We should expect to see more of this sort of thing. The Wall Street Journal headline says it all: "Frustrated ALS Patients Concoct Their Own Drug". In this case, the drug appears to be sodium chlorite, which is under investigation as NP001 by Neuraltus Pharmaceuticals in Palo Alto. (Let's hope that isn't one of their lead structures at the top of their web site).
It is an accepted part of scientific lore that scientists sometimes use themselves in experiments, and cancer patients and others with life-threatening illnesses are known to self-medicate using concoctions of vitamins, special teas, and off-label medications. But the efforts of patients with ALS to come up with a home-brewed version of a drug still in early-stage clinical trials and not approved by the FDA is one of the most dramatic examples of how far the phenomenon of do-it-yourself science has gone.
A number of patients who have been involved in the Phase II trials of NP001 have been sharing information about it, and they and others have dug into the literature enough to be pretty sure that what Neuraltus is investigating is, indeed, some formulation of sodium chlorite. Here's one of them:
Mr. Valor first read about NP001 in a news release. He tracked down published papers that led him to believe the compound was sodium chlorite, a chemical that in various forms is used in municipal water treatment plants. A friend found online the scientists' patent filings. He also consulted an engineer in water treatment to learn more and read environmental reports to get insight into toxicity levels. The chemical is easy to order online and is inexpensive. He estimates he has spent less than $150 total.
Mixed in distilled water, the sodium chlorite is delivered through Mr. Valor's feeding tube three days a week, one week per month. He says he cautions participants that the chemical isn't as efficacious as NP001 and "that this is only to buy time until NP001 is available to all."
This case is the prefect situation for something like this to happen: a terrible disease, with an unfortunately fast clinical course, rare enough for a good fraction of the patient population to be very organized, along with an easily-available active agent. If NP001 were some sort of modified antibody, we wouldn't be having this discussion (although eventually, who knows?) And as much as I agree that Phase II and Phase III trials are necessary to find out if something really works or not, if I had ALS myself, I'd be doing what these people are doing, and if it were a family member affected, I'd be helping them mix the stuff up. With a condition like ALS, honestly, the risk/benefit ratio is pretty skewed.
If NP001 progresses, look for comment along the lines of "How can this little company get a patent on the use of this common chemical for this dread disease?" But as the WSJ article reports, the sodium chlorite mixtures that people are whipping up in their kitchens don't seem to be as effective as whatever NP001 is, for one thing. And Neuraltus is basically much of their existence on whether it works or not; they're taking on the risk and trouble of a proper investigation, and good for them. But it's true that many people who have ALS right now will not be around to see the end of a Phase III trial, and I can't blame them at all for doing whatever they can to try to get some of the benefits of this research in the interim.