The ALS Association

Ice Bucket Challenge Progress

Patient Involvement in Clinical Trials Critical to Finding New Treatments for ALS

April 25, 2011

An article published in Nature Biotechnology reports that data gathered on an online patient social media site, “PatientsLikeMe,” supports information discovered during a clinical trial investigating the use of lithium as a treatment for ALS. Click here to download a PDF of the article.

"We all have a responsibility to encourage patients to understand the value of enrolling in clinical trials so we can advance our understanding of the disease” said Director of Research and Development at PatientsLikeMe Paul Wicks, co-author of the article.  “PatientsLikeMe is not a replacement for these pivotal trials, but research done collaboratively with patients using our system supplements and complements the great work that is being done every day by clinical trial networks such as those supported by The ALS Association.”

Indeed, clinical trials for ALS have been important as they were able to identify the compound Riluzole that, although modest, has an effect on survival among people with ALS.  This could not have been detected with the method proposed in the publication.  The ALS Association through partnership with The Northeast ALS Consortium (NEALS) as part of TREAT ALS/NEALS clinical trials network enables the standardization, centralization and rapid design and implementation of clinical trials, which is now being applied globally.

“NEALS, a non-profit organization that performs high-quality research has used this site to help find people with ALS to enroll in clinical studies,” said ALS Association Chief Scientist Lucie Bruijn.  “The ALS Association looks forward to working with PatientsLikeMe to encourage enrollment in clinical trials for ALS.”

“PatientslikeMe has developed an excellent community for patients to share what they are doing for their illness and what people with ALS view as most important.  It also allows easy access to information about ongoing clinical trials, said Merit Cudkowicz, M.D., M.Sc., Professor of Neurology at Massachusetts General Hospital, at Harvard Medical School.  “It is not clear whether this approach would pick up a positive treatment effect and reliably exclude a negative one.  The field would not want to throw out prematurely any treatment that has chance of success.”

The publication of the article in Nature Biotechnology led to an article published in Wall Street Journal, visit

In addition, two important trials were recently presented at the 2011 American Academy of Neurology: dexpramipexole ( and cytokinetics (  Both showed a positive effect in phase I/II trials and are now moving forward to larger trials.

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