On August 10, 2011, Joan Valor Butler diluted a solution of 5 percent sodium chlorite in 1 gallon of slightly salted water, and slowly injected 1 liter of the mixture into her 42-year-old son's feeding tube, at his request. Sodium chlorite is a chemical commonly used in low concentrations in camping water-purification kits and for municipal water treatment. Many also believed it to be the active ingredient of a promising drug in clinical trials for amyotrophic lateral sclerosis (ALS)—a disease Eric Valor has lived with for the better part of the past decade.
As the disease gradually paralyzed his entire body, Valor became an avid student of ALS, a degenerative disorder commonly known as Lou Gehrig's disease and characterized by a deterioration of motor neurons. He had first learned about the intravenous drug, called NP001, in 2010, when its developer, Neuraltus Pharmaceuticals, announced it would soon be starting a Phase 1 trial and was recruiting ALS patients. Animal toxicology tests had demonstrated the drug's safety, and a small study in an ALS mouse model suggested it may slow disease progression—most likely by reducing macrophage-initiated killing of neurons, a recently proposed mechanism for how ALS wreaks havoc on the body. Valor started a thread on an online ALS patient forum to discuss NP001, and several patients decided to inquire about taking part in the trial. But 7 years removed from diagnosis, Valor's disease was far too advanced for him to qualify as a trial participant.
Though he has outlived the average ALS lifespan of just 2 to 5 years, Valor's muscular paralysis is severe. Within 6 months of his diagnosis, Valor—once an avid surfer and snowboarder—had difficulty walking. He started using a cane, then a walker, then a wheelchair. Eventually the disease confined Valor to his bed, where a ventilator forces air into his lungs 6 to 8 times per minute. By the time NP001 was making a splash in the ALS community, his speech was barely intelligible, and he was unable to move any part of his body except for his eyes and some of the muscles of his face. In his condition, Valor was a risk for any clinical trial testing a treatment expected to slow progression: for the drug to show a noticeable effect, he would actually have to start getting better, rather than simply stop getting worse—something no drug had ever done for ALS patients.
So Valor looked into treating himself at home, scouring the Web through a computer linked to an infrared camera that tracks the movement of his right eye. (See photographs below.) Dozens of other patients who couldn't get into the trial also searched for another way to try the experimental drug. On the basis of a literature search, a Phase 1 trial consent form, and a patent owned by Neuraltus cofounder Michael McGrath, the patients believed NP001 to be a formulation of sodium chlorite. Some purchased NP001's precursor, WF10, which was known to contain just over 50 percent sodium chlorite and could be ordered from Thailand, where it was approved for treating the autoimmune consequences of cancer radiation. But for many, the cost of $12,000 for a year's supply was prohibitive. Sodium chlorite, on the other hand, could be purchased from online suppliers for just $50 per quart—15 years' worth at the dosage outlined in the patients' documented protocol.
There's a new model of medicine in my opinion. You see these DIY programs where patients are deciding, after doing an Internet search, that this is what they want to do; they go get the medicine; they take it; and they log their own output measures.
— Richard Bedlack,
Duke University ALS Clinic
Self-dosing patients went to great pains to ensure that what they were doing was safe. Because WF10 was already approved in Thailand, they knew that at least one form of chlorite had completed safety testing to satisfy that country's regulatory authorities. Patients reached out to the drugs' inventors, and spoke to their doctors and each other about the risks and the proper dosing. Even test engineer Tom Poast, who does not have ALS, chimed in on the patient forum with insights on the purity of chlorite solutions available for purchase, dilution and mixing, and the chemical's behavior. More than 1 year and 1,000 forum posts later, some two dozen ALS patients decided ingesting sodium chlorite was worth a shot.
While the ALS community has taken do-it-yourself (DIY) medicine further than most, patients across the board are starting to play a noticeably bigger role in their own care. "There's a new model of medicine in my opinion," says neurologist and clinical researcher Richard Bedlack, director of the Duke University ALS Clinic. "Once upon a time we had a very paternalistic system, where patients would come, and they would have a set of symptoms, and doctors would ask all the questions and give all the answers. And in the past decade things have really shifted almost to the other side, where a lot of medicine is autonomous now. You see these DIY programs where patients are deciding, after doing an Internet search, that this is what they want to do; they go get the medicine; they take it; and they log their own output measures."
And although Bedlack has concerns about the safety and effectiveness of patient-driven trials, he argues that patients' greater involvement is a good thing—both for managing their own health and for deciding the course of drug development. "I don't think anybody has more to gain or lose from all this than patients," he says. "So I think they should be driving the agenda."
