A medium-length article in The Scientist describes patients with fatal diseases taking their treatment into their own hands. Here’s what happened with lithium and ALS:
Humberto Macedo, an ALS patient in Brazil, started a Google Docs spreadsheet to track self-reported ALSFRS-R scores. And Karen Felzer, a research scientist on the US Geological Survey’s Earthquake Hazards team whose father had ALS, built a website to host the project. At 3 and 6 months, Felzer, who has a background in statistics (normally devoted to analyzing earthquake aftershocks), examined the data. Both times, she found no evidence that lithium slowed progression. By November 2008, when Felzer posted her second report on the project’s website, most patients had stopped taking the drug.
Drug companies don’t like the new movement:
Drug companies are understandably wary of any movement that could jeopardize their chances of success, including patient-initiated trials. Drug developers go to great lengths to control the variables in clinical trials, to optimize the dosing and the treatment window in order to reduce side effects while maximizing therapeutic gain, and to monitor patients’ health. If patients outside the clinical research system start taking experimental drugs on their own, the likelihood of something going wrong is greatly magnified. And if something does go wrong—something that may not have been caused by the drug at all—entire drug development programs could be shut down prematurely.
The author of the article, Jef Akst, was impressed enough to start a blog about the subject.