Alcohol use disorders (AUDs) are highly prevalent, cause serious personal suffering, and constitute a high burden to society as a whole. Unlike most other mental disorders, AUDs are still seen by many as some kind of moral weakness (Schomerus et al., 2011), and this includes many healthcare professionals (van Boekel et al., 2013). However, animal studies and human neuroimaging and clinical research have clearly shown that AUDs are brain disorders due to a combination of genetic vulnerability and adverse environmental circumstances with neurobiological abnormalities in the reward and motivational system, the cognitive control system and the emotional and stress regulatory system (Koob and Volkow, 2016).
The good news is that currently three compounds are approved for the treatment of alcohol dependence in North America and Europe (disulfiram, acamprosate and naltrexone) and one other compound is registered in Europe only (nalmefene). In addition, sodium oxybate is approved for the treatment of alcohol dependence in Austria and Italy. Finally, a broad range of other compounds with different mechanisms of action are recommended by professional organizations for the (off-label) treatment of patients with an AUD, including topiramate, varenicline, gabapentin, ondansetron and baclofen (Soyka et al., 2017).
The bad news is that <20% of all people with an AUD seek treatment (Kohn et al., 2004), that most primary care physicians fail to recognize and treat these patients (Rehm et al., 2016) and that even psychiatric and addiction specialists often fail to offer pharmacological treatment to their AUD patients (Scherbaum et al., 2019). Thus, although AUDs are recognized professionally as a brain disorder and although effective medical treatments are available, very few patients with an AUD are adequately treated.
Baclofen has been a game changer in this unfortunate situation. After publication of the book of Ameisen (2008), a French–American cardiologist, about his personal experience with baclofen for the treatment of his alcohol problem, French AUD patients demanded treatment with baclofen from their general practitioners, and in 2013, about 200,000 patients with an AUDs were treated with baclofen. Since then, many randomized controlled trials and some large cohort studies in both primary care and specialist settings have been performed, including studies with both high and low doses baclofen and targeting either abstinence or reduced drinking. In their scientific and clinical evaluation of the current status of baclofen for the treatment of patients with alcohol dependence, the Cagliari consensus group made a series of recommendations emphasizing that baclofen is not (yet) a proven-effective medication for AUDs, that its use for this indication is off-label, that it should only be considered as a second-line treatment in patients not responding to or with a contraindication for currently approved medications, that dose titration should be according to the adagio "start low and go slow" and that possible adverse events should be carefully monitored (Agabio et al., 2018).
Taking into account the large public health issue at stake, the current treatment situation with very few AUD patients receiving adequate pharmacological treatment and the current scientific benefit–risk evaluation, it is a courageous and justifiable decision of the French National Agency of Medicine and Health Product Safety (ANSM) to support the use of baclofen in alcohol-dependent patients with a maximum dose of 80 mg baclofen per day. We can only hope that other agencies in other countries will take similar decisions to facilitate (controlled) access to promising medications for the large population of AUD patients that currently does not receive potentially life-saving pharmacological treatment. Meanwhile, more research is needed and we should not hesitate to change our decisions based on new data. There are many promising compounds waiting to be tested, registered and implemented in the treatment of (specific subgroups of) AUD patients. The future is bright for both patients and healthcare professionals if patients demand and professionals provide the best available treatment, including well-documented, second-line off-label treatments with informed consent and careful monitoring of the effect and possible adverse events.
Alcohol Alcohol. 2020;55(1):46-47. © 2020 Oxford University Press
Copyright 2007 Medical Council on Alcohol. Published by Oxford University Press. All rights reserved.
