Could a cure for one of the most destructive addictions have been lying dormant on the shelves of the FDA?
In searching for a potential cure or treatment for alcoholism, clinical researchers have thrown almost every drug they can think of at the condition. Precious few of them stick, but one of them appears to work, and it’s a U.S. Food and Drug Administration (FDA) approved muscle relaxant called baclofen.
Baclofen and its link to alcoholism and other addictions has been hotly debated since 2002. Media coverage has been fickle and despite a number of vocal experts speaking out in favor of the drug, it remains to be definitively proven to eradicate cravings.
One theory links alcoholism to an acid called GHB, which is a relaxant and naturally produced by the body. When an individual is deficient in GHB, other relaxants such as alcohol pick up the slack, meaning that people can become gradually dependent. Baclofen, also a relaxant, acts on the same brain receptors as GHB, making you feel relaxed without having to reach for the bottle. That’s the theory anyway.
This sets the drug apart from other drugs on the market that aim to treat alcohol abuse. Antabuse, often prescribed to alcoholics doesn’t stop the craving—just punishes it. If a patient drinks while taking antabuse, a negative reaction such as vomiting is induced. Scientific and clinical research with an aim to find a pill to treat alcoholism is rarer than narcotic dependence.
Now though, the uncertainty surrounding baclofen’s credibility might be about to change one way or the other. The French government has granted funding to the tune of $920,000 to back a placebo based clinical trial by Dr. Philippe Jaury of the Paris Descartes University, which is currently underway and nearing completion.
For many people, alcoholism sufferers and experts alike, knowing how it works would be nice but for the time being they are content to be excited, they say, simply knowing that it does work.
The French government is now convinced enough to sanction the prescription of the drug for alcoholics. The ANSM, the French equivalent to the FDA, recently gave Baclofen the green light, but American alcoholics still have find a physician willing to prescribe the drug off label.
“I knew within half an hour that it was going to work,” says Beth Fields—a former alcoholic and a self-proclaimed baclofen believer from Oklahoma—as she remembers her cravings slipping away after the first pill.
But the science doesn’t necessarily agree with Field’s anecdote.
A 2007 U.S. based trial, which looked at baclofen’s potential to reduce drinking, found no significant evidence that the drug actually helps participants anymore than a placebo does. Despite that, Dr. James Garbutt who led the study says that he still believes baclofen could be a credible alcoholism treatment. He speculates that the 30 mg per day dose that he used may have been too low.
There have been a couple of previous clinical trials in Italy, which contradict Garbutt’s findings to come out in favor of a positive link between baclofen and abstinence. However, these studies had a small number of participants (82) and a running time of just 12 weeks.
The French study will follow 320 participants for 12 months, and as such it carries the promise and potential of more meaningful and conclusive evidence.
If the French trial turns out to bolster baclofen’s abstinence credentials, the results from the trial could theoretically be submitted to the FDA to request a label change for baclofen to be formally acknowledged as a treatment for alcoholism. “The FDA pays attention to other regulatory boards—they don’t make decisions in a vacuum,” says Dr. Eliot Gardner, head of Neuropsychopharmacology at the National Institutes of Health (NIH). A spokesperson from the FDA also confirmed that foreign studies may be submitted as evidence.
A sponsor is required to table such a request to the FDA, which would usually be a pharmaceutical company, but in theory it could also be a private individual. Dr. Willenbring, a Minnesota physician and founder of a specialist addiction clinic called ALLTYR, says that it is unlikely that a drug company will ever sponsor a label change for baclofen. “Since the drug is a generic there’s no way to make the investment back,” he says.
It also seems unlikely that a private citizen would act as a sponsor. The only people who are really interested in doing so are the researchers and doctors prescribing baclofen, i.e. those who want to see baclofen succeed as an alcoholism treatment. But, as Gardner says, “as a medical research scientist, I have no clue about how to raise money for such an endeavor.”
The cost of tabling a serious application that stands a chance of succeeding is estimated to be in the millions of dollars. This is because a typical application for the FDA relies on the evidence of several clinical trials, which don’t come cheap. Although an application could technically be submitted based solely on the evidence of the French study, the chances of success are greatly increased with more studies.
When asked whether they have considered raising the money to act as a sponsor, all researchers responded similarly to Gardner. Only the Franco-American Dr. Olivier Ameisen confessed to having entertained the thought, “I have considered fund raising,” he says, “but I am not sure how to do it because I am not a business man.”
Some experts remain cautious. Dr. Benjamin Rolland of the Université Lille Nord in France, where off label baclofen prescription is much more common, calls the situation a “French craze.”
Rolland believes baclofen might have potential in the fight against alcohol addiction; he’s just baffled that the drug hasn’t so far followed the normal approval protocol. Although he does actually prescribe baclofen to some of his patients, by and large he thinks that the medical community should wait for proof from the placebo trial before doctors casually prescribe baclofen. “We work on evidence, not belief and we must remember that,” he says.
