Earlier this month, the journal Lancet Neurology published a study showing that the generic drug lithium did nothing to slow the course of amyotrophic lateral sclerosis (ALS), a devastating neurological disease. The findings would likely have been a disappointment to patients–they refuted an earlier, much smaller study suggesting that lithium could alter the disease’s rapid decline–but many already suspected this outcome. Eighteen months earlier, PatientsLikeMe, a for-profit patient networking site and data aggregator based in Cambridge, MA, had come to a similar conclusion, much more quickly and at much less cost.
The site, part social networking and part health 2.0, has gathered a wealth of data on its 65,000 members, which span 16 different disease communities, including epilepsy, fibromyalgia, and depression. It provides users with tools to track their health status and communicate with other patients, and then removes the personal details and sells the data to pharmaceutical companies and others. The company’s cofounder, James Heywood, believes the site will ultimately change the way drugs and other interventions are evaluated. Heywood, his brother Ben, and a former MIT classmate, Jeff Cole, founded PatientsLikeMe in 2006 as a way to help a third brother, Stephen, who was diagnosed with ALS in 1998.
The approach won’t replace clinical trials, at least anytime soon. But some experts do believe it could have enormous benefits, highlighting how different types of patients use drugs, when they stop, or what side effects they experience. “The beauty of observational trials is that you can see how an intervention works in the real world,” says Mark Roberts, a physician and professor of Health Policy and Management at the University of Pittsburgh. For example, many trials eliminate patients with secondary ailments, such as renal failure or chronic obstructive pulmonary disorder. “All my patients have those things, so how do I know it works in people I see?” he says.
PatientsLikeMe put its database to the test in 2008, after a small Italian study published in Proceedings of the National Academy of Sciences suggested that lithium could delay the progression of ALS. About 10 percent of PatientsLikeMe’s ALS users began taking the drug, not wanting to wait for a larger trial to confirm the results. Inspired by a member in Brazil who wanted to know if lithium was truly helping, the company rolled out a number of tools to allow patients to track their progress.
The founders, who trained as engineers at MIT, began building models of how the disease typically progressed in individuals with certain characteristics, incorporating variables such as age, gender, disease severity, time since diagnosis, and other factors. Heywood says the models allow researchers to predict the course of an individual’s disease more accurately than the standard prognostic tools. “We can predict when a patient will die 16 months ahead of time, compared to the typical doctor report of ‘you have two to five years to live,’ ” he says.
Because the company had such extensive data on the patients, researchers could analyze how an individual’s symptoms changed 12 months before they began taking lithium, as well as after. Unlike a typical clinical trial, this allowed scientists to search for unique characteristics in the people who decided to take the drug. They found that people who chose to take it were somewhat worse off before starting the drug than those who didn’t. (This group may have been more motivated to try an experimental treatment.)