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The latest chapter in the seemingly never-ending saga of dichloroacetate as a cancer treatment

The road from an idea to a useful drug is a long one, and in cancer it is often particularly long. One reason is that to be able to tell whether a given treatment is effective against cancer often takes several years at a minimum, in order to determine if patients receiving the new treatment are surviving their disease longer than those who are not. Surrogate endpoints are usually not enough. Tumor shrinkage in response to a drug often does not correlate with prolongation of survival, although the converse (i.e., lack of tumor shrinkage in response to a new drug) does strongly correlate with failure of a treatment to prolong survival. In other words, effects observed on surrogate endpoints are not enough to judge whether a cancer therapy is working or not.

Three years ago, predating the existence of this blog by nearly a year, I became aware of a story that involved many of the issues in bringing a compound from the laboratory to the clinic. The case was unusual in that is is very rare to see the scientific process by which new drugs progress through the stages of cancer research, from concept to testing in cell culture to testing in animals to testing in humans challenged so strongly by patients themselves. The reason that this normally doesn’t occur is that new cancer treatments are almost always the product of either university-conducted research, pharmaceutical company-conducted research, or partnerships between the two. This case was markedly different in that it involved a chemical that was not only easy to synthesize, but cheap and long out of patent. Even more intriguing, it targeted a metabolic abnormality found in many cancer cells, an abnormality first described nearly 80 years before by Otto Warburg in 1928. This latter aspect of the drug gave it every appearance of a “rediscovery” of old wisdom that big pharma had ignored for 80 years, and that only added to its mystique.

The chemical was dichloroacetate (DCA), and three years ago it created a world-wide sensation. Last week, it created a sensation again, as breathless news reports once again overhyped its promise. Since I’ve been following the story since early 2007, I appear to be in as good a position as anyone to tell the story thus far and put the new findings into context. To begin that process, let’s head back to January 2007.

“The cure for cancer big pharma doesn’t want you to know about”

The DCA saga began in January 2007, when I started noticing a bunch of posts by various bloggers as well as news stories that all had similar titles, such as Cheap, safe drug kills most cancers, Objectively pro-cancer, Gotta pay, When promising cures are ignored, and, my personal favorite, Potential cheap, safe cure for cancer: Will Big Pharma Allow It? These stories described some intriguing work by University of Alberta researcher Evangelos Michelakis regarding this old/new drug that showed great promise in cell culture and rodent models of cancer (more on that shortly) that was published in Cancer Cell. Typical quotes I saved at the time:

  • “Big Pharma won’t put up the dough to fund human research and enable this drug to come to market, there’s no money in it. In fact, it wouldn’t surprise me to discover that they had an interest in actively preventing the research so as to maintain demand for more expensive less effective drugs. This drug looks to be extremely promising, and I can’t imagine that it won’t get government funding for human trials, but that said, it doesn’t pay to underestimate the power of Big Pharma…” (unfortunately the original link is gone, but the text lives on)
  • “And here I thought the pharmaceutical companies had to charge such high prices because of all the research they were doing. Seems without the possibility of future revenue they can’t be bothered. Of course, a cheap cure for cancer would cut into profits in so many ways, wouldn’t it?” (From Digby, who really, really should have known better, but didn’t.)
  • “It seems to good to be true. A cheap, effective cancer cure that BigPharma doesn’t own. If further research proves effective in humans, it could be the answer to many peoples prayers. I’ve always thought something simple, rather than the current convoluted regimen of surgery, radiation and chemicals would be the cure for cancer. Again, if proven effective, will we ever see it in use in this country? Will patients have to take ‘DCA tours’ to Canada for treatment?” (Source: Randular’s diary.)
  • “Here’s the big catch. Pharmaceutical companies probably won’t invest in research into DCA because they won’t profit from it. It’s easy to make, unpatented and could be added to drinking water. Imagine, Gatorade with cancer control.” (Source; this particular editorial was truly overwrought.)
  • “My bet is that the dichloroacetate news will fade into the background very quickly. Because neither the US government, nor any major university, nor any private company (pharma or otherwise) will have the least bit of interest in funding further studies. There will be a small handful of tiny, underfunded studies, and that will be that. Indeed, already those with vested financial and personal prestige issues will be doing their best to put the brakes on anyone who takes this development seriously. Not because the megacomplex of big government and big business wants to kill people–it doesn’t, there are few if any mustache-twirling villains–but because of the age old factors of bureaucracy, personal pettiness, ego, and conflicts of interest.” (Source: Dean Esmay, scroll down for post.)

Note that there were two assumptions here three years ago. First, these bloggers and pundits assumed that the cell culture and animal work were definitive evidence that DCA might be a “cure” for cancer. Second, the assumption was that, because the drug was out of patent and very cheap to make, neither the government nor pharmaceutical companies would be interested in funding it, thus condemning thousands, maybe millions, of people to die of cancer unnecessarily. Unfortunately, the New Scientist article and articles in the Edmonton Sun featured headlines to that effect and quotes by the investigator Evangelos Michelakis lamenting how he had had difficulties finding funding to do the next step, clinical trials in cancer. As a result of these sensationalistic stories, unscrupulous “businessmen” sought to bring DCA to the masses.

DCA and the Warburg effect

DCA is a very simple molecule, deceptively simple. Basically, it is an analog of acetic acid in which two of the three hydrogen atoms of the methyl group have been replaced by chlorine atoms. Interestingly, it is only one chlorine atom off from trichloroacetic acid (TCA), a chemical we routinely use in the laboratory to precipitate nucleic acids and proteins from solution. The structure of DCA is depicted below.

