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Making Their Own ALS Drug

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.

23 comments on “Making Their Own ALS Drug”

  1. Huh. I don’t know much about drug discovery beyond what I read here, but it surprises me that the patients in the trials apparently need to guess what the active ingredient is.
    Is it really legal to dose human subjects with something without filing some kind of disclosure about what that something is?

  2. luysii says:

    Well, it sounds safer than something some of my desperate ALS patients tried back in the day — snake venom.
    If you really think about what a motor neuron (the type of neuron affected in ALS) must actually accomplish, it’s remarkable that we all don’t have it. It has to maintain a process (the axon) extending from the lower spinal cord (which stops in the lumbar region) to the muscles of the leg. The axon can be 3 feet long (longer in the NBA). All this by a cell body invisible to the naked eye, which is furiously synthesizing as much protein as fast as it can. This is the origin of the Nissl substance of motor neurons which basically tons of ribosomes.

  3. dick says:

    Many years back, a similar “simple chemical” seemed to have a promising future; logical chemical development followed (http://www.ncbi.nlm.nih.gov/pubmed/17211407) and eventually a drug. Sodium chlorite may have a similar development, or not?

  4. BCP says:

    I read this a few days ago and thought “good luck to the ALS patients” but this sounds a little dicey to me. It wouldn’t surprise me to learn that sodium chlorite had a pretty narrow therapeutic index, so making errors in dose outside of a controlled clinical trial worries me. Also, what kind of grade of NaClO2 is being used would be a concern – what stabilizers/impurities are put into commercial supplies that these folks are also ingesting?
    To Henning, @1 – patients don’t normally get to understand chemical structures, rather they should be aware of the risks (as much as they are known) of an experimental agent being used in a clinical trial. The investigators who are running the trial on behalf of the sponsors are aware of the identity of the chemical that they are testing however.

  5. Volphar says:

    Will this become more common as Industry clinical trials target smaller and smaller sub-populations?

  6. LittleGreenPills says:

    The compound at the top of their website looks like gossypol. After a quick look around Wikipedia, I would not be surprised if they had not at least looked at it. Some of its targets appear to overlap with their therapeutic interests.

  7. Greg Q says:

    I’d title this a prime entry in “why the FDA needs its powers scaled back.” These people shouldn’t HAVE to be “whipping up” the drug in their kitchens. They’re adults, with a deadly disease, and they don’t have time to wait. Let them have control of their own bodies, let them buy NP001 from Neuraltus Pharmaceuticals.
    The FDA should be free to say “we think you’re an idiot for buying this.” No one should have the power to tell those people “you aren’t allowed to buy this.”

  8. NJBiologist says:

    @Derek: “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.”
    You’d think that. Really, it just seems like common sense.
    But a review of some of the posts on melanotan.org show that people mail-ordered the peptide API from Palatin Technologies’ Bremelanotide, dosing themselves in the search for enhanced libidos and erections.
    @7–Personally, I think the melanotan.org people are an example of why the FDA should retain it’s authority on things like this.

  9. cynical1 says:

    I think NP001 is a mixture of tetrachlorodecaoxide used in combination with the chlorite, not sodium chlorite alone.

  10. anonymous says:

    @7 Greg Q: The FDA as we know it evolved from 100 years of snake oil, poison, and inadequately tested drug crises. There is always someone who will attempt to profit off the situation you describe without providing any benefits.

  11. Bruce Hamilton says:

    Not sure, haven’t searched literature, but this site may have the structure of NP001.
    http://www.als.net/ALS-Research/NP001/ALS-Topics/
    Bruce Hamilton

  12. MoMo says:

    Good for you Neuraltus!
    Don’t let these naysayers here get you down, especially if you are testing that chromane compound instead of an industrial oxidizer.
    But you are into chemical space hardly ventured into by the Big Boys- Far too simple for their complex Egos.
    But what Mental Giant thinks NP001 is sodium chlorite?

  13. milkshake says:

    that chromane would be a delight to make, from 2-acetylphloroglucinol (aka trihydroxyacetophenone) which costs $5/g at Aldrich and acetone and a dash of pyrrolidine in one step, possibly in the kitchen

  14. Bruce Hamilton says:

    Somebody misheard chromite from chromane?.

  15. Bruce Hamilton says:

    Just took time to check US patents. Neuraltus has several US patents for pH-buffered sodium chlorite formulations for neurodegenerative diseases, mainly in 2011, but starting in 2006.
    The site I pointed to above probably has an incorrect structure for NP001.

  16. MoMo says:

    Thanks Milkshake and Bruce for the recipe and fact checking.
    Ye Gods! What’ll they think of next! Industrial Oxidizers to chemically blanch your neurons! Brilliant!

  17. Lu says:

    Well, my cousin whipped up a drug Cinnarizine for grandma after stroke. I knew terminal cancer patients who drank kerosene which is a popular snake-oil treatment in Russia. But bleach? Ingesting industrial bleach?

  18. Ben Harris says:

    Hey everyone, just stumbled on this site. I am one of the people with ALS mentioned in the WSJ article. I’d be happy to answer any questions.
    Thanks,
    Ben

  19. Hans says:

    It’s pretty sure sodium chlorate is the main ingredient, but perhaps there’s other ingredients in NP001. There’s a lot to be found about the safety of SC because it’s used in the production of drinking water. So it’s a calculated risc we’re taking. Of all the things i tried, this is the easiest one. And very cheap too.

  20. Jonadab says:

    > With a condition like ALS, honestly,
    > the risk/benefit ratio is pretty skewed.
    With late-state ALS, are risks even relevant?
    What could a drug do to make your situation very much worse? I mean, let’s just say for the sake of argument that the drug has a 2% chance of helping you a little with your ALS and a 50% chance of giving you kidney stones, as well as (independently) a 30% chance of damaging your liver. I imagine there would be patients who would want to take it. What’s a couple of kidney stones next to unrelenting body-wide agony, and what good will a healthy liver do you when you’re dead?
    On a side note, Lou Gehrig’s Disease would be a great name for a heavy metal band.

  21. Luke Weston says:

    Personally, I think this smells like it’s a reboot of the “Miracle Mineral Solution” pseudomedicine scam.
    http://www.guardian.co.uk/science/2010/sep/15/miracle-mineral-solutions-mms-bleach

  22. ENV says:

    it does share some similarity with MMS. But MMS is touted as a cure-all. NP001 has good preclinical and P1 data to support efficacy and we are just trying to see if those of us who don’t qualify for the trial can get a little benefit until NP001 is available. In my case the answer is yes.

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