Patients With ALS Disease Crowdsource Their Own Clinical Study

Experiment shows potential value of citizen medicine

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Members of a health-related data-sharing website evaluated the use of a drug for treatment of a debilitating degenerative disease, the first time a social network was used to monitor patient treatment in real time. The results contradicted an earlier clinical study that had shown promise for the treatment of amyotrophic lateral sclerosis, also known as Lou Gehrig's disease.

The effort started after a tiny 2008 clinical study involving 16 patients suggested the chemical lithium carbonate could slow the progression of ALS. The disease gradually robs patients of muscle function, ending in death. One drug, riluzole, has been shown to slow the disease's progression, but the prospect of a new drug excited many ALS patients, most of whom only live about three to five years after diagnosis.

Lithium is already approved to treat a wide range of other disorders, including mental illness, so a group of ALS patients convinced their doctors to provide them with the drug. It wasn't a randomized trial, but for the patients involved, that wasn't the point.

"While not a replacement for the gold standard double blind clinical trial, our platform can provide supplementary data to support effective decision-making in medicine and discovery," said Jamie Heywood, co-founder of the network PatientsLikeMe.

Heywood, whose brother succumbed to ALS in 2006, he has been an advocate for biotechnology to treat ALS and other disorders.

After the 2008 study was reported in the Proceedings of the National Academy of Sciences, PatientsLikeMe created a data-acquisition form that allowed patients to track their disease status over time, as Ars Technica explains. More than 3,500 patients started tracking their progress, with 150 beginning lithium therapy.

To come up with a control sample, PatientsLikeMe used an algorithm to compare patients who took lithium with other patients who had similar disease progression. Ultimately, the social network found lithium therapy was not having any observable effect on the patients who used it — not even a placebo effect. The evidence was reported earlier this month in the journal Nature Biotechnology.

A recent clinical study with similar findings lends even more weight to the patient-generated study, Ars Technica reports.

Though the results were disappointing for ALS sufferers, the study suggests crowdsourced data can be useful for accelerating clinical discovery. PatientsLikeMe has about 100,000 patient members who suffer from more than 500 conditions, so there are likely many potential study candidates and plenty of information waiting to be tapped.