DCA

DCA has been around for a long time (which is why is it no longer under patent) and has primarily been used for inherited diseases of mitochondrial metabolism. Mitochondria are often (and correctly) referred to as the “powerhouses” or the “batteries” of the cell, because it is in the mitochondria that the main energy-containing molecule, ATP, is produced using byproducts of glycolysis and the Krebs citric acid cycle to generate a proton gradient across the mitochondrial membrane, which supplies the energy for the enzyme ATP synthase, which, true to its name, synthesizes ATP for use in the cell as a chemical source of energy. The key thing to remember about oxidative phophorylation is that it requires oxygen, whereas glycolysis does not. When there is insufficient oxygen, the end products of glycolysis end up being turned into lactic acid, which is one of the things that make muscles feel tired after a rapid workout that exceeds the capacity of the body to deliver oxygen to the tissues. The primary activity of DCA in cells is to inhibit the enzyme pyruvate dehydrogenase kinase. The result, to boil it down (and not to have to stress my knowledge of the basic biochemistry of glycolosis, the Krebs cycle, and oxidative phosphorylation too much) is to shift the metabolism of pyruvate from glycolysis towards oxidation in the mitochondria. To boil it down even further, DCA shifts the cell’s metabolism from anaerobic to aerobic metabolism.

The three components of glucose metabolism are shown below in simplified illustrations:

glycolysis
krebscycle
oxidativephos

Why, then, would such an activity be useful as an anticancer therapy?

It all boils down to something known as the Warburg effect, which Otto Warburg first described way back in 1928 and reported in Science back in 1956. Over the last five years or so, cancer researchers have been increasingly coming to appreciate the role of abnormalities in metabolism, in particular the mitochondria, in cancer. To put it briefly, many cancers (approximately 60-90%) favor glycolysis, even in the presence of adequate oxygen for oxidative phosphorylation, leading to a voracious appetite for glucose. Indeed, it is this very avidity of cancer cells for glucose that is the basis of the PET scan, which detects the high uptake of a radiolabeled form of glucose by cancer cells relative to the surrounding normal cells.

Over the last few years, there has been a sort of “chicken or the egg” argument about what is more important and what is the first abnormality leading to cancer. The traditional view has long been that mutations in DNA lead to the activation of protooncogenes into cancer-initiating and causing oncogenes and to the shutdown of tumor suppressor genes. Under this model, mutations leading to cancer also lead to the observations of abnormalities in metabolism. In the wake of the DCA furor, there have been data reported suggesting that the metabolic derangements may actually occur first or simultaneously with the mutations. p53, for instance, the granddaddy of tumor suppressor genes, can trigger the Warburg effect when mutated. Whatever the case, it is now fairly clear that abnormalities in cancer cell metabolism are very important in driving cancer growth and could well represent targets for cancer therapy. AS a result of these new data, studying the metabolism of cancer cells has become a much hotter topic of research than it has been in the past. Everything old is new again, it seems. Why cancer cells might have an advantage due to the Warburg effect is a matter of debate, although, given how tumors frequently outgrow their blood supply, being able to maintain themselves in low oxygen situations would be advantageous.

This fascinating basic science met the public in January 2007, when Michelakis and his colleagues at the University of Alberta in Edmonton published a seminal paper in Cancer Cell. In the study, DCA was tested in multiple cell culture and rodent models of cancer. In rats, tumor weights in animals treated with DCA were approximately 60% lower than the tumors in the untreated control groups. The drug increased apoptosis, decreased proliferation, and inhibits tumor growth by acting on a critical enzyme that controls the switch between aerobic and anaerobic metabolism without harming non-cancerous cells. Even better, DCA had already been FDA-approved for mitochondrial disorders, meaning that using it in humans would be an “off-label” use of an already existing drug to test it in humans. Thus, the regulatory requirements were considerably easier to meet for early drug trials in cancer.

The “Pet DCA” phenomenon

The media coverage of the Michelakis study unleashed the proverbial perfect storm of conspiracy mongering about big pharma. Never before (at least that I can recall) had a chemical that was so cheap and so easy to synthesize, not to mention already used in patients for another indication, been reported as being a “cure” for cancer. This hype, of course, neglected what those of us who do cancer research as part of our living, namely that many are the candidate drugs that show a great deal of promise in animals but fail in humans and that at present there is no such thing as a “cure for cancer.” I like to think of the example of angiogenesis inhibitors, which I’ve been studying for 13 years now. Part of what attracted me to the study of these compounds were the amazing results of the late Dr. Judah Folkman in mice, which I’ve described on this blog before. However, the amazing results in mice did not translate to humans. That’s not to say that angiogenesis inhibitors don’t work, but in humans they did not produce anywhere near as dramatic results as they did in Dr. Folkman’s mouse experiments. That’s why, rather than being a “magic bullet” for cancer, angiogenesis inhibitors entered our armamentarium of anti-cancer drugs as a useful, but not miraculous, new addition. Avastin, for instance, has increased the five year survival rates in colorectal cancer metatastic to the liver and several other cancers, when added to the current standard of care, but it has not resulted in the cure of any metastatic cancer.

That being said, I can understand how cancer patients incurable with standard therapy would leap at this drug as their last hope. Unfortunately, there were unethical “entrepreneurs” who were more than eager to supply DCA to them. The most famous of these who appeared in the months following the publicity surrounding Michelakis’ study was a pesticide dealer named Jim Tassano, who leapt to create a website known as TheDCASite.com and BuyDCA.com (the latter of which appears to be unreachable at the moment). His way of getting around the FDA? To market his DCA as “Pet DCA,” to be sold for pets dying of cancer. It was as disingenuous an approach as could be imagined, because not only did Tassano openly admit that he knew that people were buying the drug for themselves but he blithely dismissed the possibility of serious side effects in adults. Although children could take the drug with few side effects, adults who took the drug often developed serious severe peripheral neuropathy. Since peripheral neuropathy is a side effect of some commonly used cancer chemotherapeutics, taking DCA with these cancer chemotherapies has the potential to do serious harm. Moreover, when asked about the source of DCA, the webmaster Heather Nordstrom replied:

In my opinion, it is not that difficult to get, but that may be because my step father has connections with manufacturers since we invent and sell tools for our family business. We know of a chemical company in China that makes DCA. It is pharmaceutical grade. More information on the quality and source will be posted on the website that sells it.

Naturally, I was…skeptical. Worse, scammers were hyping DCA as not being “chemotherapy,” when, if it works for cancer, that’s exactly what it is.

