Dr. Olivier Ameisen is a prominent French cardiologist who believes that the muscle-relaxant drug baclofen relieves the cravings of alcoholism. This is indeed an interesting, and as yet unsettled, scientific medical question. Dr. Ameisen has decided to take his personal scientific opinion directly to the public in his book – Le Dernier Verre (The Last Glass). The result has been a surge of interest among alcoholics for this new “miracle cure” for their affliction.

Increasingly the medical community is caught between two opposing imperatives. There is the desire to make medical information freely available and the process of medical research transparent. On the other hand, the public is best served when new ideas in medicine are put through the mill of science before they become part of medical practice. As we enter headlong into the information age these two imperatives are increasingly at odds.

Problems arise when a new treatment, syndrome, intervention, or concept in medicine is promoted to the public prior to undergoing a reasonable degree of scientific vetting. What is the point, after all, of spending tremendous resources on medical research if proponents are going to bypass the process altogether to market their modalities and promote their ideas directly to the public?

The downside of evading the process of science (even though it can be frustratingly slow) manifests in many ways. Patients who hear about a new possible treatment are likely to demand it from their doctors. Or they may seek the treatment from a paraprofessional or even non-professional (take bee-venom therapy, for example).  Charlatans who set themselves up as gurus in their own clinic exploit this phenomenon to market their fraudulent treatments and products. Research may be hampered as patients are unwilling to enter a study and perhaps be assigned to the placebo arm.

Direct-to-consumer promotion of new untested or inadequately tested ideas also undermines the public confidence in science-based-medicine. Most new ideas turn out to be false, and this will inevitably lead to disillusionment. Appropriate skepticism toward the new claim often seems to the public (and is sometimes deliberately made to seem so) as obstructionism, protectionism, and closed-mindedness.

And of course bypassing the scientific community puts the public at risk for being subjected to unsafe or ineffective treatments.

Let’s take a look at the baclofen controversy. Dr. Ameisen is convinced of the efficacy of baclofen because of his own personal experience. He is a recovering alcoholic who treated himself with baclofen and feels it significantly decreased his desire for alcohol. So his conviction is based mostly on personal anecdotal experience. This is a particularly weak form of evidence, although it is a human foible that it is also very compelling, even though it shouldn’t be.

There are currently no published trials of baclofen for alcohol dependence (although one study is in progress that I could find). Dr. Ameisen decided to try baclofen because of a few published studies of its use in cocaine dependence.  There is likely a common mechanism underlying some forms of addiction, including alcohol and cocaine, so this was not entirely unreasonable.

Therefore at present baclofen for alcohol dependence is a plausible but unstudied treatment. This is not sufficient to recommend it as a treatment, it is only sufficient to warrant performing clinical trials. Most plausible treatments in fact do not work, and that is why research should come before promotion to the public.

One rationale for using unproven but plausible treatments is compassionate care – for serious or terminal incurable diseases. There are already standard therapies for alcoholism, however. In fact the treatment community is concerned about the hype surrounding baclofen partly because it undermines a more proper focus on multi-modality treatment in favor of a silver bullet approach. (Admittedly, this is a separate issue.) In any case the situation does not appear desperate enough to bypass research for compassionate use. And further, compassionate use should be done, whenever possible, in the context of a clinical study (even if open label) in order to gather some useful data.

The baclofen story is, unfortunately, not an isolated case. The mental health field is rife with practitioners who come up with some new treatment philosophy and then bypass the research path in order to write a popular book. This leads in some cases to widespread adoption, dedicated institutions, and popular belief in a treatment that is unlikely to work. This is exactly what happened with the recovered memory (now called the false memory) syndrome. The Courage to Heal by Ellen Bass sparked an industry of recovered memory therapy that still lingers decades later, even after the research showed it to be harmfully misguided.

The public is best served when the time is taken for new ideas in medicine to be tested and for controversies to be reasonably settled before the standard of care is altered and new treatments are accepted.  But there is a huge temptation to popularize one’s work and ideas (and the internet makes this an almost trivial exercise) without first going through the tedium and often harsh confrontation involved in the scientific process.

I will follow with interest the story of baclofen for alcohol dependence, and I hope it turns out to be an effective treatment. I also hope it does not take on a life of its own, becoming popularized and politicized in a way that is independent of the scientific evidence and ultimately distorts the process of science. We will see.

Posted by Steven Novella

Founder and currently Executive Editor of Science-Based Medicine Steven Novella, MD is an academic clinical neurologist at the Yale University School of Medicine. He is also the president and co-founder of the New England Skeptical Society, the host and producer of the popular weekly science podcast, The Skeptics’ Guide to the Universe, and the author of the NeuroLogicaBlog, a daily blog that covers news and issues in neuroscience, but also general science, scientific skepticism, philosophy of science, critical thinking, and the intersection of science with the media and society. Dr. Novella also contributes every Sunday to The Rogues Gallery, the official blog of the SGU.