Scientists Like You: Using Social Media to Advance ALS Research

by Miriam Davidson on Fri, 2011-07-01 14:29
Article Highlights:
  • People with ALS may be able to advance research by participating in studies via social media.
  • The article explores the pros and cons of social media-based studies that rely on self-reporting data.
  • Medical experts caution that social media studies — which may enhance ALS research — shouldn't be replacements for traditional clinical trials.

Karen Felzer studies earthquakes, not ALS.

But after her father learned he had the disease, Felzer helped conduct a study that caused a small shake-up in the world of ALS research.

Working with Humberto Macedo, a Brazilian computer analyst who also had ALS, Felzer initiated an online, patient- and caregiver-directed study of the mood drug lithium, which reportedly had been found to dramatically increase survival time in ALS.

“When the news came out that lithium was supposed to be this wonder drug, we were very optimistic,” said Felzer, who has a doctorate in geophysics from Harvard and works for the U.S. Geological Survey in Pasadena, Calif. “And if it was really going to help, we wanted to be able to demonstrate that it did.”

Karen Felzer with her baby Emmet and late father, Alan.

Felzer and Macedo posted messages on online forums to recruit some 200 ALS patients who wanted to take lithium, set up a website to track their self-reported data and created an algorithm to analyze the information.

The pair later joined forces with the website PatientsLikeMe, which provided additional study tools, including matching controls (people of similar ages and stages of ALS progression who weren’t taking lithium) and a more rigorous algorithm.

The results of PatientsLikeMe’s yearlong study confirmed Felzer and Macedo’s earlier, disappointing result: Lithium didn’t work.

But the method used to arrive at that conclusion — online, self-reporting by patients, with matched controls and immediate results — demonstrated that people with ALS (and other conditions) may be able to play a role in advancing research by participating in social media.

Promise and pitfalls

ALS experts say the PatientsLikeMe lithium study reveals both the promise and the pitfalls of this trend.

The advantages include:

  • speeding up the pace of clinical trails, especially recruitment of participants;
  • supplementing information on side effects and adverse events of proposed treatments;
  • providing information on off-label, alternative and fraudulent treatments;
  • lowering the cost of clinical trials; and
  • quickly identifying both promising avenues of inquiry and drugs that are unlikely to work.

But disadvantages are numerous, including:

  • unknown source and quality of data (for example, diagnoses have not been verified);
  • potential over-, under- or misreporting of symptoms;
  • possible placebo effect unaccounted for;
  • selection bias (for example, younger people are more likely to use social media);
  • trial dropouts may be unaccounted for;
  • statistical interpretation of results may be incorrect;
  • encourages people to self-medicate; and
  • confidentiality may be compromised.

“It’s a new field, and we’re still in a learning period,” said neurologist Merit Cudkowicz, director of the MDA/ALS Center at Massachusetts General Hospital in Boston and a member of MDA’s Medical Advisory Committee.

Cudkowicz urged caution going forward. She warned that such studies must be well-designed and the results correctly interpreted and validated in order to avoid coming up with wrong answers, or missing safety problems or important treatment effects.

Social media studies may be useful in patient education, information sharing and improving enrollment in standard clinical trials, Cudkowicz said, or for generating hypotheses that then could be tested through more rigorous methods.

A social media component also could be added to traditional clinical trials to make them more efficient, noted MDA Vice President of Research Sanjay Bidichandani.

“Via empowerment of patients, social media can speed up and increase enrollment and ease of reporting symptoms,” Bidichandani said. But he and Cudkowicz strongly emphasized that social media-based studies are not a replacement for traditional clinical trials.

The lithium study

The originators of the online lithium study — Karen Felzer and Humberto Macedo — are examples of the “patient push” driving social media research. Both were professionally educated, computer literate and had a direct emotional connection to the topic: Felzer was helping care for her father Alan, an engineering professor who had ALS, and Macedo, a 42-year-old father of six, had ALS himself.

After working on the project on their own for a while, the pair ultimately turned the study over to PatientsLikeMe, a health data-sharing website that already had thousands of people with ALS reporting regularly on what drugs they take, what symptoms they experience and other information.

PatientsLikeMe charted and tracked the data, established the control group, conducted the analysis and, earlier this year, published the results in the scholarly journal Nature Biotechnology. The study ended up being more about the utility of social media in research than whether or not lithium works. (Other, more traditional studies were already confirming that it didn’t.)

Tom Masters, 54, of Anaheim, Calif., who received a diagnosis of ALS in 2008, was one of the participants in the lithium study. He said he entered his data — including scores from his self-administration of the Revised ALS Functional Rating Scale — into the PatientsLikeMe site about every two weeks. It took about 10 minutes each time.

