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The perils and pitfalls of “patient-driven” clinical research

Dying of cancer can be a horrible way to go, but as a cancer specialist I sometimes forget that there are diseases that are equally, if not more, horrible. One that always comes to mind is amyotropic lateral sclerosis (ALS), more commonly known as Lou Gehrig’s disease. It is a motor neuron disease whose clinical course is characterized by progressive weakness, muscle atrophy and spasticity, with ultimate progression to respiratory muscles leading to difficulty breathing and speaking (dysarthria) and to the muscles controlling swallowing. The rate of clinical course is variable, often beginning with muscle twitching in an arm or a leg or slurring of speech. Ultimately, however, ALS progresses to the loss of ability to move, speak, eat, or breathe. The most common cause of death is from respiratory failure, usually within three to five years after diagnosis, although there is the occasional outlier with a less malignant form of the disease with a slower course of progression who can live a long time, such as Steven Hawking.

In other words, ALS is a lot like cancer in some ways. It is a progressive, fatal disease that usually kills within a few years at most. On the other hand, it is different from cancer in that, at least for many cancers we actually do have effective treatments that prolong life, in some cases indefinitely. In contrast the most effective treatment we currently have for ALS is a drug (riluzole) that is not particularly effective—it prolongs life by months—and can be best described as better than nothing, but not by a whole lot. So it is not surprising that ALS patients, like cancer patients, become desperate and willing to try anything. This is completely understandable, but sometimes this desperation leads to activities that are far more likely to do harm than good. I was reminded of this when I came across a post in the antivaccine propaganda blog, Age of Autism, referring to an article in The Scientist entitled Medical Mavericks. The fortuitous posting of this story, which was apparently designed to try to show that it’s not as crazy as critics have said to be treating autistic children with “Miracle Mineral Solution” (MMS) (which is a bleach) given that the introduction explicitly mentioned Kerri Rivera and the patient described in the article used sodium chlorite to treat his ALS, provided me the opening to discuss a group whose existence and advocacy brings up a complex tangle of issues that boil down to questions of how far patient autonomy should be allowed to go. I’m referring to a company, PatientsLikeMe, which describes itself thusly:

PatientsLikeMe was co-founded in 2004 by three MIT engineers: brothers Benjamin and James Heywood and longtime friend Jeff Cole. Five years earlier, their brother and friend Stephen Heywood was diagnosed with ALS (Lou Gehrig’s disease) at the age of 29. The Heywood family soon began searching the world over for ideas that would extend and improve Stephen’s life. Inspired by Stephen’s experiences, the co-founders and team conceptualized and built a health data-sharing platform that we believe can transform the way patients manage their own conditions, change the way industry conducts research and improve patient care.

The first thing that needs to be kept in mind is that PatientsLikeMe is a for-profit company. Consequently, it’s selling a product, and that product is its analyses of the information patients who sign up with the service reveal about their experiences with various medicine and products. It is true that the company has an admirable list of core values, such as putting patients first, promoting transparency, fostering openness, and creating “wow” (i.e., information that “wows” you), but the company is still selling a platform to its partners and using that platform to attract investors. It is described as a platform “where patients can share and learn from real-world, outcome-based health data.” In essence, PatientsLikeMe is a social media company for patients that mines the reported experience of its patients as, if you believe the hype, thousands of “N of 1″ clinical trials.

What I see PatientsLikeMe as doing is trying to mine a very unreliable source, namely thousands of testimonials.

Bleach for ALS?

I first learned about PatientsLikeMe four or five years ago, in part because in my pre-SBM blogging life I had written a series of posts about self-experimentation by cancer patients with an unproven, unapproved drug, dichloroacetate (DCA), a saga I later summarized for SBM two years ago. As regular readers might recall, DCA is a small molecule drug that was used to treat congenital lactic acidosis in children through its inhibition of the enzyme pyruvate dehydrogenase kinase. This inhibition shifts the metabolism of glucose towards oxidative metabolism in the mitochondria and away from glycolysis, the product of which is lactic acid. In January 2007, Dr. Evangelos Michelakis of the University of Alberta had the clever idea of using DCA to target the Warburg effect in tumors and published an article in Cancer Cell describing preclinical experiments in rats in which DCA resulted in marked shrinkage of multiple tumor types. The Warburg effect was first described in 1928 by Otto Warburg and refers to the tendency of many tumors to rely on glycolysis for their energy supply rather than oxidative phosphorylation, even in the presence of oxygen, which in the case of normal cells favors oxidative metabolism. It turns out that DCA showed some promise against glioblastoma in a phase I clinical trial, but it is a long way from being approved.

Because DCA is a small molecule and relatively easy to synthesize, a man named Jim Tassano decided that he would sell “pet DCA” to treat cancer in pets. Tassano fooled no one, of course; people were not buying DCA to treat their dogs. His real purpose was to take advantage of desperate cancer patients, some of whom flocked to his BuyDCA.com website to purchase what he claimed to be pharmaceutical grade DCA. It also led to what I saw as a most disturbing phenomenon, namely self-experimentation by cancer patients with DCA and discussion among patients on online forums, which was even likened to “clinical trials,” complete with reports of success based on magical thinking more than anything else. You will see echoes of this same story and the issue it raises in the article referenced by AoA.

This brings us to the link between MMS and PatientsLikeMe. MMS, as you recall, is being touted as a “miracle cure” for conditions as disparate as cancer and autism. It is nothing more than a 28% solution of sodium chlorite, a chemical widely used for water purification. A couple of months ago at that yearly quackfest of the “autism biomed” movement, Autism One, Terri Rivera gave a talk in which she advocated using MMS to treat autism. Her method of delivery included oral, bathing in it, and per rectum, as in MMS enemas. Yes, we’re talking bleach enemas to treat autism. Administered orally according to Rivera’s protocol, the amount of chlorine dioxide (the active chemical liberated when sodium chlorite is mixed into aqueous solution) is more than 120 times the amount that a child could expect to consume drinking tap water.

It turns out that the story being touted by AoA is about a man named Eric Valor who is taking sodium chlorite to treat his ALS:

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.

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.

