- Last Updated on 29 December 2016
Beyond the handful of "unproven" therapies discussed on this site, this post is meant to be a general discussion of the countless "alternative" or "natural" treatments often advertised on the internet.
As a general rule, the following are clues that a medication or treatment may be dangerous, a scam, or otherwise ill-advised:
- Very expensive
- Not covered by insurance
- Not offered in the United States
- Your pediatric neurologist has never heard of it
These guidelines are by no means perfect and some effective therapies meet the above criteria, and several ineffective or harmful therapies do not meet any of the above criteria.
The internet is overflowing with reports of "cures" supported with testimonials from practitioners and parents of affected children. Although emotionally laden, and in some cases highly compelling, caregivers should use caution in interpreting such advertisements and carefully consider the risks and benefits before embarking on these therapies.
Why would so many people speak highly of a treatment if it doesn't work?
Many children with infantile spasms (as well as other forms of epilepsy and neurologic disease) will exhibit improvement, or even resolution of infantile spasms, no matter what therapy is administered—effective or not. In fact, almost all children will have resolution of infantile spasms by the age of 3 or 4, though many will suffer other forms of epilepsy. Furthermore, the severity of infantile spasms often fluctuates over time. If a given therapy is accompanied by success, it means that either the therapy works, there was coincidental improvement, or simply that there was a biased belief that therapy has worked (discussed below). In general, online contributors are much more likely to offer commentary about unconventional treatments that seem to work, and less likely to blog about success with typical therapy and failure of unconventional treatments. We call this blog bias, a phenomenon that permeates nearly all online discussions of medical issues. There is also a natural tendency to assign blame to treatments or events that immediately precede the beginning of infantile spasms, and conversely, give praise to events or treatments that immediately precede the resolution or improvement of infantile spasms. This is generally referred to as confusion between causation and correlation. Recommendations of therapies need to be backed by compelling scientific data, or "proof".
What constitutes proof that a treatment is effective?
In general, the standard of "proof" among those in the medical community is a successful clinical trial with:
- placebo controls
What are placebo controls and what is the placebo effect?
In rigorous scientific studies, some patients receive the experimental therapy and other patients receive a placebo therapy. Experimental therapy is usually standard-of-care therapy combined with an experimental drug/treatment, and placebo therapy is usually standard-of-care therapy combined with a pill (or liquid, or other treatment) designed to look and feel exactly like the experimental therapy. The most common example of a placebo is a pill that is the same size, shape, color, and texture as a pill containing an experimental drug, but filled with sugar or an inert substance instead of the experimental drug. Placebos are important to assess (1) the impact of the belief that a treatment will work regardless of how effective it really is, and (2) the impact of coincidental improvement. It may surprise many people to know that patients receiving placebo therapies routinely exhibit improvement. This is because simply believing that a therapy may be of benefit affects the way we perceive results (e.g. seizure frequency, side-effects). This is true of patients, parents, and healthcare practitioners.
What is randomization?
Randomization refers to a procedure within clinical trials in which patients are randomly assigned to receive experimental therapy or placebo therapy. By randomly assigning patients to receive drug or placebo, scientists can help reduce the chance that there are meaningful differences between the experimental and placebo groups, and thus less chance that the results of a clinical trial are misleading or incorrect.
What is blinding (masking)?
Blinding refers to a specific effort to eliminate the bias of patients, parents, and scientists conducting clinical trials. As mentioned above, if a parent believes their child is receiving a potentially helpful drug, they are more likely to report that it is working. The same is true of physicians and scientists conducting studies. If scientists believe a patient is receiving a helpful drug, they are more likely to record improvement in their assessments. For this reason, rigorous clinical trials go to great lengths to ensure that patients, parents, and scientists who evaluate the drug do not know which patients are receiving the experimental drug, and which patients are receiving the placebo. One might ask who knows drug/placebo assignment if the patients, parents, and scientists are all blinded. Typically, group assignment is known only by a study coordinator or study pharmacist who does not interact with the patient, and who does not determine response to therapy; study assignment is revealed after determinations of effectiveness are finalized.