“The whole story revolves around one man and we must remember that one man is only one man,” says Rolland. He’s referring to Ameisen, who became the first human to take baclofen to treat his own alcoholism when he wrote himself a prescription in the spring of 2002 and has subsequently written books about his experience.
Rolland is right, the story does revolve around Ameisen, you can’t talk to anyone about baclofen and alcoholism without his name popping up at some point—and everyone has their opinions about him.
Some are grateful and admire his courage; “He absolutely deserves a Nobel Prize for his work, he tested the drug on himself!” says Dr. Fred Levin, a Chicago based physician who prescribes baclofen.
Others are less certain of the value that Ameisen can bring to the baclofen debate. “Sometimes he speaks like a physician and other times like a patient,” says Rolland, “We don’t know if he’s a lobbyist or a scientist.”
Ameisen says that his past experiences as an alcoholic and a baclofen pioneer make him all the more qualified to speak on the matter because “it is very degrading for a patient to hear 'I understand what you are going through' from a non-alcoholic,” he says.
WHERE IT CAME FROM
In reality, the story began before Ameisen put pen to prescription pad 12 years ago; it began with a researcher called Dr. Dave Roberts in a Carleton University lab during the late 90s. Roberts says it was a student of his who suggested they look into the possibility that baclofen might help fight addiction, but he says they “had no theoretical axe to grind, we were just throwing all the drugs in the cabinet at the problem to see what might work.”
They realized they had hit something with their stab in the dark when they started to test the effects of baclofen in cocaine addicted mice. The mice on baclofen no longer had the drive to seek out a hit of cocaine compared to the mice not given a dose of baclofen.
It was after reading Roberts’ research that Ameisen decided in his desperation to give baclofen a try. “I tried every medication on the market and found that they didn’t work at all. I chose myself as a guinea pig because I knew I was going to die,” he says.
The French trial is expected to come to a conclusion in early June of this year, but it could be significantly longer before its results are known. In the mean time, a number of doctors in the U.S. are prescribing baclofen to alcoholism sufferers. Levin says that each day his phone rings with new people asking him to write a baclofen prescription and he accepts almost all of them. “This isn’t something that I hesitate to prescribe, you have to give [patients] the opportunity,” he says. Levin adds that his success rate with baclofen is extremely high, “if you’d had the experience that I have, you wouldn’t refuse baclofen.”
It takes Levin about two to three months to find the right dose for each patient, gradually increasing the prescription until the patient no longer has a desire to drink.
Willenbring doesn’t see the point in requesting a label change. “More drugs in the U.S. are prescribed ‘off-label’ than for FDA indications … so FDA indication is largely meaningless in terms of what doctors prescribe,” he says.
He’d be right if there were a large number of doctors in the U.S. prescribing off-label baclofen. But there isn’t, they’re just a handful in reality, and a label change would almost certainly empower patients to inquire about baclofen therapy. Online message boards are a flurry with recovering alcoholics offering each other tips on how to get hold of baclofen and how to get your doctor to consider writing a prescription.
Fields says that it isn’t easy to convince a physician in the U.S. to prescribe baclofen for alcoholism and it took her some time to find one who was willing to help her. This difficulty has driven alcoholism suffers, through their desperation, to buy baclofen online rather than seek a legitimate prescription from a physician.
Fields is a somewhat active member of one of the online message boards, which lists the contact details of physicians who are known to prescribe the drug as well as websites where you can illegally buy baclofen without a prescription. She says that she begun her baclofen treatment with pills she bought online and self medicated before finding a doctor that wrote her an off label-prescription. “I’ve never heard of anyone complaining that the baclofen they ordered online wasn’t good,” she says. But clinicians and experts universally and strongly advise against buying online. Gardner says that you never really know what you’re getting: “They could be sugar pills or worse, something sinister.”
Where’s all this going? What function will the clinical trials present if there isn’t going to be a label change by the FDA? They would tell us a little more about the safety of the drug, and more comprehensively prove or disprove that baclofen works as an addiction treatment.
“My end goal is for any doctor to treat alcoholism with baclofen in the same way they treat other diseases with drugs,” says Ameisen. Other experts are less optimistic and think there remains a huge distance to travel to reach Ameisen’s end goal.
“Where we are right now is like where we were 60 years ago with depression,” says Willenbring—comparing depression patients, who were institutionalized, with alcoholism patients who are today sent to rehab.
“What’s going to be the Prozac moment for alcohol dependence?” Willenbring asks, unable to answer. But, he predicts that the way we treat alcoholism is going to look radically different within a decade, and he thinks drugs like baclofen are needed to bring about that change. “We need to change the addiction treatment system to one of science rather than ideology,” he says, adding that the “time is ripe” for such a change.
by Benjamin Plackett, a New York based science and health journalist. His work has appeared in Wired, CNN and Business Insider.
March 24, 2014
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Could the Cure for Alcoholism Be Lying Dormant on FDA Shelves?