This is an interesting story on many levels, not the least of which is the apparent finding that sodium chlorite might have an actual therapeutic use. But what is NP001? Due to its proprietary nature, it is not straightforward to figure out just what NP001 is from online sources. For instance, I found what was purported to be the structure of NP001 at the ALS Therapy Development Institute. Notice that there’s no chlorine atom anywhere in chemical structure, making it incredibly unlikely that this molecule would generate chlorite as its active compound. However, more searching around the web made me question whether this is the actual structure of NP001, starting with this press release from the company describing promising results for the drug in a phase I study. Unfortunately, the trial appears not to have been published yet (PubMed searches using the usual suspects, such as “NP001,” “Neuraltus Pharmaceuticals,” and the study’s principal investigator Robert G. Miller, among other strategies, failed to turn up anything); so I went to patent searches and found that Neuraltus Pharmaceuticals has patents for various buffered release systems for sodium chlorite, for example, this one, although searching using the term “NP001″ didn’t turn up anything related to ALS. Elsewhere, it is stated that NP001 is a modified form of WF10, which is basically sodium chlorite and has been tested with varying results as an immune modulator for various conditions. In brief, WF10 is a stabilized chlorite matrix that appears to have immunosuppressive effects, although it also appears to stimulate natural killer cell cytotoxicity and decrease macrophage activity. This patent describes the claims for WF10.

To sum it all up, the best that I can figure out is that NP001 appears to be some form of sodium chlorite in a matrix designed to control its release, perhaps with another compound. Certainly, this FDA application for orphan drug status by Neuraltus Pharmaceuticals, Inc. for sodium chlorite pretty much cemented in my mind that that’s what NP001 is, as did the information on this discussion forum, which described how WF10 and NP001 relate to the drug from Thailand referenced in The Scientist article. Moreover, NP001 was granted not only orphan drug status but fast track status as well.

Now here’s the problem with the sort of “do it yourself”-style “N of 1″ clinical trials advocated by PatientsLikeMe:

Last June, 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).”

So, basically Valor is taking MMS for his ALS. We don’t know the concentration or the dose that he’s taking. The key phrase above is that Valor thinks it’s working; we have no idea whether there is any objectively measured improvement in his motor function. There are well-established rating scales for severity of ALS symptoms, and it would be much more useful to know whether these patients have any measurable improvement in concrete, objective measures of motor neuron and muscle function. I’ve looked, and I haven’t been able to find any. Valor’s impression could be the result of expectation bias, confusing correlation with causation (i.e., waxing and waning of symptoms correlating with drug administration by random chance alone), or other cognitive quirks to which humans are prone that lead to a mistaken impression of causation.

In addition, in The Scientist article, there is the story of Ben Harris, an ALS patient who was enrolled in the phase II clinical trial of NP001 last year:

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.

Here is Harris’ phase II trial report, as posted on PatientsLikeMe, along with others. Note that most of the patient evaluations for NP001 or sodium chlorite can only be viewed if you have a PatientsLikeMe account. However, the two reports that are publicly available list the drug as having “major effectiveness,” and have a lot of subjective accounts of symptomatic improvement with precious few objective measures (one, actually). At one point, Harris reports a large increase in the grip strength of his hands (the only objective measurement), but by the end of his participation in the trial in November 2011 his impression was this:

I just received my very last infusion of NP001. I think overall if I have declined during this study period it has been at a small fraction of the rate before starting the study. The only thing I can say that is worse is that I am more frequently wiping my lips. I think my lip muscles have atrophied but my tongue has not, neither have my left arm or leg. My left arm was getting weaker just before I started the study but it has held strong since. Along with the decrease in strength on my lips has been a very slight decline in my speech. I have been recording my speech weekly since August and it is very difficult to detect a change so the difference is very small. I believe overall NP001 has drastically slowed my progression if it hasn’t stopped it. Overall, I am better than I was when I started the study.

However, elsewhere, he wrote:

The one way I think I may have declined is in drooling. I seem to have to wipe my lips more often than before and I noticed night time drooling for the first time about one month after starting the study. But the difference is very very small compared to what it was like about 9 months ago. I think perhaps my tongue is stronger but my lips are a little weaker. It is difficult to decide whether I am improving or declining, which in itself is a good thing. Before starting the study I would notice differences in my state every month and they were obvious to me. Right now I am struggling to decide whether there are changes in my state from 4 months ago.

Another thing that you should note: There is a 50-50 chance that Mr. Harris received the placebo. He even acknowledges this elsewhere by telling a reporter that he always tells patients to whom he speaks of this possibility. Yet he wrote his PatientsLikeMe diary as though he knew he was receiving the experimental drug. He seemed to assume that he was in the experimental group and, when the NP001 trial was over, decided to do what Eric Valor has been doing and start taking sodium hypochlorite on his own, as described in this Wall Street Journal story back in April:

Although no patient knows whether or not he received the drug or a placebo, Mr. Harris says he experienced dramatic improvement in his ability to swallow after his first infusion of NP001. After he finished with the infusions on the Neuraltus trial, he wanted to continue. He worried that swallowing sodium chlorite wouldn’t be effective because it would be neutralized in the stomach, so in January, he started injecting it every three weeks. He says he feels no side effects. Mr. Harris says he didn’t inform Neuraltus about what he is doing, but feels that it shouldn’t have bearing on the formal trial since he didn’t start infusing himself until after the NP001 infusions were completed.

Mr. Harris isn’t sure he is benefiting but said last week that he gave himself an infusion and by evening, his swallowing had improved. The real proof, he says, will come when the participants do an analysis of the data collected online. Mr. Harris says he knows that the results will never compare to what Neuraltus is doing.

Eventually, the blinding will be lifted, and Mr. Harris will know. However, he could well be mistaken when he says that his use of sodium chlorite after his treatment as part of the trial was done has no bearing on the trial. If there are late measurements of motor neuron function and survival (and I assume there are), his use of what he believes to be NP001 could skew the trial data.

Unfortunately, this PatientsLikeMe “trial” is not a proper clinical trial at all, and it is highly unlikely that the “analysis” of the patient reports and data collected online will show any sort of conclusive evidence of benefit or lack thereof. The reason, of course, is that this is no more than a collection of anecdotes without a control group, without a standardized protocol covering dosage and administration of the drug, and without even a standardized source of the drug. That’s not to say that analyzing the data might not be useful for examining patients’ perceived side effects and for comparing perceived benefit to actual benefit measured objectively, but in reality what PatientsLikeMe is doing is more likely than not pretty close to useless for determining actual efficacy.