Meanwhile, cancer patients and their families flooded the discussion boards of TheDCASite.com to write about their self-experimentation. Here are a couple of posts that I saved (the DCASite.com purged many of its forums when it started to get into legal trouble):

My husband has been using DCA since early February. He has Glioblastoma, an aggressive brain cancer that DCA is proposed to target. The naDCA he is using was made in a private lab. We turned away from our medical community, realizing that we would not receive blessings from them, since they considered him a “dead man walking”. From what I have read here, my husband seems to be the earliest “labrat”. We obtained the DCA in early February, started at a 5% dose,(to test toxicity or side effects, I suppose) and after 4 days , he insisted on taking 25mg per kg. He takes a liquid dose twice a day( totaling 25mg per kg). He has been taking DCA since Feb 7, 2007, with full dose as of Feb 11. No side effects to report as yet. Though side effects of DCA(numbness in fingers and appendages) are also symptoms of his disease, there are none to report at this time. He is also taking 100mg per day of thiamine. He is also on CCNU, Heparin, and 16 mg steroid. So far, so good. I am taking weekly urine samples to check his billyrubin, ph , etc. He still sees an oncologist, and takes chemo( CCNU). His doctors do not know that he is taking DCA. I do not trust them, they have not been terribly compassionate through this, and I do not feel that they would be as knowledgable as those of us that are in these desparate situations. I pray for all of us that I can report great news in the weeks to come. I still can’t decide at this point if he should take THiamine or not? Any thoughts?

My thought at the time was that this woman’s husband was endangering his life by taking DCA along with his chemotherapy and not telling his doctors about it. Meanwhile, as time went on, someone on the DCASite actually questioned the anecdotes in May 2007:

Maybe I am crazy but it seems like every time I ask this question the admin removes it – if not then where is the question I asked last time?

I am asking this Question -

Do we want to know or not? is this a site for lies or truth? I have DCA and I also sent money in for the clinical trials as well as having my wife dealing with stage four lung cancer currently stable with antioxident regimens. Today we go in with DCA to see if her oncologist will support her using it.

Some news here would sure be great but NO ONE who has been CT scanned after using DCA for a couple months is talking or coming back except for squareb who gave us bad news. What is going on here? are we participating in a venting session where these folks are linked with those folks selling us DCA? what exactly is going on – our lives and our loved ones lives are on the line and not ONE CT SCAN in 4 months!!!! or the TRUTH is being removed from the site and people who had scans did post bad results so it was also removed like my questions are.

I am not happy about this admin – gee sorry I lost everything sorry – what? didn’t this happen last month on another persons testimony who DIED!! while using DCA. Something is definitely missing here and I am starting to think its THE TRUTH.

I believe in DCA but I am starting to think these admins are manipulating the information to suit them which is pretty typical for us trying to survive to run into. Sorry but these admins need to either post it ALL or explain why they are not allowing CT scan results to be listed on this site. Folks just think about it – Jan, Feb, March, April and now May – 4 months and nothing – come on folks lets get real here – do you really think its a coincidence?.

It is too bad that more people didn’t ask questions like this. squareb, by the way, was an early adopter of DCA whose tumor progressed on the drug.

Unfortunately, Jim Tassano wasn’t alone. For example, a family physician in Toronto named Dr. Humaira Khan, even though agreeing with the warnings not to use DCA outside the auspices of a clinical trial, decided to start selling DCA for $150 a week, using arguments of “health freedom” and wanting to “supervise” patients who would take the drug anyway. Dr. Khan’s webpage on DCA appears not to have been updated since 2008 but is claiming a high response rate. Odd, though, I haven’t seen this published anywhere in the peer-reviewed scientific literature, and a PubMed search turns up…nothing, at least nothing by Dr. Khan or anyone associated with her. Jim Tassano himself also tried to do a “clinical trial” of DCA that was basically nothing more than a questionnaire that–I kid you not–included questions like:

  • Do you think DCA is working for you? Why or why not? Scans? Examinations? Blood level testing? Here, I encourage you to freeform answer.
  • How do you rate your health compared to when you started DCA? 1. Much better; 2. Somewhat better; 3. About the same; 4. Somewhat worse; 5. Much worse

I trust that SBM readers will immediately recognize why such questions are worse than useless. Basically, they are doing nothing more than soliciting testimonials, and testimonials in cancer are often highly misleading, and that’s all TheDCASite has now. This is just as true for something like DCA as it is for any alternative medicine cancer therapies. Fortunately, in July 2007 the FDA finally acted to shut down TheDCASite.com, at least as far as selling DCA went. In response, Tassano shut down BuyDCA.com and morphed TheDCASite.com into an “informational” website. Much of what was on the forums there disappeared. Meanwhile, conspiracy theorists had a field day denouncing the FDA’s action.

Back to the future

Over the last couple of years, my biggest fear was that the activities of “entrepreneurs” like Jim Tassano would taint what is a scientifically fascinating and potentially very useful new cancer therapy with the indelible stain of quackery. DCA is most definitely not quackery. It is, however, unproven in humans and thus may not be effective, which is why self-treatment and treatment by doctors who have no clue what they are doing are not advisable. Fortunately, while ignorant businessmen like Tassano and opportunistic doctors were selling DCA to desperate cancer patients, a clinical trial was beginning by Michelakis and his team, and last week the results were reported in Science Translational Medicine entitled Metabolic Modulation of Glioblastoma with Dichloroacetate.

The first part of the study was something quite fascinating that I don’t recall ever having seen before in a clinical trial. Michelakis and colleagues studied 49 consecutive surgically excised glioblastomas. Glioblastoma is an aggressive form of brain cancer known to exhibit the Warburg effect and thus a good candidate for the first attempts at testing DCA in humans. These tumors were then tested in vitro with DCA. The excised tumors did demonstrate evidence of the Warburg effect, and treatment with DCA resulted in significant reversal of some of these features in the excised tumors, particularly mitochondrial hyperpolarization, while not having any effect on normal tissues excised with the cancers. The second part of the study is what most media and blog reports focused on. Michelakis treated five patients with neuroblastoma with oral DCA. However–and this needs to be emphasized–patients were also treated with temozolomide and radiation, with DCA added to the mix. The DCA dose started at 12.5 mg/kg orally twice a day for 1 month, and then the dose was increased to 25mg/kg orally twice a day. Michelakis then followed a dose de-escalation protocol, decreasing the dose by 50% when dose-limiting toxicity occurred. The patients were followed clinically for up to 15 months. In other words, this appeared to be a combined phase 0/phase I clinical trial.

