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Medical Mavericks

ALS patients take their fate into their own hands, self-administering an unapproved chemical and collating their results online.

By | July 1, 2012

MRI scan of the brain of a patient diagnosed with ALS RADIOPEDIA.ORG

Eric Valor used to be an avid surfer, catching waves off the California coast near his home in Santa Cruz every weekend, and occasionally early in the morning before work. But in October 2004, his left foot started misbehaving, failing to find the correct position on his surfboard when Valor popped to his feet. “The foot would drag on takeoff, resulting in poor placement and subsequent wipeout,” he recalls.

After a series of minor muscle twitches, his dragging foot was the last straw, that finally got him to the doctor. In April 2005, Valor was diagnosed with amyotrophic lateral sclerosis (ALS). By that fall, his surfing days were over.

Valor is now paralyzed from the neck down, and he breathes with the help of a respirator. But he’s not giving up. In June 2010, he learned of an experimental ALS drug called NP001, being developed by Palo Alto–based Neuraltus Pharmaceuticals. Then just beginning Phase I trials, the drug targets ALS patients’ overactive immune cells, attempting to reduce the chronic neuroinflammation associated with the disease. It seemed promising to Valor, but unfortunately, his disease was too advanced for him to qualify for the trial. “I made various attempts to get [enrolled], but failed,” says Valor, who responded to The Scientist via e-mail because his ventilator limits his speech.

The more I learned about sodium chlorite, the more excited I got, and I felt it was too important to keep a secret. —Ben Harris, ALS patient

If he couldn’t participate in the trial, maybe he could get the drug another way, Valor reasoned. After an exhaustive literature search on PubMed, he identified the drug’s “cruder” precursor as WF10, which is available for purchase abroad. But he quickly learned that importing it from Thailand would cost more than $12,000 for a year’s supply. He then set out to see if he could get his hands on what he suspected—based on a literature search and a 2006 patent tracked down by fellow ALS patient and friend Rob Tison—was the active ingredient of NP001: sodium chlorite, which is used in water-purification kits for campers and in municipal water treatment.

Last August, Valor began taking oral doses of the chemical, which is not approved for the treatment of any disease. And he thinks it’s working. “I have improved breathing, which makes transfers [off his ventilator] much more comfortable,” Valor says. “My voice is louder and speech somewhat clearer. I am able to flex muscles that were previously still (though not enough for useful movement).”

At first, Valor didn’t tell other ALS patients about his experimentation, hoping to first establish that it was safe, “but by September, it had leaked,” he says. Medical physicist and ALS patient Ben Harris, who was enrolled in the NP001 trial, was the one to get the ball rolling. NP001 had improved his strength, speech, breathing, and ability to swallow, and he wanted to keep taking the drug after his participation in the trial ended. “The more I learned about [sodium chlorite], the more excited I got, and I felt it was too important to keep a secret,” says Harris, who also responded to questions via e-mail due to limitations associated with his disease. So he started a discussion thread on the website of Cambridge, Massachusetts-based biotech ALS Therapy Development Institute (ALS TDI), sparking widespread interest that helped launch the do-it-yourself (DIY) trial. Now more than two dozen other ALS patients are taking oral sodium chlorite, and recording their results on the social networking site PatientsLikeMe.

It’s not the first time PatientsLikeMe has hosted this sort of DIY experiment. “This is kind of an ongoing process on our site,” says company cofounder and chairman Jamie Heywood, a mechanical engineer turned biomedical researcher who also founded ALS TDI in 1999, after his brother was diagnosed with the disorder. Just last year, for example, Heywood and his colleagues published an analysis of data reported on PatientsLikeMe by ALS patients taking lithium carbonate, a chemical approved for the treatment of bipolar disorder (Nat Biotech, 29:411-14, 2011).

But this sodium chlorite case “is particularly interesting” and “radical” in many ways, Heywood says. “People are buying [sodium chlorite] from chemical companies; [they’re] not buying a human therapeutic.” Furthermore, most patients reporting their data on PatientsLikeMe are taking sodium chlorite orally, whereas Neuraltus is giving patients NP001 intravenously. And Heywood is still not sure if sodium chlorite is the true active ingredient of NP001. “Without question in ALS, this is the biggest topic of conversation that we as researchers [and doctors] are talking about right now,” Heywood says.

But the reports on PatientsLikeMe have put Heywood in a tough position. On the one hand, “I think there’s a moral obligation to deliver value in real time,” he says, looking at an analysis of the data on his computer screen. On the other hand, “I know that it will be misinterpreted by many people,” he laments. “The hard question for us is, ‘What’s the right thing for us to do? How do we share that information?’”