A previous PatientsLikeMe ALS “trial”

The Scientist article mentions a previous PatientsLikeMe trial, which, as it turns out, was what brought PatientsLikeMe to my attention in the first place. For example, here is a story from 2008 about ALS patients trying lithium to treat their ALS based on one promising study.

Just as Tassano sparked “wild experimentation” of patients using DCA to treat cancer with little or no medical supervision based on a single study using rat tumor models, patient experimentation with lithium to treat ALS appears to have been sparked by an Italian study that reported a significant slowing of the progression of ALS. This study looked at both a mouse model and reported the results of a small randomized trial of lithium plus riluzole versus riluzole alone. As a result of this study, ALS patients are taking lithium off-label:

Felzer began taking lithium in January, and his scientifically minded family reached out to other ALS patients. “All those people are taking it anyway,” said Alan Felzer, whose smile remains bright and his gaze sharp even as the rest of his body fails him. “So it only made sense to keep track of what was happening.”

The task of leading the ALS-lithium project fell to Felzer’s daughter, Karen, a U.S. Geological Survey earthquake researcher. Her partner in the effort was Humberto Macedo, a 42-year-old computer systems analyst, father of six and ALS patient in Brasilia, Brazil.

The study grew naturally out of the strong reliance of ALS patients on one another for information, Macedo said.

Working online, Karen Felzer and Macedo recruited nearly 200 patients worldwide to take a specific lithium dosage and answer standard surveys to gauge their symptoms. They began running their study through a Web site called PatientsLikeMe.com, using it to attract volunteers and track their progress.

However well-intentioned Humberto Macedo and Karen Felzer, the organizers of this effort, may have been, they clearly didn’t understand why unrandomized, uncontrolled trials, particularly trials with no objective outcome measurements, are so prone to bias and false positives. After all, what they did was nothing more than the collection of anecdotes, and, as I like to say, the plural of “anecdote” is not data. Granted, that’s a bit of an overstatement in some cases, but not by much. The reason is that this survey didn’t even rise to the level of a case series, given that it relied only on Internet reporting rather than the actual direct assessment of the patient’s condition by a health care professional. That didn’t stop PatientsLikeMe from proclaiming:

Together, with all the patients involved, we will run the first real-time, real-world, open and non-blinded, patient-driven trial. We believe we will have the power, within months, to begin answering the question of how much lithium modifies the progression of ALS. Unlike a blind placebo control trial, we are watching the use of this drug in the real world, and because of the number of patients and our system’s sophisticated data modeling, we can determine the significance of each reported change in each patient as he/she deviates from his/her predicted course. There are many risks to our approach, patient optimism, the placebo effect, uncertain quality, and many other variables will compromise our data. Despite these, and many other challenges, we remain committed to solving this problem.

This “problem,” as it was described, probably cannot be solved because it is inherent in the very design of this study, which was neither randomized nor blinded. There were no valid controls, only in essence historical controls (i.e., the “predicted course”). What’s even more puzzling is that the organizers of this trial even seemed to recognize these problems, acknowledging that the placebo effect, patient optimism, and many other variables may compromise the data. Yet they nonetheless expressed optimism that their “sophisticated data modeling” could overcome these problems. How? They never said. They didn’t seem to realize, either, that what they were doing is not new. There’s a reason why randomized, double-blind clinical trials using either placebo or the standard of care as the control have come to be viewed as the strongest form of clinical evidence for the efficacy of a treatment, and it’s because non-blinded and non-randomized trials are prone to all sorts of biases. They just don’t give answers reliably unless the superiority of one therapy is so much greater than that of the control that it’s undeniable and detectable after just a few patients treated.

So what’s the harm? This is, of course, not an unreasonable question for patients faced with a fatal disease like metastatic cancer or ALS. As hard as it is to believe, even for patients with ALS it is possible to make things worse. Lithium, for instance, is not a benign drug. It can cause neurological complications and diabetes insipidus. It also requires monitoring to achieve therapeutic serum lithium levels. In the case of DCA for cancer, the drug could cause neuropathy and other complications. Overall, when taken as a whole, it is far more likely that patients will be harmed by taking experimental or off-label drugs than significantly helped. Worse, in the “real world” case of most drugs, with effects that are not as dramatic as an antibiotic curing a urinary tract infection, it’s difficult, if not impossible, using methodology like that of PatientsLikeMe.com to detect an actual therapeutic effect

As it turns out, lithium doesn’t improve survival or neurologic function in patients with ALS. Ironically, PatientsLikeMe published its observations several months before the official phase IIb clinical trial in a Nature Biotechnology paper and trumpeted on its blog that it had “refuted a published clinical trial,” specifically, the original clinical trial in PNAS that suggested that lithium might be useful in ALS, stating:

PatientsLikeMe developed a novel algorithm designed to match patients who reported taking lithium with a number of other ALS patients that had similar disease courses. By using a matched control group, PatientsLikeMe was able to reduce biases associated with evaluating the effects of treatments in open label, real world situations and improve the statistical power of the study making each patients contribution more meaningful.

Maybe. More likely, it was nothing more than wishful thinking to believe that this novel algorithm, whatever it is, does an adequate job of controlling for the enormous number and quantity of biases that exist in anecdotal reports from patients. Indeed, I can’t help but think that it is a rather apt comparison when AoA equates PatientsLikeMe “to many of the autism biomed boards.” The main difference is that in the case of PatientsLikeMe there is a company asking members of its website to take polls about the effectiveness of whatever they are trying at the moment and then trying to analyze the anecdotes in order to sell the results to its business partners. In fact, when you come right down to it, PatientsLikeMe is a lot like another social networking site, namely Facebook. Like Facebook PatientsLikeMe has members communicating online. Also like Facebook, its business model is to sell the results of its analyses of the data these members provide. Facebook sells to advertisers; PatientsLikeMe sells to its business partners. In all fairness, to be sure, PatientsLikeMe appears to be a lot more transparent than Facebook. It also doesn’t sell its members’ personal information to advertisers and marketers. Even so, its business model is not that different from that of Facebook. Certainly, there is a lot of marketing going on.