For those not familiar with the various types of clinical trials, phase I clinical trials are not trials of efficacy. They are designed to determine two things: dose and dose-limiting side effects. They generally use a few patients (although five patients represent a rather small number, even for a phase I trial, which usually requires around 10 or 20), and it is not uncommon to perform a dose escalation. Researchers don’t expect necessarily to see tumor response in a phase I trial, as that is not the purpose of the trial, but it is heartening when tumor shrinkage is observed, for obvious reasons. Phase 0 trials similarly are not therapeutic trials but rather seek to determine if the drug is doing biochemically what it is expected to do based on preclinical studies. The usual design is to take a biopsy of the tumor, test it for biochemical markers in the laboratory, treat the patient with experimental drug, and then resect the tumor. The biochemical markers in the resected tumor are then compared with those measured in the pre-treatment biopsy. The idea is to see whether the drug can recapitulate biochemical changes in actual living tumors in human patients, the idea being that, if it can, then the drug is “hitting the target” (i.e., its molecular target) and therefore “working.” Whether its “working” actually shrinks tumors or results in prolonged patient survival is then the next question that has to be tested.

Michelakis went one further in that he isolated tumor cells from the pretreatment biopsies and produced glioblastoma cell lines. He could do this in three of the patients because he had tissue from their first debulking surgery, and these patients had recurrent glioblastoma that had failed additional chemotherapy (patients 1 through 3). All of them had had multiple rounds of different chemotherapy. Patients #1 and #2 showed signs of improvement or were at least stable for the full 15 months of the study, while patient #3 died three months after starting DCA therapy. Two other patients (patients #4 and #5) had new diagnoses, allowing initial tumor tissue from debulking surgery to be examined. Their treatment was somewhat different. After his first surgery, patient #4 underwent DCA therapy for three months followed by DCA and standard therapy. However, by the end of the third month, the patient showed evidence of progression requiring a second operation. Patient #5 underwent surgery followed by DCA and radiation with temozolomide for six months and then stayed on DCA for nine more months and is doing well.

The reason I mention this is because not only were there only five patients, but they were not all even treated the same way. They were treated with varying regimens of surgery, with drug therapies combining DCA and chemotherapy (mostly temozolomide), some with and some without radiation. Even so, the tissue from these tumors showed signs of metabolic reversion to an oxidative phosphorylation phenotype. The problem is, as both our very own David Kroll pointed out, we don’t know for sure if the DCA was responsible for this effect. As a cancer researcher, I can’t say whether the regressions observed were due to DCA, although the regression of paraventricular masses in patient #1 and the regression observed in patient #5 are certainly fairly suggestive (at least to me) of an anti-tumor effect due to DCA alone. The only side effect Michelakis reported was a reversible change in peripheral nerve function.

So what does this all mean?

I’m frequently asked why we shouldn’t just use DCA now–or even let people use it the way that they were using Jim Tassano’s homemade DCA? What’s the harm? That’s a rather difficult question, because there is always a conflict between wanting to do something now for suffering patients, damn the consequences, and following the scientific method to demonstrate efficacy and safety. Our nation has been at both extremes. Indeed, until 1906, pharmaceutical companies could make essentially any claims 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, as Dr Jerome Groopman points out, 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. There was a reason we moved away from such a system.

I think Dr. J. Leonard Lichtenfeld, Deputy Chief Medical Officer for the national office of the American Cancer Society, put it well in writing about this latest DCA study on his blog, Dr. Len’s Cancer Blog:

This research still needs lots of work before we know whether it works or doesn’t work, and whether it is really safe or not when given to patients with cancer under a variety of circumstances.

If that sounds overly cautious, so be it. I have seen too many dashed hopes in my medical career which make me a bit cautious about reports like this. That’s not to say I don’t think it could work—it could, as I mentioned above—but I want to see evidence in well done trials that prove the point that DCA is effective in the treatment of which cancers under what circumstances.

Early in my cancer training there was a substance isolated by a researcher that was supposedly non-toxic and would cure leukemia. The research center where I was working was inundated from people around the globe who wanted this treatment, especially after the lead researcher injected himself on a nationwide morning show to demonstrate its apparent lack of toxicity.

Only grams of this medicine existed. Fortunes were offered in return for getting this miracle drug.

But the miracle drug—after reasonable clinical trials were done—didn’t work after all.

Many are the lists of new “miracle cures” that have met this same fate. The difference today is that the Internet has allowed news of these drugs to be disseminated to more people than ever before–and faster than every before. Moreover, it has linked patients and activists into mutually supportive disease-specific communities, who can inform and educate each other, as well as publicizing research about their disease and lobbying legislators. The dark side of this power, however, is that it can facilitate the spread of false hope and the demand for a drug after only cell culture and animal work, before it even makes it to human trials. Add unscrupulous “entrepreneurs” into the mix, and the potential for harm is great.

One has to remember that cancer is not just one disease. Not only that, but even a single type cancer is often not just one disease. As I have written extensively about before, cancer is incredibly complex. Because of that complexity, it’s incredibly unlikely that any one drug will be any sort of “magic bullet” to cure cancer. Worse, simply using a drug like DCA outside the auspices of well-designed clinical trials will virtually guarantee that we will never know for sure whether the drug actually works. Because of that, as frustrating as it is, as slow as it is, letting science take its course to determine if DCA works, how it works, and for what cancers it works, is the best method to make sure that the most patients are helped and the fewest are harmed. I don’t say this because I want DCA to fail; I say it because I would very much like to see DCA succeed.

Other good posts about DCA:

Posted in: Cancer, Clinical Trials, Health Fraud

Leave a Comment (48) ↓

48 thoughts on “The latest chapter in the seemingly never-ending saga of dichloroacetate as a cancer treatment

  1. romanmd says:

    It’s always sad to see patients and their families so invested and hopeful in unproven therapies. Hopefully DCA will prove to be a useful adjunct to current chemo regimens but until it’s shown to work the jury is out.
    No matter how often I read about people like Tassano and Khan, my anger never subsides. Shame on them.

