From the Wall Street Journal -- many important lessons to be learned -- as well as one really cool name: Merit Cudkowicz.
ALS Study Shows Social Media's Value as Research Tool
A new clinical trial found that lithium didn't slow the progression of Lou Gehrig's disease, but the findings released Sunday also showed that the use of a social network to enroll patients and report and collect data may deliver dividends for future studies.
The study was based on data contributed by 596 patients with the disease, formally called amyotrophic lateral sclerosis or ALS. By showing that the drug didn't have any effect on progression of the condition, it contradicted a small study three years ago that suggested such a benefit was possible.
The new study, published online in the journal Nature Biotechnology, represents an early example of how social networking could play a role in clinical trials, an area of medical science with strict procedures that many would consider especially difficult to apply in the online world.
"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. Standard clinical trials play a central role in the research enterprise of both of those institutions.
Dr. Hartwell, who wasn't involved in the study, said social-network trials aren't likely to replace conventional randomized, double-blinded, placebo-controlled trials, the gold-standard for generating medical evidence. But such trials have become so complicated and time-consuming that new models are needed, he said.
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.
More than 4,300 patients are on the PatientsLikeMe ALS site, where they frequently share information on how their disease is progressing and strategies they are using to fight it.
Jamie Heywood, chairman and co-founder of PatientsLikeMe, said the idea for the new study came from patients. After the 2008 paper reporting lithium slowed down the disease in 16 ALS patients, some members of the site suggested posting their experiences with the drug in an online spreadsheet to figure out if it was working. PatientsLikeMe offered instead to run a more rigorous observational study with members of the network to increase chances of getting a valid result.
The company developed a tool to standardize collection of patient data, including lithium blood levels in patients. They used a questionnaire from conventional ALS trials to gather patients' self-reported data on functions such as swallowing, walking, and breathing.
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.
| Standard Clinical ALS lithium trials | Social Network ALS lithium trial |
Speed | About a year and a half to design and recruit. Additional time to analyze data | Nine months to design, recruit and present preliminary results |
Recruitment | Patients are recruited via doctors, usually at specialist centers in urban areas | Patients self-select through the Internet, regardless of where they live |
Control Group | Patients randomly selected to receive placebo | Patients selected by closeness in historical progression of the disease |
Data Openness | Group data are published | Group and individual data are made available online |
Source: PatientsLikeMe, WSJ research
Write to Amy Dockser Marcus at amy.marcus@wsj.com