Patient autonomy versus clinical trials, PatientsLikeMe-style

ALS is a horrible disease. I know I’ve already said that, but it is something that bears repeating. Consequently, I can’t blame a single patient for wanting to get his hands on NP001, even to the point of being willing to mix up some home-brewed sodium chlorite or buy non-pharmaceutical grade chemical from whatever supplier he can find. I get it. They don’t have the time to wait for drugs to wend their painfully slow way through the FDA approval process any more than patients with advanced cancer do. Like Derek Lowe, I can’t even say that I wouldn’t be sorely tempted to do the same thing myself, particularly with chemicals that are small molecules that can readily be purchased elsewhere outside the context of a clinical trial. I can even somewhat understand the motivation behind PatientsLikeMe. No doubt the founders of the company think they are somehow accelerating drug discovery while doing good. Unfortunately, they have yet to show that they are accelerating anything. If you look at their publications, none of them demonstrate positive efficacy, and most of them (for instance, the latest three) are about patient attitudes and validation of ratings scales. Nowhere is there any example of a study that has shown anything about the efficacy of a new treatment that adds anything to what conventional clinical trials show. For example, in the case of the lithium trial, no one’s going to believe the results of and Internet survey (which is what, in essence, the lithium trial was); they’re going to wait for the results of a real clinical trial.

PatientsLikeMe has always promoted a rather radical idea, namely that it is possible to tell whether a drug works by analyzing, in essence, a bunch of testimonials from a group of self-selected patients self-medicating with therapies in an unblinded fashion with no proper control group. I suppose it’s worth seeing if it is possible to glean any useful information out of the morass of stories that constitute the database utilized by PatientsLikeMe, but I’ve never had much confidence that the company would be successful at it, and I’ve seen no publications yet that suggest to me that my original assessment was wrong. From what I’ve seen thus far, from a scientific and drug development standpoint PatientsLikeMe appears to have failed, even as it has been wildly successful as a social network for patients. Unfortunately, with this most recent foray into reporting on home-brewed NP001-lookalike drugs (a.k.a. sodium chlorite), PatientsLikeMe is entering dangerous territory that it has eschewed in the past. Remember DCA? PatientsLikeMe could have done then for DCA what it’s doing now for sodium chlorite, but at the time it did not—and rightly so, I might add. Indeed, from a strictly analytic standpoint, DCA would have been a much better test case because the DCA patients were using was chemically identical to DCA that showed promising results in the original rodent studies. In contrast, we have no idea whether the sodium chlorite patients reporting to PatientsLikeMe are using is chemically identical to NP001. Very likely it is not.

There’s a reason why AoA loves PatientsLikeMe, and that’s because it cannily sees it as a more sophisticated version of what “autism biomed” proponents have been doing in discussion groups for a long time: Setting up an online place where people trying anything under the sun up to and including the rankest form of quackery can get together and swap anecdotes and testimonials. One wonders how long it will be before members of the “autism biomed” movement starts infiltrating PatientsLikeMe in order to see if the company will examine their assessments of the various interventions to which they subject their autistic children, such as MMS enemas, hyperbaric oxygen chambers, untold varieties of supplements, chelation therapy, and other dubious and sometimes dangerous interventions. Indeed, given that PatientsLikeMe has recently published on its assessment of the efficacy of drugs given for off-label indications, one wonders whether its founders would be willing to examine whether Lupron works for autism. The precedent has been set with sodium chlorite, after all.

There will always be a conflict between wanting to do something now for suffering patients, damn the consequences, and following the scientific method to demonstrate efficacy and safety. It’s perfectly understandable, and the need of dying patients is especially compelling. We have seen both extremes. Indeed, until 1906, drug sellers could make essentially any claim and sell essentially anything to the public as a drug without regulation. We all know how well that worked out. Early in the history of the FDA, companies often tested new drugs by sending them to doctors to offer to their patients, asked for little information regarding side effects and complications, and had no standard criteria for efficacy. Efforts of groups like PatientsLikeMe.com may be an improvement over a century ago in that they do go to great efforts to ask patients about side effects and changes in how they feel, but, boiled down to its essence, this patient-driven trial is the same thing as the unscientific experimentation of that era. It benefits neither science nor patients. Unfortunately, the genie is out of the proverbial bottle; we are not likely to be able to put it back in.

Posted in: Cancer, Clinical Trials, Neuroscience/Mental Health

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24 thoughts on “The perils and pitfalls of “patient-driven” clinical research

  1. BiolArtist says:

    Thank you for this analysis. I suspected as much when my DIYBiology peer group pressured me to join PatientsLikeMe, 23AndMe, and the Quantified Self movement, but I realized that analyzing their claims critically would just make people angry.

    I’m also concerned about data privacy with these companies that collect health and genetic data. Whether they sell data to other companies or have a security failure, I don’t particularly want this information floating around where I don’t have control over it.

    Another concern I have is how these Health 2.0 organizations encourage, directly or indirectly, people making health decisions based on data that is highly preliminary. There have been multiple reports criticizing the genetic testing industry and the interpretation of genetic risk factors… which of course I’m having trouble digging up now, but this is a side issue, so if anyone’s interested I’ll try to find citations.

    Back to the subject of this post: PatientsLikeMe seems to have the potential for abuse by MLM quackery purveyors, similar to the BCO alt-med boards discussed on a thread over at Respectful Insolence. I don’t see anything stopping alt-med purveyors from encouraging customers from signing up with PatientsLikeMe to tout their experiences with placebo effect and regression to the mean. Of course they won’t realize that’s what’s going on, will assume they’re benefited by the quackery, and promote it to a whole new audience. Better yet, this is an audience already tending to believe in brave mavericks fighting the evil or incompetent medical industry.

  2. windriven says:

    “There will always be a conflict between wanting to do something now for suffering patients, damn the consequences, and following the scientific method to demonstrate efficacy and safety.”

    Must the two be mutually exclusive? People who are at the end of the road may try extremely unlikely therapies on the premise that they’re dying anyway. Confusing any anecdotal result with scientific evidence is a logical failure on the part of the confused, not an ethical lapse on the part of the self-experimenter.