  2. BillyJoe says:

    This article is a good follow up to Steven Novella’s article “Low Dose Naltrexone – Bogus or Cutting Edge Science?”

  3. Angora Rabbit says:

    I hadn’t heard this story. The loud sound you just heard was my jaw hitting the desk. DCA is one of the metabolic endproducts of Trichloroethylene (TCE). TCE is an industrial degreaser used in manufacturing, dry cleaning, and computer chip manufacturing. TCE is one of the top 10 groundwater contaminants in Superfund sites. TCE is a suspect carcinogen, a topic nicely reviewed in the NRC’s recent panel and published by the Natl Academy Press. EPA is currently preparing new guidelines to regulate its water content. It is also a suspect teratogen, a topic that several labs including my own have studied for some years and find evidence in support.

    Studies indicate that the end toxicant is not TCE itself but its oxidized metabolites TCA and DCA. And these people want to willingly take this stuff???? The stupidity and gullibility of the Public is limitless. On the one hand they are screeching about TCE and its metabolites in the drinking water as a carcinogen, and on the other they want to ingest it as a putative chemotherapeutic.

    I’m reminded of the old Saturday Night Live sketch about the product that’s both a floor cleaner and a dessert topping. Except in this context, the joke isn’t funny.

  4. Ash says:

    An informative read. I blogged about this last week from the perspective of how should research into drugs like this be funded, but I’m not sure I really have an answer – I can’t see a for-profit company investing that much money in a non-patentable drug (though the researchers are trying to patent its use as a cancer treatment). Whether this drug was likely to be an effective treatment was way outside my area of expertise though, so it’s nice to see a cancer expert confirming my initial gut feeling (some promise, but way too early to say anything definitive). Hope you don’t mind if I update my post to link here.

  5. KarlS says:

    Amen, David. Well worth the read – and about much more than DCA

    Karl (Patients Against Lymphoma)

  6. BobbyG says:

    Interesting. Love this blog.

    Different day, same [bleep].

    A dozen years ago my daughter was nearing the end of a horrific 2+ year struggle with stage 4b hepatoma in L.A.

    see http://www.bgladd.com/1in3

    I am neither a clinician nor a “scientist,” but I did cut my professional teeth across a 5 year period in a forensic level environmental radiation/mixed waste lab in Oak Ridge in the mid-late 1980′s as a programmer and QC analyst, so I know just a tad about the scientific method.

    After Sissy died, I didn’t know WHAT I believed any more.

  7. superdave says:

    I am sure some pharma company could combine this with other drugs and patent that combo if they really thought it was worth it., so that argument doesn’t hold.

  8. bwmbagus says:

    excellent article, there is a lot oif overhyped stuff around when in fact the effects are minimal.
    How about an artivcle on the effects of met-5-enkephalin, which binds to the mu opiate receptor and inhibits tumour growth, subsequently allowing lower doses of chem to kill the tumour?
    Now, a positive article would go well with all these negative ones. Sometimes, but rarely, new approaches do show more than just a small effect.

  9. Maz says:

    Superdave, I was thinking the same thing. Someone correct us if we’re wrong, but can’t drug companies repackage old drugs with a new capsule or time-release strategy and re-patent them?

    Obviously, those in the know might just buy DCA, but I’m sure most people would just go with the nice insurance-approved drug. If this drug was a magical cure, the drug companies WOULD be all over it.

    As it stands, I’m sure that (if it does have clinical effects) it will become another drug that is useful against some types of cancers.

    Seems like the “magic bullet” is just going to be a process of studying thousands of types of cancers and looking for markers that can be used to identify the most appropriate interventions for each.

  10. Ash says:

    Superdave/Maz – that’s a good point. I’m not directly involved in drug approval myself – could a company conduct all the safety/efficacy testing on DCA + some other chemical without doing testing on DCA alone? Ethically it seems a bit shady to market DCA + another chemical without demonstrating that the combination would be better than DCA alone; essentially the only purpose of the other chemical would be to inflate the cost (and would certainly give the anti-pharmaceutical industry groups some fodder). If you could find another (patentable) chemical that, in combination with DCA, would be even better, then there would definitely be some drug company interest. (All of this, of course, is assuming DCA really would be effective in treating at least some cancers)

    If DCA does turn out to be a worthwhile treatment, I’m sure the drug companies would be happy to sell it; they just wouldn’t be happy to spend the money necessary to get FDA approval (which I’ve heard is getting to be close to 1 billion dollars now – for a low cost drug they’d have to sell an awful lot to get that money back).

  11. windriven says:

    Interesting ethics wrapped around all of this. If an investigational drug had a 50-50 chance of prolonging an otherwise terminal patient’s life for, say, 60 days, I suspect that most of us would want the patient to have access. What about 25%? 2%? 0.2%? What has changed other than the odds? Who gets to draw that line? On what grounds?

    Now of course if we knew that an investigational drug had a 50-50 chance of prolonging life it wouldn’t be nearly so investigational. But the point is that for the terminal patient the chance of imminent death is right about 100% and so they and their families are inclined to clutch at whatever straws are within their grasp. (Ponder for a moment the psychology of the scum who would consciously exploit that desperation for their own gain)

    Someone who meant a great deal to me died a while back of anaplastic thyroid carcinoma, a Colt .45 disease if there ever was one. I am not an emotional sort nor a magical thinker and neither was she. In the event only a bit more than a week passed from her first treatment till her death. But had the disease taken longer I would have happily tried fermented banana skins mixed with goat saliva if a one-eyed snake handler from redneck junction claimed it might work.

    At some point one has nothing to lose. Nothing sounds quite as preposterous as doing nothing in the face of imminent death.

  12. Prometheus says:

    Angora Rabbit,

    Although DCA isn’t “out of the woods” as a potential carcinogen, the biggest problem with TCE metabolites is vinyl chloride. Most bacteria that can metabolise TCE only take it as far as vinyl chloride, which is much more toxic and carcinogenic than either TCE or DCA.

