PatientsLikeMe, a patient social networking and data collection site, has published a patient-driven study refuting a previous paper suggesting that lithium could help people with amyotrophic lateral sclerosis (ALS), a devastating neurological disease. The findings highlight how internet-based tracking tools can enable patients to explore medical questions most relevant to them in a way that augments traditional clinical research.

“This is the first time a social network has been used to evaluate a treatment in a patient population in real time,” said ALS pioneer and PatientsLikeMe Co-Founder Jamie Heywood in a statement from the company. “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. Patients win when reliable data is made available, sooner.”

As I noted in a previous story on the study (before the results were published),

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.

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.)

“The approach has tremendous potential,” said Lee Hartwell, a Nobel Prize-winning scientist now at Arizona State University, and formerly president of the Fred Hutchinson Cancer Research Center, in an article in the Wall Street Journal. Hartwell was not involved in the study.

According to the WSJ,

Paul Wicks, a co-author of the paper, said social network-run studies may be most useful for testing efficacy of so-called off-label or off-patent compounds that patients are using but are unlikely to ever attract pharmaceutical company interest.

In many diseases, “sometimes the alternative is not our way or the old way. It is our way or it is not studied at all,” said Dr. Wicks, the research and development director at PatientsLikeMe, a closely held health-data sharing company in Cambridge, Mass., that ran the lithium study.

… In conventional studies, patients are randomly assigned to a treatment or control group to reduce sources of bias. Neither doctors nor patients know who is getting the drug.

In the on-line study, patients decided themselves if they wanted to take lithium. They needed to persuade a doctor to write a prescription. They were also able to see on the website how others taking the drug were faring in real-time. All of this raised chances that the study could lead to a false conclusion.

To address the concern, PatientsLikeMe developed an algorithm that matched 149 patients taking lithium with at least one other ALS patient on the site who didn’t take the drug. A total of 447 patients were among this group that researchers considered controls.

The study didn’t find any difference in disease progression a year after treatment between those taking lithium and the control group, researchers said.

Mr. Heywood said the result was apparent nine months after the study was launched. Conventional trials typically take more time just to enroll patients, he noted. Costs for drugs and recruiting patients were avoided.

Merit Cudkowicz, an ALS researcher at Harvard Medical School who was an investigator on a standard lithium clinical trial, said social network-generated data can offer valuable insights, but she cautioned that the PatientsLikeMe study was not a substitute for more rigorous studies. Two conventional on-going ALS studies are designed to see if lithium has a very small effect on survival, something the PatientsLikeMe study wouldn’t be able to pick up.

“The thing you don’t want to do in a fatal illness is to throw out potentially good drugs that might have small but meaningful effects,” she said.