  3. eNOS says:

    A relative diagnosed with a particularly aggressive form of ALS has recently discovered a similarly profit-based “treatment” that is backed up by patient testimonials and, coincidentally, not one journal article. (Not even Medical Hypotheses!). Apparently a radiologist by the name of Dr. Jason R. Williams is claiming to restore movement in advanced cases of ALS using stem cells harvested from the individual’s fat, in a procedure he dubs InVivo ReprogrammingTM. Nowhere on his site does he link to a detailed, or even vague, procedure. Some of the press releases mention this all completed within one office visit. If I could guess on the procedure, I would assume mesenchymal stem cells are harvested from adipose tissue, these are isolated from other hemopoaetic cells, the MSCs are reimplanted into the areas of the spine (brain?) to differentiate into motor neurons previously destroyed by the ALS. For the life of me, I can’t understand why this would ever be attempted on humans prior to any animal models (that I can find) and outside the realm of a clinical trial. Does this guy know anything about stem cells? Or their potential for tumor formation? Or cell culturing? How in the world could he claim to even attempt this in one day?

    Despite my attempt at a lengthy email to my family about why this is unscientific, unethical, and potentially dangerous, they’ve chosen to schedule and appointment and make the journey to Gulf Shores, AL, where the the clinic is located. Oh, and the best part; they’ll be ponying up $15,000 for this “revolutionary” treatment.

    I wouldn’t wish ALS on anyone, and I certainly understand they’re grasping at straws at this point. It pains me to see someone so undeserving get taken in by such a person. Images of Stanislaw Burzynski keep cropping up in my head.

    Oh, I suppose someone may want to see the website…
    ww_w.precisionstemcell.com

  4. windriven says:

    It would appear that the good Dr. Williams is quite the entrepreneur. He appears to be the same Jason R Williams, MD who operates the American Cancer Ablation Center as well as LASERlipo/Gulf Coast. I’m not sure what’s next; maybe a chain of lasik centers?

    A search of Pubmed for ‘williams jr’ produced a number of hits but none appeared to be the Williams in question. Most of the citations appear to belong to an Australian JR Williams.

  5. ENV says:

    I find your attempts to discredit me by dissembling most curious for a chap claiming to champion honest science. First of all, the multiple conflations between NP001, my personal project, and MMS are deliberately misleading. The only relationship between the three is that they contain some amount of sodium chlorite. MMS is a rather high concentration that is broken down to chlorine dioxide by the citric acid with which it is mixed. NP001 is a very low concentration of sodium chlorite delivered intravenously. My oral delivery is the same concentration as NP001, with at best about 30% delivery of NaClO2 to the blood after gut passage (according to FDA publications on animal exposure). My project (https://sites.google.com/site/alschlorite/) was intended more as an open-label experiment for people who didn’t qualify for the Phase 2 trial and in the absence of an Expanded Access Program (which some of us are working on for during the Phase 3).

    NP001 and it’s cousin WF10 have excellent safety and tolerability in multiple studies and the case for sodium chlorite having the desired immune modulation is extensively covered in PubMed despite your claims to the contrary (please see http://friends4eric.blogspot.com/2010/08/salty-dog.html for an easy guide through a few of the publications by Dr. Michael McGrath, the inventor). And despite your casting doubt as to the integrity of Neuraltus by pointing at a lack of publication in PubMed, the record on clinicaltrials.gov clearly shows the Neuraltus has satisfied thus far all FDA requirements to move forward in the regulatory process for NP001 and another candidate drug (http://www.clinicaltrials.gov/ct2/results?term=neuraltus). Further, I trust the results published on Patients Like Me done by my friend known online as Persevering (http://friends4eric.blogspot.com/2012/01/guest-persevering-on-np001.html). We have good reason to believe that our correlation between side-effects and live drug is accurate. I won’t reveal that right now because NP001 still has trials to go and I don’t want to pollute the subject pool.

    I didn’t just guess about NP001 and run to buy a bottle of MMS like you insinuate here. I did a careful review of literature by Dr. McGrath, including email with Dr. McGrath himself. I had other sources of information which explicitly stated that sodium chlorite indeed was the active ingredient in NP001. I then worked with a chemist with years of experience using sodium chlorite in the water purification industry to examine feasability and safety. Congratulations on your own discovery, but you could have emailed me for documentation (I am quite easy to Google).

    The lithium project (in which I participated) was not a collection of anecdotes but rather a self-reported trial using the standard measuring tool of the time – the ALS Functional Rating Scale or ALSFSR. This is the same questionnaire used in neurological clinics. Karen Felzer did a fantastic job analyzing the data. Unfortunately the data showed futility, like the official trials that followed ours.

    The lithium project (http://alslithium.atspace.com) was organized because all other ALS organizations expressed no interest in testing the fantastic report making headlines. We patients therefore took it upon ourselves to make an initial, admittedly unofficial, trial to test the report. We knew that the results would not be as reliable as a real clinical trial, but our results would be enough to carry on further to a real clinical trial if the findings in the study paper were real. Shamed by our effort, other organizations hastily constructed trials which proved our findings. Even years later, organizations were burning precious money doing real trials when they could have looked at our results and not wasted precious time which could have been spent on other studies.

    My reports are not a figment of my imagination as you suggest. Go to my site listed above and look at the video evidence I have posted. My doctors have also physically seen the improvements, much to their surprise. Although I don’t make ANY claim of definitive evidence, I do take my effort quite seriously and soberly.

    The real story here, which you ignore or dismiss, is patient empowerment. For too long have we been isolated and told to just wait to die. We can use technology now to organize and, more importantly, mobilize. We now can and will take action to promote OUR agenda, not the feable selfish agenda of certain advocacy organizations which do very little beside provide employment for their directors.

    We are trained in highly-skilled technology trades. We might not be doctors, but we are engineers trained to research and resolve highly complex problems. We are capable of learning and comprehending complicated concepts. We understand the limits of our abilities and, further, that without our efforts the pace of the fight against disease is much too slow for people living today. And we have no delusion that anything we are doing is intended to replace clinical trials. Rather we intend to augment and push forward the actual science.

    I will let Jamie Heywood respond further on behalf of PLM.