    Also:

    Several people have asked whether “Big Pharma” can simply repackage compounds that are “off patent” and make new, patentable (and, presumably, profitable) drugs.

    Short answer: yes.

    Examples:

    Niaspan (sustained-release niacin) – Abbot
    Circadin (sustained release melatonin) – Lundbeck

    For that matter, are the companies selling vitamins, “supplements” and – yes – DCA selling it at cost or are they (gasp!) making a profit?

    Let’s face it, if DCA looks promising, some company will do the necessary studies to get USFDA approval so they can sell it and say “for the treatment of glioblastoma” on the label rather than the “Quack Miranda” (i.e. “Not meant to treat or diagnose any medical condition. Consult your physician before use.”)

    It would be great if DCA turns out to be a safe, effective adjunctive treatment for glioblastoma or even a range of cancers. However, the “safe” part has yet to be demonstrated, as has the “effective” part. I am encouraged that research is underway, but it is still too early to tell.

    As for the inevitable anecdotes that start “The doctors gave me six months to live…” – what physician tells a patient with cancer that they have six months to live? I’ve been with two family members when they received a cancer diagnosis and here is what I heard:

    Patient: “How long do I have?”

    Doctor: “It’s hard to say. With current treatment, people with this type and stage of cancer can live for several years.”

    Patient: “What if the treatments don’t work? What’s the worst-case scenario?”

    Doctor: “Well, if the cancer turns out to be resistant to all treatments, I’ve heard of people dying in as little as six months.”

    Patient: “So I’ve only got six months to live!?!”

    Doctor: “No, I can’t say how long you will live, but most people in your situation respond to treatment and go on to live several years.”

    Patient: “But you said I could only have six months!”

    Doctor: “That’s if all the treatments fail and if the cancer is particularly aggressive. It’s the worst-case scenario – very few people have disease that aggressive.”

    Patient (two years later, after surgery, chemotherapy and radiation): “That doctor said I had only six months to live. It must be the bee pollen I’ve been taking.”

    I mean no disrespect to people suffering with cancer or who have family members that are. However, I’ve seen it happen – people in those sort of stressful situations tend to have highly selective recall.

    In addition, there are unscrupulous purveyors of “alternative” cancer treatments who – believe it or not – actually make up their testimonials. Amazing, I know, but sadly true, nonetheless.

    If you go to a physician who, after giving you a diagnosis of cancer, confidently tells you that you have “six months to live” (or some other time span), run out of their office immediately – they are either incompetent, insane or an imposter.

    No physician – not even an oncologist – can give you more than the average survival time and, sometimes, a range of survival times. The only exception is if you arrive at their doorstep in the final, fatal stages of cancer – as you might, if you decided to try “alternative” therapies first.

    After all, what’s the harm?

    Prometheus

  13. Harriet Hall says:

    windriven said “At some point one has nothing to lose. Nothing sounds quite as preposterous as doing nothing in the face of imminent death.”

    Yes, it seems that way, and desperate people grasp at straws. But it is not really “nothing to lose.” Money, time, comfort, psychological equanimity, and contact with reality can still be lost.

    It sounds preposterous to me to refuse to accept the reality of imminent death and seek false hope in highly improbable treatments just because “someone” thinks they might work. It is sad to see people go to Mexican cancer clinics and endure coffee enemas, restricted diets and all the uncomfortable rigamarole when they could have confronted reality, stayed home with friends and family, prepared for death, and found whatever comfort and small pleasures they could still enjoy.

    Some people see value in “going gentle into that good night” while others insist on raging until the last moment. Different strokes…

  14. markwells says:

    I object to the comments from the author and readers suggesting that the trials and/or DCA should be treated suspiciously because of claims that pharmaceutical companies aren’t interested in funding the research.

    The faulty logic in this suspicion of the drug and the researchers involved goes like this: “If a drug has potential as a treatment, then a pharmaceutical company would spend money on researching it; pharmaceutical companies aren’t spending money to research DCA as a treatment so it doesn’t have any potential.” I know that strictly, this is logically valid in the same way that saying if “if it’s the sky, then it is blue, but it’s not blue, so it’s not the sky” is. The problem is that one of the conditions is flawed, since a you can’t truly determine that a drug has potential UNLESS you spend money researching it.

    On the other hand, if it is out of patent, you can definitely determine if it would be profitable, or acceptably profitable, or compete with you other products in a way that would harm overall profits. (And by the way, don’t assume we’re all stupid by making arguments how a drug formula could be reworked to make a new patent – just because someone has added an ingredient to niacin doesn’t mean regular niacin is ineffective or unavailable.)

    Finally, contrary to the suspicions of some commenters, the DCA trials are in fact going forward with Government of Canada support: the University of Alberta is public and government-funded; Health Canada approved the trial; $700,000 in funding was provided by the Canadian Institute for Health Research, a Crown Corporation (that is to say, government-owned); and large individual community financial contributions brought in another $800,000 ($360,000 of that raised in bake sales and auctions in a small rural area of northern Alberta).

    Prefacing and colouring the very earliest results of an ongoing DCA trial with multiple accounts of criminals profiting off of cancer patients’ misery and desperation seems rather unjust.

  15. Wolfy says:

    Gorski: Another brilliant post!

    Prometheus: Nice anecdote. I can’t tell you how many times i’ve had this exact conversation.

  16. windriven says:

    Dr. Hall: When the terminal patient is you or someone who has entrusted you with their end-of-life decisions you are welcome to indulge yourself in empty platitudes.

    You would be the first to jump at someone who posted such a blatant false dichotomy. Choosing a macrobiotic rice diet on the off chance that it might extend by a few days the life of a pancreatic cancer patient does not require eschewing ‘friends, family and whatever small pleasures they might still enjoy.’

    Not all straws grasped are coffee enemas. Taking DCA does not mean ‘raging until the last moment.’ Is it a false hope? Sure. Is that somehow worse than collapsing into maudlin self-pity and despair? I’ve certainly seen my share of that.

