Amyotrophic lateral sclerosis (ALS) is a pretty grim disease. Victims experience progressive muscle weakness, leading to death; patients survive a median of only two to five years following the onset of symptoms. Currently, there are no effective therapies. So it's not surprising that when a preliminary study shows a hint of efficacy, patients will do what they can to get access to the drug and share their experiences with fellow patients. A company called PatientsLikeMe has now used this tendency to share information to crowdsource a sort of clinical trial, obtaining data on the effectiveness of off-label use of a drug.
Although ALS remains untreatable, it's a focus of active research, and promising results have been seen in some animal models and small clinical trials. One of these, a 2008 study, involved only 16 patients, but suggested that a simple chemical, lithium carbonate, could significantly slow ALS progression. With no other treatments and the drug already on the market, a number of patients convinced their physicians to provide the lithium carbonate off-label. Although these patients didn't provide a randomized clinical trial population, they did present the opportunity for at least testing whether the lithium carbonate might have an impact on disease progression.
To get an answer, the site put together a data-acquisition form that enabled patients to input their disease status over time using a standardized assay for ALS symptoms. All told, over 3,500 patients started tracking their disease progression; 150 of them started taking lithium, with nearly 80 continuing the treatment for a year.
A control sample was identified by comparing disease progression profiles (how rapidly symptoms got worse) from those who started lithium to those who hadn't. By matching similar progression curves, the authors felt they would increase the probability of providing a relevant comparison.
When the numbers were crunched, the lithium carbonate did not come out looking good. There was essentially no difference at all between those who took the drug and those who didn't when it comes to ALS progression. This result is the same as one obtained from a larger clinical trial that was completed recently. One somewhat surprising result is that the drug didn't even exhibit a placebo effect, which is rather unusual.
So even if the results weren't very promising for those with the disease, the approach looks like it has potential. The authors go out of their way to point out that "observational studies using unblinded data are not a substitute for double-blind randomized control trials," and warn that ALS patients may be somewhat unusual, in terms of their high motivation to seek out any alternative treatment. Still, they conclude that even with its limitations, "data reported by patients over the internet may be useful for accelerating clinical discovery and evaluating the effectiveness of drugs already in use."
There's a little added motivation for focusing on ALS by the team behind the website, too. The MIT engineers that founded it have a brother with the disease. Despite that focus, however, the site currently claims to be tracking over 500 other diseases.
Nature Biotechnology, 2011. DOI: 10.1038/nbt.1837 (About DOIs).
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