    - Eric N. Valor

  6. ENV says:

    I find your attempts to discredit me by dissembling most curious for a chap claiming to champion honest science. First of all, the multiple conflations between NP001, my personal project, and MMS are deliberately misleading. The only relationship between the three is that they contain some amount of sodium chlorite. MMS is a rather high concentration that is broken down to chlorine dioxide by the citric acid with which it is mixed. NP001 is a very low concentration of sodium chlorite delivered intravenously. My oral delivery is the same concentration as NP001, with at best about 30% delivery of NaClO2 to the blood after gut passage (according to FDA publications on animal exposure). My project (https://sites.google.com/site/alschlorite/) was intended more as an open-label experiment for people who didn’t qualify for the Phase 2 trial and in the absence of an Expanded Access Program (which some of us are working on for during the Phase 3).

    NP001 and it’s cousin WF10 have excellent safety and tolerability in multiple studies and the case for sodium chlorite having the desired immune modulation is extensively covered in PubMed despite your claims to the contrary (please see http://friends4eric.blogspot.com/2010/08/salty-dog.html for an easy guide through a few of the publications by Dr. Michael McGrath, the inventor). And despite your casting doubt as to the integrity of Neuraltus by pointing at a lack of publication in PubMed, the record on clinicaltrials.gov clearly shows the Neuraltus has satisfied thus far all FDA requirements to move forward in the regulatory process for NP001 and another candidate drug (http://www.clinicaltrials.gov/ct2/results?term=neuraltus). Further, I trust the results published on Patients Like Me done by my friend known online as Persevering (http://friends4eric.blogspot.com/2012/01/guest-persevering-on-np001.html). We have good reason to believe that our correlation between side-effects and live drug is accurate. I won’t reveal that right now because NP001 still has trials to go and I don’t want to pollute the subject pool.

    I didn’t just guess about NP001 and run to buy a bottle of MMS like you insinuate here. I did a careful review of literature by Dr. McGrath, including email with Dr. McGrath himself. I had other sources of information which explicitly stated that sodium chlorite indeed was the active ingredient in NP001. I then worked with a chemist with years of experience using sodium chlorite in the water purification industry to examine feasability and safety. Congratulations on your own discovery, but you could have emailed me for documentation (I am quite easy to Google).

    The lithium project (in which I participated) was not a collection of anecdotes but rather a self-reported trial using the standard measuring tool of the time – the ALS Functional Rating Scale or ALSFSR. This is the same questionnaire used in neurological clinics. Karen Felzer did a fantastic job analyzing the data. Unfortunately the data showed futility, like the official trials that followed ours.

    The lithium project (http://alslithium.atspace.com) was organized because all other ALS organizations expressed no interest in testing the fantastic report making headlines. We patients therefore took it upon ourselves to make an initial, admittedly unofficial, trial to test the report. We knew that the results would not be as reliable as a real clinical trial, but our results would be enough to carry on further to a real clinical trial if the findings in the study paper were real. Shamed by our effort, other organizations hastily constructed trials which proved our findings. Even years later, organizations were burning precious money doing real trials when they could have looked at our results and not wasted precious time which could have been spent on other studies.

    My reports are not a figment of my imagination as you suggest. Go to my site listed above and look at the video evidence I have posted. My doctors have also physically seen the improvements, much to their surprise. Although I don’t make ANY claim of definitive evidence, I do take my effort quite seriously and soberly.

    The real story here, which you ignore or dismiss, is patient empowerment. For too long have we been isolated and told to just wait to die. We can use technology now to organize and, more importantly, mobilize. We now can and will take action to promote OUR agenda, not the feeble selfish agenda of certain advocacy organizations which do very little beside provide employment for their directors.

    We are trained in highly-skilled technology trades. We might not be doctors, but we are engineers trained to research and resolve highly complex problems. We are capable of learning and comprehending complicated concepts. We understand the limits of our abilities and, further, that without our efforts the pace of the fight against disease is much too slow for people living today. And we have no delusion that anything we are doing is intended to replace clinical trials. Rather we intend to augment and push forward the actual science.

    I will let Jamie Heywood respond further on behalf of PLM.

    - Eric N. Valor

  7. daedalus2u says:

    If patients are giving themselves treatments with low prior plausibility, it is better if the data is recorded so that people can learn from it.

  8. ReallyWOW says:

    This has got to be the most impressive misuse of information that I’ve ever encountered, so kudos on that.

    There is so much wrong here. There is so much here that is so poorly referenced and obviously not properly researched that I wasn’t sure if it was worth anyone’s time to actually respond to.

    You obviously tried very hard. You obviously think you’re very smart. You obviously have not a single clue about what you’re talking about (really).

    I’d suggest that you start by getting to know Mr. Valor or Mr. Harris (or many others referenced here) and go from there. I have more suggestions but based on your obvious lack of knowledge and clear bias in regard to many things…that’d be a waste of time. Good luck to you, and many thanks for trying. Damn…

  9. David Gorski says:

    First of all, the multiple conflations between NP001, my personal project, and MMS are deliberately misleading.

    Actually, MMS is sodium chlorite, as you have admitted. The reason I even mentioned it at all is because (1) it was an antivaccinationist who referenced the article about your project and antivaccinationists have recently been pushing MMS for autism, including enemas; (2) it was rather interesting to me in the wake of my discussion of such quackery that there might be an actual therapeutic use for sodium chlorite for a fatal disease; and (3) an antivaccinationist likened PatientsLikeMe to “autism biomed” discussion boards, and that struck me as an unfortunately somewhat apt comparison. In other words, the MMS was a “hook” into the story that relates it to why antivaccinationists might find it so compelling. Moreover, please read again. I never said (or even insinuated) that you ordered up a bottle of MMS. I simply said you were using sodium chlorite., and sodium chlorite is MMS, and MMS is sodium chlorite, just at different concentrations. We could argue about how you administer it IV versus orally or per rectum and what the effective plasma concentrations of chlorite end up being, but none of that really matters. The drug is the same, and that’s part of what’s interesting about this.

    As for denigrating PatientsLikeMe, I’m not sure that’s the right term. Suffice it to say that I am very skeptical of PatientsLikeMe and highly doubt that what it’s doing will show much value in determining the efficacy of any drug or treatment. In particular, it concerns me that it is a for-profit company that works on a model much like that of Facebook. I also think PatientsLikeMe ignores at its peril the precedent it has just set by getting into the analysis of sodium chlorite administered in various ways as home-brew mixtures or non-pharmaceutical grade chemical sometimes purchased from questionable sources. It’s a slippery slope, as I pointed out. After all, if sodium hypochlorite is OK, and PLM doesn’t have a problem with it, then why not MMS for autism? Or MMS for cancer? Or the various quackery to which autistic children are regularly subjected, such as chelation therapy? Why not use PLM to investigate whether those work? I hope that the powers that be at PLM are sufficiently shocked by my criticism that they don’t do something like this again. They were right not to look at cancer patients using DCA. I don’t think they’re right when it comes to NP001 knockoffs. Off-label drugs and patients on clinical trials of drugs whose companies are seeking FDA approval are one thing. Home brew chemicals based on a drug under clinical trials are another.