    There are people who face their imminent demise with equanimity; curiosity even. I expect that I’ll be one of them. Perhaps you will too. But there are a lot of people – probably the majority of people – who aren’t wired that way. Don’t they have as much right to choose how they’ll die as they had to choose their profession, their mate, their politics? After all, it’s the last thing they’ll ever do.

  17. Wolfy says:

    windriven:

    for the reasons you mention, we should not fault patients for grasping at straws. it is difficult to know what any of us would truly do in identical or even similar situations.

    i do think, however, we can fault people for taking advantage of patients when they are at their most vulnerable and grasping for straws.

  18. windriven says:

    Wolfy-
    I couldn’t agree more. They should be flogged skinless and packed in salt.

  19. Wolfy says:

    windriven: yes, yes, yes! and then they should be made to bathe in lemon juice :D

  20. David Gorski says:

    I object to the comments from the author and readers suggesting that the trials and/or DCA should be treated suspiciously because of claims that pharmaceutical companies aren’t interested in funding the research.

    I call strawman argument, at least for me. The whole point of mentioning the “big pharma” pharma conspiracy mongering is because that’s part of what fueled the DCA craze based on a single animal study. My point was that such hype derives from not knowing just how many compounds look promising in cell culture or in animal trials and then fail in humans. DCA should be viewed no more “suspiciously” than any other promising candidate chemotherapeutic that showed some activity in animals–that it, it should be viewed very cautiously. The problem is that the scammers didn’t understand that, and they misled desperate cancer patients to believe that DCA was some sort of cancer cure.

  21. Harriet Hall says:

    windriven,

    Apparently you didn’t realize I was not talking about DCA but responding to your comment that in the case of imminent death from an incurable cancer you “would have happily tried fermented banana skins mixed with goat saliva if a one-eyed snake handler from redneck junction claimed it might work.” Some straws are more reasonable to grasp than others: it is not a dichotomy but a spectrum, and goat saliva falls at the same end of the spectrum as coffee enemas; DCA is towards the other end, since there is some plausibility. I was not prescribing what anyone should do, but showing that there were other points of view about what seems preposterous. I fully support the right to choose: I even said “Different strokes…”

  22. windriven says:

    Dr. Hall,
    Banana skins and goat saliva were specifically chosen to humorously suggest unlikely palliatives well short of coffee enemas and Mexican laetrile clinics. I hadn’t heard of DCA before Dr. Gorski’s post. My point was that faced with the imminent and certain death of someone I cared deeply about, idiotic nostrums from which I would have otherwise recoiled might not have seemed so repellent if they offered a one-in-a-million chance of adding a day to her life.

  23. BillyJoe says:

    windriven,

    But had the disease taken longer I would have happily tried fermented banana skins mixed with goat saliva if a one-eyed snake handler from redneck junction claimed it might work.

    It seems you are a sceptic until it really counts.
    Then you’re just like everyone else.
    What’s the point?

    (Maybe you’ll also have a spontaneous deathbed conversion – just in case!)

  24. windriven – “At some point one has nothing to lose. Nothing sounds quite as preposterous as doing nothing in the face of imminent death.”

    It has always been hard for me to understand the use of alt remedies in cancer. Both of my parents died from cancer. My mother was 64 when she died, 62 when diagnosed, so none of us considered her death timely.

    Like you friend, my father was diagnosed late, his cancer had metastasized to his hip, liver, brain, etc, so the idea that prayer for a cure, or any remedy would cure him seemed preposterous to me. As to controlling his pain and discomfort we looked to conventional medicine because we didn’t have any time to fool around with long shots.

    My mother was treated for her cancer by conventional medicine (and alot of personal prayer, I’m sure) because, once again, it was the best bet. If any alt approach had come up, the biggest concern would have been, ‘how could this interfer with her medical care?’ Since there is no science or documentation we couldn’t know.

    I’m sure there are things that are harmless, but to me, it seems if there is an actual physiological mechanism at work, it has as much chance of interfering with medical care as helping it, until proven otherwise.

    When it was decided that they could no longer treat her cancer, once again it seemed that conventional medicine was the best chance for relieving pain and discomfort.

    Honestly the only thing that medicine did not offer was marijuana, perhaps that could have helped her nausea, appetite. But, my Dad would have hit the roof if one of us kids suggested it. Which would have upset my mom to no end, so we did not broach the subject.

    It just seems to me, that at a certain point we had to decide what strategy we were going to take and then stick to it. If only for our own mental health.

    I don’t want to suggest that people who have a different attitude are wrong. I would think that cancer in younger people and cancer that is treated for far longer is a very different experience.

    I guess I just wanted to share why some people don’t take the alt approach and still don’t feel that they gave up prematurely.

  25. BillyJoe,

    Why does scepticism really count only in the case of terminal illness?

    windriven is not talking about a treatment that would adversely affect quality of life; it’s just bizarre. It makes sense not to con people into expensive, painful and ineffective treatments when their last days might be better spent saying good-bye, but if they can stay at home with their families and have a nightly banana skin–goat saliva milkshake, what does scepticism add?

  26. windriven says:

    @BillyJoe

    Is tone deafness a common concomitant to skepticism?

    Alison Cummins got it exactly right: neither a fully-functional and mature understanding of death nor fully-fledged skepticism about non-SBM treatments mitigates the desire to stretch the remaining quality time that a loved one has left. That does not mean that one abandons skepticism in favor of potentially dangerous remedies or those that reduce the quality of life.

  27. BillyJoe to Windriven- “It seems you are a sceptic until it really counts.”

    BillyJoe, I think it’s bad form to judge how someone handles a terminal illness. I would guess that Windriven’s first priority was not carrying the skepticism banner.

    Also, to me your comment sounds like an argument that skepticism required loyalty to the cause. I thought it was a tool for helping to gather information and make decisions. I only use that tool when I feel I need it. But that’s just my approach. I don’t self identify as “a skeptic”.

  28. Angora Rabbit says:

    Prometheus, yes, groundwater contains a variety of TCE metabolites. My comment was emphasizing human metabolism. Humans produce 1,2-divinylchloride-glutathione from TCE although the pathway is less relevant for TCE disposal than the oxidative metabolism via 2E1. Having said that, the glutathione conjugates may be longer lived and vinyl chloride is no fun either.

