Today premiers an article I reported for The Scientist magazine, where I have worked for almost 4 years. It describes a new movement in medicine in which ALS patients are taking a larger role in their own medical care, even going so far as to organize their own unofficial clinical trials to test experimental drugs. The story is grossly over-researched for a 3,000-word magazine feature, and condensing more than 9 months of reporting, 50 hours of interviews, and 200-some pages of notes into what I hope is a cohesive story was as hard as it sounds. Still, I’ve only just scratched the surface.
My interest in these patient-driven efforts started with an April 2012 Wall Street Journal article about a group of ALS patients who were taking a homemade mixture of sodium chlorite, which they hoped would mimic a chlorite-based drug currently in development. That month, my editor at The Scientist tasked me with writing a short piece on the sodium chlorite “trial” for the magazine. My article, published in July, recounts the story of three patients—Eric Valor, Ben Harris, and Rob Tison—who spearheaded the effort to gather patient results online as ALS patients started ingesting sodium chlorite on their own at home. But it was clear that 700 words could not do it justice. This story is years in the making—from the creation of the US Food and Drug Administration to protect patients from predatory drug salesmen, to patients’ circumvention of the agency’s clinical trial process to test drugs on their own.
Today, FDA administrators are under constant pressure to give dying patients access to experimental treatments earlier and earlier in the drug development process, before it is convinced of a drug’s safety and efficacy. The agency is understandably conflicted. Regulators cannot in good conscience put a drug on the market that they aren’t 100% sure is safe enough to warrant the benefits it could yield. But what is “safe enough?” How are those calculations made? Don’t they depend on circumstance? Surely a patient losing control of his own body and facing imminent death has a different risk-to-benefit calculation than someone much earlier in his disease. And surely they should have a right to partake in decisions regarding their own care.
Today’s article centers on the story of Eric Valor, an active member of the ALS patient community. Eric was diagnosed with ALS in 2005, and his body has nearly completely succumbed to the disease. His mind, however, is as strong and motivated as ever. I have come to know ALS as one of the worst diseases to afflict humankind, leaving its victims paralyzed and bed-ridden in less than 2 years, and usually taking their lives shortly thereafter. But it leaves the mind untouched—which can be a blessing or a curse depending on how you look at it. Eric has decided this is a blessing, and equipped with modern eye-gaze computer technology, he makes full use of his days (or more accurately, his nights, as his average day runs from about 1pm to 3am) researching ALS and advocating for early access to experimental drugs.
In 2011, Eric started taking sodium chlorite and helped organized the patient effort to collect data on the treatment. While wholly patient-organized trials like this are uncommon, and largely limited to the ALS community, patients across the board are becoming more involved in their own health care. Doctors, the FDA, and drug companies all have their concerns about this trend. At the same time, patient involvement is creating a veritable deluge of data, which researchers are only just beginning to take full advantage of.
So, nearly a year into this, I feel like my work has only just begun. There are innumerable stories of inspiring patients, fascinating tales of the successes and failures in the drug development and regulatory world, and countless questions left to be answered. I have aspirations to write a book on this topic someday, but for now, I plan to bring you updates here, in my new blog. I welcome any and all feedback as I go along. Thanks for reading!