  10. ENV says:

    I will indeed grant that MMS is hyped as a panacea. I also found the autism angle quite curious because to my knowledge that is a disorder of ‘miswiring” and not of chronic inflammation which is the aspect of ALS which sodium chlorite addresses.

    Thank you for the clarification.

  11. David Gorski says:

    There is so much wrong here. There is so much here that is so poorly referenced and obviously not properly researched that I wasn’t sure if it was worth anyone’s time to actually respond to.

    One can’t help but note that you responded to it anyway.

  12. daedalus2u says:

    I posted my one line comment above before I saw ENV’s comment, which has clarified my thinking.

    I think that Dr Gorski is caught up in the meme of “perfect being the enemy of good”. Yes, Patients-like-me trials are not the gold standard of prospective double-blind-placebo-controlled trials over multiple centers. That does not mean they are harmful.

    What would “perfect” look like? If there were perfect clinical research in ALS, there would be many high plausibility candidate drugs in multiple trials. There would be more spots for patients in trials than there are patients. The gating factor would be patients, and choosing between multiple high plausibility treatments, not funding or drug candidates. We are a very long way from that.

    Another way to look at this is in the context of harm reduction. Self injecting illegal narcotics imported from Afghanistan with reused needles infected with HIV, hepatitis C and other things is bad. Injecting those same drugs with new and clean and sterile needles is less bad. The current “war on drugs” is causing great harm. In my opinion, the harm is worse than if drugs were legalized. There would be less transmission of disease, the drug lords wouldn’t make so much illegal money, the prisons wouldn’t be full of non-violent drug users learning violence in prison, people wouldn’t need to resort to crime and prostitution to afford drugs. People addicted to drugs wouldn’t be drawn to medical facilities, divert filled syringes and as was recently noticed in Detroit infected 30 in NH and exposing at least hundreds to hepatitis C.

    Yes, having quacks make empty promises and bilking desperate patients out of money with zero benefit and causing actual harm is bad. What if the Gonzales protocol had been tested on something like PLM? There wouldn’t be the enormous confirmation bias that was in all his 20 years of pre-trial results, there would have been a more objective and permanent record, patients would have reported their difficulties taking the hand fulls of supplements and the lack of success would have been apparent. There would never have been a clinical trial. Patients would have abandoned the Gonzales protocol a decade earlier if they had been able to read what actual patients were writing about what they were actually going through.

    The problem with quack treatments is that they do elicit the placebo effect, the patient and quack notice the placebo effect, the quack gets paid and moves on to the next patient. When the placebo effect wears off, the quack has already moved on and the wearing off of the placebo effect is never noticed or reported by the quack.

    Publication bias is an enormous problem in clinical research, one that we are all aware of, but for which there are no good solutions. Journals don’t want to publish negative results, negative results don’t help one advance in one’s career, and the rat-race for funding doesn’t reward negative results, so why publish them, why waste time writing them up? Quacks never report negative results either. Quack reports where the patient is “cured”, but then lost track of are can be counted as a “success” because the patient has been lost track of.

    How many “stem cell” clinics are there? How many treatments have there been with stem cells? How many of those have been analyzed for success and reported accurately? How many of them have “success” analogous to the Gonzales protocol? Probably all of them because if they had real success they would be written up as successful trials.

    There is also the human element, which Dr Moran often brings up. ALS is a fatal, progressive disorder that usually kills people in a couple of years through asphyxiation due to failure of motor neurons. There isn’t a perfect research program in place dealing with ALS. Who is willing to simply tell ALS patients to go home and die quietly? That there is nothing that they can do that helps move ALS treatments forward in the slightest bit? I can’t tell someone there is nothing they can do. What they are doing might not be successful, but that is true of many if not most research trials. If they do something, and it is not successful, but the data is recorded, it does advance the science.

    The ALS patient community in the context of PLM is not behaving the way the anti-vax autism biomed community is behaving. The anti-vax autism biomed community has been holding back and impeding autism research. That is not the case for the ALS patient community.

    The anti-vax autism biomed community does exhibit crank magnetism. As far as I know, the ALS patient community does not. Understanding the differences that lead to those different outcomes is something I think is important to understand.

  13. eNOS says:

    Who is willing to simply tell ALS patients to go home and die quietly? That there is nothing that they can do that helps move ALS treatments forward in the slightest bit? I can’t tell someone there is nothing they can do. What they are doing might not be successful, but that is true of many if not most research trials. If they do something, and it is not successful, but the data is recorded, it does advance the science.

    I think this is a good point to consider, especially considering the progressive, irreversible nature of the disease. I won’t begin to speculate what those with ALS would consider to alleviate some of their symptoms or change the course of their illness, as I’m fortunate enough to only be a spectator. However, financially preying on those same people who are so likely to be open to anything seems egregious, at best. $15,000 (Dr. Williams fee, stated above) for an unproven treatment with nothing but anecdotal evidence as its foundation can put a huge financial strain on families suffering emotionally from watching their loved-ones decline.

  14. David Gorski says:

    Indeed. It is very much like how Dr. Stanislaw Burzynski charges tens or even hundreds of thousands of dollars to administer his “antineoplaston” therapy to stage IV cancer patients. I’ve perused Dr. Williams’ website and done some Google searches. What amazes me is that anyone thinks that what Dr. Williams is harvesting are actually adult stem cells. He has nothing I can find in his background to suggest to me that he has the necessary skills to isolate and purify adult stem cells and then verify what he has. He’s a radiologist, with no evidence of a heavy duty molecular biology or cell biology background. He does, however, have the skills to inject whatever it is he’s isolating very precisely. That’s about all you can say.

  15. daedalus2u says:

    I suspect that quacks such as Dr Williams and Dr Burzynski would very much oppose any of their victims patients using a site like PLM to record their experience so that others can learn from it, because what they would learn is that the treatments don’t work.