Fortunately, the risk is moderate, Heywood believes, as do the patients taking sodium chlorite, most of whom have done some research before ingesting the chemical. “I have done hundreds of hours of research to prove to myself that what I am doing is safe,” says Harris. “Moreover, if you add up the time spent by other forum contributors, we are probably talking about thousands of hours. . . .The story is not about a few desperate people with a terminal illness grasping for anything. . . .This is a solid scientific venture, it just so happens it is being done outside of an academic institution.”

Correction: This story has been updated from its original version to reflect that Valor started taking oral sodium chlorite in August 2011, not June. The Scientist regrets the error.

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Comments

Avatar of: Kathy Barker

Kathy Barker

Posts: 9

July 4, 2012

Terrific article and a great example of citizen science/medicine!

Avatar of: Bill

Bill

Posts: 1457

July 10, 2012

"This is a solid scientific venture, it just so happens it is being done outside of an academic institution."

So I can be assured there is a control arm to this "study?"

Avatar of: EllenHunt

EllenHunt

Posts: 74

July 25, 2012

It is perfectly acceptable in the case of ALS to use the large volume of ALS patients who are simply not in the study as a control. ALS is not particularly responsive to placebo treatment.

There are peer reviewed articles that utilize previously recorded outcomes as the control. This is ethically necessary when a condition being treated is relatively rare and/or the progress of the disease is well established to be fatal.

What throws off the results of clinical trials is not lack of controls. What throws results is cherry picking and manufacturing of data.

The biggest problem this clinical trial will face is recording relative doses and adherence to protocol.

Avatar of: Barbara Oakley

Barbara Oakley

Posts: 1

July 25, 2012

What an interesting article!  Yes, it's a different approach, but it's nice to know see that intriguing information can be gleaned outside the usual pathways of science, using an "Army of Davids" approach.

Avatar of: EllenHunt

EllenHunt

Posts: 74

July 25, 2012

Good for them.

Avatar of: MikeSerfas

MikeSerfas

Posts: 35

July 25, 2012

I am very pleased to see these patients taking the initiative and seeking answers; I respect them deeply and wish them all the luck in the world.  But tracking down the study at http://www.patientslikeme.com/... (also see https://sites.google.com/site/... ) --- as _science_ it is, alas, simply not good enough.  I don't see a randomized control group, and the assessment of "slight improvement" or "major improvement" can be influenced by subjective factors.  Even if a single physician diagnosed a group of patients with identical disease, and half of them chose on their own initiative to try chlorite with beneficial result, the choice might reflect a difference in mood or energy level that actually predicted the course of the disease, rather than effective treatment.  And the placebo effect might influence the symptoms a patient observes, simply due to increased attention or effort.  These patients, of course, have every right to put their own lives ahead of science, and to pursue any possibility - I hope to God they are right.  In ancient times some of our most important medicines were discovered without using formal control groups.  But if they are to _prove_ the treatment is fully effective, they will need to improve their techniques - collectively sacrificing some of the health benefits they might have obtained, for those receiving control treatments.  It is a terrible choice to have to make.

Avatar of: MikeSerfas

MikeSerfas

Posts: 35

July 25, 2012

(Sorry, duplicate posting)

Avatar of: Steven

Steven

Posts: 1457

July 25, 2012

Can't blame them for taking a risk. What have they got to lose?

Avatar of: Susan Runowicz-Smith

Susan Runowicz-Smith

Posts: 1457

July 25, 2012

Chock this one up to the power of the internet. Coupling global information sharing with people passionate about a subject enables a new approach to drug trials and discovery. 

Avatar of: mleuther

mleuther

Posts: 1

July 25, 2012

mikeserfas, could a less than perfect control group be established? Obviously,  self medicaters have a different psycho/social profile but physiological measurements  could be standardized.   Does the NIH or NCI have a department to help co-ordinate self medicaters, a non interference group that would strive to get some stats on what people are doing anyway.  A lot might be learned either way.

Avatar of: Joanne Drayson

Joanne Drayson

Posts: 1457

July 28, 2012

When the patient is a doctor he can help drive the science and medicine - (soon to be published data) listen to Dr Martz speak on a video 
http://lookingatlyme.blogspot....
Diagnosed with ALS by top US University hospital in 2003 he went on to discover it was an underlying bacterial infection- since his recovery he opened a clinic and treated 850 patients diagnosed with ALS, MS and Parkinson's disease.

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