Even though lithium didn’t work for him, Masters said he would definitely participate in such a study again. “The advantage is time to results,” he said in an email. “Instead of waiting years, results can be had in months.”

Nevertheless, questions remain about the quality of the self-reported data in the study. How do we know these people even have ALS? Or that they’re reporting their symptoms accurately? According to PatientsLikeMe, about 70 percent of trial participants gave the name of their diagnosing physician, but the site was unable to independently confirm participants’ diagnoses.

Felzer, who is trained in statistical analysis, believes that the large size of the final study group (149 people taking lithium and 447 who weren’t) should “wash out” the effects of people who might not be reporting their symptoms accurately.

She also noted that, when studying a drug as inexpensive and widely available as lithium, people have little incentive, financial or otherwise, to fake data. But if the drug or treatment wasn’t easy to obtain and/or profits were at stake, the situation might be different.

Tom Masters, shown top left with wife Heidi, participated in the online lithium study by entering personal health data into his page on the PatientsLikeMe website about every two weeks. Experts caution that social media-based studies must be well-designed and correctly interpreted to avoid coming up with wrong answers, or missing safety issues or important treatment effects.

Social media research boom

PatientsLikeMe, which now has more than 100,000 people entering data on some 500 different medical conditions, may be the most comprehensive, but it is only one of a growing number of health data-sharing websites.

Some of these sites are disease-specific. The Life Raft Group, for example, focuses on a rare form of intestinal cancer and limits its membership to patients and family caregivers. It functions as an online support community that also conducts research on drugs and treatments its members are taking.

Another site, 23andMe, which focuses on genetics, just published its first peer-reviewed, scientific study utilizing self-reported data from its members. The site hosts a research arm, called 23andWe, that collects patient-entered information on various conditions.

Researchers have found that sites like these, and networking tools like Facebook and Twitter, are useful for disseminating information and for identifying potential study subjects. But the open channels of communication can cut both ways.

In the last few years, investigators in Canada have found themselves inundated with requests from people using social media who want them to study an unproven treatment for multiple sclerosis that involves widening patients’ veins.

“The case indicates the unprecedented pressure scientists … worldwide now face to alter research priorities even in the absence of credible scientific evidence,” the Canadian physicians recently wrote in the journal Nature.


One research website that is open to social media-based inquiries is ALSUntangled. The site was founded in 2009 by a group of respected physicians from the World Federation of Neurology — some MDA affiliated — who wanted to provide scientific answers to the many questions posed by ALS patients and their families about alternative, off-label and experimental treatments.

Using Twitter, ALSUntangled takes suggestions for treatments and therapies to be studied and then conducts quick reviews of available research on the most popular topics. It also sometimes conducts its own investigations (for example, visiting stem cell clinics or interviewing people who claim to have found “cures” for ALS).

ALSUntangled so far has published on its site the results of 10 reviews of alternative and off-label therapies (see Naltrexone: benefits questionable, harm possible). It is collaborating with PatientsLikeMe and other websites to provide accurate and comprehensive reviews of purported ALS treatments and to warn people about frauds and rip-offs.

Going forward

In the three years since Karen Felzer and Humberto Macedo began their innovative project, both Felzer’s father and Macedo have passed away from ALS. Neither benefited from the study they helped pioneer.

But the work they inspired lives on, providing new tools and methods to researchers hoping to speed the search for ALS treatments.

For more information

  • “Accelerated clinical discovery using self-reported patient data collected online and a patient-matching algorithm,” Nature Biotechnology, April 24, 2011.
  • “The rise of people power,” Nature, April 28, 2011
  • Numerous news articles also are available on this subject under the search terms “social media and research.”

In a series of videos produced by the American Society of Nephrology, a Mayo Clinic physician and a social media expert discuss how physicians, researchers and patients can use current social media tools to benefit their patients, themselves and their organizations.

Victor Montori, medical director of the Mayo Clinic Center for Social Media, explains how social media allows organizations to participate in “unmediated” conversations with patients. Lee Aase, of the Mayo Clinic Center for Social Media, discusses organizational uses of social media.

Social media provide direct patient benefits, say these experts. Research results and the most effective therapies can be disseminated more quickly, and patients learn more about managing their health. Health care organizations can teach the public how to make sense of scientific studies and the public then can spread this information, improving overall health literacy.

Mayo Clinic’s Center for Social Media hosts a wealth of information on its recently launched Social Media Health Network.

Check out these YouTube videos:

Miriam Davidson
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