    I think when there are web pages set up to collect donations, if the donors made their donations contingent on the victims patients using a site like PLM, the donation sites would go dark because recording and publicizing what happens after and during the treatment is not what the quacks are looking for.

  16. “Mr. Harris says he experienced dramatic improvement in his ability to swallow after his first infusion of NP001.”

    This is a bit of a red flag to the distinct possibility of a placebo response, regardless of whether Mr Harris was in the experimental or control group.

    In order for such a dramatic, immediate response to be plausible, it must be supported by the supposed mechanism of action.

    Not being a MD or medical researcher, I could be wrong, but I would think that even if NP001 immediately eliminated the inflammation and suppressed the overactive immune function, it would also have to regenerate the motor neurons and revitalize muscles in order to be able to produce such an immediate improvement rather than just a cessation or reduction of degeneration and decline.

    I’m not aware of any scientific claims that NP001 has any regenerative properties in this regard. One would assume it would take some time for the motor neurons & muscles to regenerate after the overactive immune function is corrected..

    Also as an amusing side note, looking at the article on the-scientist.com, I am reminded of the TAM 2012 presentation that advised to always include an image of a brain in any nuero-science story to make it more credible.

    Brain Image In Story? Check

  17. daedalus2u says:

    Karl, a reduction in inflammation can cause prompt improvement in neurologically mediated symptoms. There are reports of prompt (minutes) improvement in Alzheimer’s when Etanercept (a TNF-alpha blocker) is injected into the CSF.

    In the Rett Syndrome mouse (targeted and switchable turn-off of the MeCP2 gene), the characteristic symptoms of RS go away when the MeCP2 gene is turned back on. That tells us that the neurological problems are not due to adverse neuronal wiring (or they wouldn’t resolve so quickly).

    In children with autism, there are reports of prompt and temporary resolution of autism symptoms during acute fever. The resolution of these symptoms can’t be due to regeneration because there isn’t time.

    In my blog post on fever therapy I discuss some of the physiology of how prompt neurological symptom resolution was observed in Fever Therapy for a variety of neuroinflammatory disorders and it was the “standard of care” treatment for neurosyphilis for a couple of decades.

    http://daedalus2u.blogspot.com/2008/01/resolution-of-asd-symptoms-with-fever.html

    I think that sodium chlorite is not a good way to deal with inflammation, but if it was resolving inflammation, it could have prompt effects. But what is more important are the long term effects and they are much more complicated. Like all aspects of physiology, inflammation is regulated and controlled. Any long term inflammation can only be mediated through disruption of the long term control of inflammation. This is a tautology. If long term regulation of inflammation was not disrupted there could be no long term inflammation. Either what controls long term inflammation has allowed long term inflammation to happen, or it has not resolved the long term inflammation.

  18. daedalus2u,

    “Karl, a reduction in inflammation can cause prompt improvement in neurologically mediated symptoms.”

    Yes, but the relevant question here is, could that apply to ALS mediated symptoms?

    My admittedly limited understanding of ALS is that there is degradation of the upper and lower motor neurons and a resulting atrophy of the muscles, and the symptoms are not directly caused by inflammation or the immune system activity.

    If this is the case, then I would think that a reduction/elimination of inflammation would not be expected to result in prompt and dramatic improvement in ALS symptoms like the ability to swallow since the elimination of inflammation would not reverse that atrophy of the muscles.

  19. daedalus2u says:

    There is inflammation of nerves and there is also degradation of nerves. Inflammation usually precedes degradation and if the inflammation goes on long enough there will be degradation. Functional degradation occurs before actual degradation. Atrophy of muscles occurs last.

    Inflammation all by itself can cause paralysis, but the paralysis from inflammation is often temporary as in Bell’s palsy.

    https://en.wikipedia.org/wiki/Bell%27s_palsy

    Bell’s palsy is paralysis due to inflammation of the involved nerves. I presume that ALS has a similar course, in that it starts out with inflammation and progresses to paralysis and ultimately to degradation. Paralysis very likely precedes degradation but paralysis prior to degradation would be reversible. Paralysis following degradation would not be promptly reversible. The time scale of paralysis reversal could be variable, fast (minutes) or slow (months). How a particular patient is going to respond to a particular incidence of a resolution of inflammation is going to be dependent on the particulars of that patient and would very likely not be predictable in advance.

    If there is any reversal of paralysis, then there was not yet degradation sufficient to cause irreversible paralysis. It doesn’t matter what caused the reversal of paralysis, even if it is placebo, if there is a reversal, then there was not permanent paralysis.

    According to my conceptualization of physiology, the placebo effect is mediated through nitric oxide, and it is nitric oxide that is the primary anti-inflammatory agent, so the effect could be real and it could still be mediated through the placebo effect. If swallowing is still possible, then there is still some nerve activity and the muscles have not fully atrophied. Resolution of inflammation would improve nerve activity much faster than it could improve muscle atrophy.

    I don’t think that swallowing muscles atrophy from non-use the way that skeletal muscles do. Much of swallowing is reflex, so the neurological control of swallowing is more complicated than that of skeletal muscles.

    Maybe someone who knows more than I do about the measuring of swallowing and how it is quantified
    can comment on whether or not there are instrumental measures of swallowing that could be used to quantify any changes? I suspect that there are not because swallowing is difficult to measure externally and any kind of external test would risk choking which would be bad. If the only measures are subjective, then that this test was not done in an “official clinical trial”, doesn’t change its reliability of the measurements much.

  20. Reductionist Nurse says:

    I stumbled on this in 2010: breastcancernaturalcure.blogspot.com

    It was a blog started by a woman who was diagnosed in the early stages of breast cancer. She ignored the advice of a surgeon to operate immediately after being told by their naturopath that she could cure her breast cancer by eating all natural foods. She started this blog after the breast cancer literally began to break through her skin, which her naturopath stated was an expected side effect of her healthy diet, because it was “driving the cancer out” of her body.

    As expected, the cancer eventually metastasized and she passed away about a year after experimenting how to cure breast cancer naturally. This is the most likely end product of patient driven research. There is a point when you simply cannot protect people from themselves, but as any regular reader here can see, we all have a responsibility to recognize the established innate danger of testimonials and confirmation bias. You can’t do the science without science.

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