive educational and behavioral interventions and certain medications for a "blunt instrument" approach to managing challenging behaviors. At the same time we have to be optimistic with families that some day the complexity of autism will be penetrated by advances in neuroscience.

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Commentary by Ronald J. Kallen, M.D., ©1999
This page last updated on 6/3/2000