    There are reports in the literature of both TCA and DCA causing adverse cardiac effects at ppb levels. If something changes metabolic flux through ox phos instead of anaerobic metabolism, this is not always a good thing. For example, developing heart emphasizes glycolysis over ox phos.

    Since DCA is being used clinically to address mitochondrial metabolism errors, I gather that DCA lacks the ability to poison TCA Cycle intermediates the way that fluoroacetate can?

  29. superdave says:

    @harriet
    Your point is summed up nicely in the economic maxim that everything has an opportunity cost.

  30. Robmx says:

    Read and appreciate your comments about DCA when it was first in the news and today.

    Took them into consideration along with my primary doctors comment on DCA, “Don’t touch the stuff” and decided to try it anyway.

    I have metastatic colon cancer in lung and Hylar region. Am seeing oncologist and getting CT scans every 2 months now 2.5 at top cancer hospital. Have been seen by oncologist at two other top cancer center hospitals.

    One year after starting DCA I have had two reductions and four no change in size reports. I am healthy, have no symptoms other than peripheral neuropathy which is under control.

    Two of three oncologist tell me to continue what I am doing without specifically agreeing that DCA is working. The third emphatically tells me last week to continue exactly what I am doing until my tumors grow again and said obviously DCA is working for me.

    Each time I face a CT scan report I expect the worst and have been totally dumbfounded each time I get good news.

    This term between CT scans hoping to get another reduction, next middle of June, I have upped my dosage back to my original dose and triggered another side affect I have become familiar with, constipation. Which continues to increase along with peripheral neuropathy even after you stop DCA for a few days.

    Constipation bad enough to have me in the emergency room of a local hospital for the day last Sunday. But that is all it was and I am back to being fine.

    One question, why do you think that having people like me self medicating will destroy medical science as we know it? Seems to me that there should be some middle ground where doctors in good faith could work with terminal patients like me who want to turn the risk table on its head.

    I want to be in Phase One clinical trials, the reason I am seeing other oncologist at such as John Hopkins and Karmanos in Detroit. I want to take the risk while I am in good shape and not when I have exhausted all Palliative treatments.

    And what about the rest of the world where doctors are scarce and taking DCA to treat your dog is an insane use of scarce money while possibly an Internet chat with other self medicators and the stray scientist or technician about using DCA or other cheap available drugs may be helpful.

    There is a real world out there and it has a lot more gray in it than you allow.

  31. superdave says:

    @rob
    I we are all glad you are doing well, but the problem with what you are doing is that without testing for controls, we have no way to know what is actually going on. Maybe the DCA is helping you, maybe it isnt. If it is helping you, it’s hard to say why itis hellping you. Maybe there is a specific combination of treatments you hit on by accident. Maybe its your specific biochemistry. Without a rigorous controlled study, there is no way to know these answers.

  32. superdave says:

    pardon my grammar.

  33. superdave – In my opinion the world would be better off with less grammar. :)

  34. JMB says:

    @Robmx
    We are glad you are hanging in there, and every CAT scan you receive that shows no growth, you can consider as evidence that you are gaining ground. Under no circumstances should you let any discussion here on SBM dissuade you from the recommendations of your oncologists. Every case is different, and your oncologist knows your case the best. They are qualified by their training to give you the best medical advice.

    SBM is about how practicing doctors can utilize skepticism in the assessment of medical scientific literature to arrive at strategies for diagnosing and treating patients. The real gist of this article is that more research is needed before we know how effective DCA is, or how we can select a subgroup of patients in which the risks outweigh the benefits. None of this applies in your case, because you are already showing a lack of tumor progression that is hard to explain on the basis of current evidence. The oncologists also know that you are taking DCA, so they can properly observe for side effects. If your results cannot contribute to medical science, then that is more of a statement of the lack of organization of our healthcare system, and the need for greater sophistication in our methods of analyzing evidence. I think we will get there some day.

    Your oncologists may not be able to know based on current evidence in the medical literature if DCA is making the difference for you. However, they do not have to know, so long as your clinical course is beating the odds and your side effects are not overwhelming (and your treatment is not outrageously expensive).

  35. JMB says:

    Sorry, I should have said, benefits outweigh the risks. It is important to recognize subgroups in which the benefits outweigh the risks, or vice versa. The information about both subgroups can be useful in decisions.

  36. BillyJoe says:

    Alison & micheleinmichigan & windriven

    It is just easy to be sceptical and pro EBM when it’s someone else.

    But if you abandon it as soon as it applies to you or your loved ones when they are in pain, or have a serious, or chronic or terminal illness, what use is it really.

    How genuine can your support of SBM be if you abandon it as soon as it’s you with the disease.

  37. BillyJoe says:

    ON the other hand, I see no problem with the following scenario:

    On “The 7:30 Report” on the ABC last night here was an interesting case of a teenager with rapidly progressive MS that responded dramatically to unproven stem cell treatment.

    The clinical trials had not been done, but there was a plausible mechanism and the patient was in a serious clinical condition on a respirator with nothing to lose.

  38. “But if you abandon it as soon as it applies to you or your loved ones when they are in pain, or have a serious, or chronic or terminal illness, what use is it really.”

    I have no idea whether I would or not. I tend to be temperamentally conservative, so I probably wouldn’t. But maybe I would. My vet prescribed glucosamine with chondroitin for my chihuahua and I gave it to him because it probably didn’t hurt, even though I looked it up on the Internet and it didn’t seem to have an evidence base. On the other hand, my dentist likes homeopathy and I don’t use it. (I don’t fire him because he is actually really, really good with teeth and on top of all the latest tooth-related research.) I guess I’ll find out when I get there.

    I think the banana skin–goat saliva impulse reflects an emotional need to feel like we did everything. If something bad happens, it’s not because we stood idly by.

    My 36 year old friend with terminal metastatic cancer has had various brief flirtations with alternative modes of healing (eg yoga) but has always kept his focus on what his oncologists are telling him. He knows that he is not standing idly by – the surgeries, chemo and radiation therapy are no walk in the park – and he feels as though he has been presented all the options. Science-based medicine is meeting the emotional need to feel as though he is doing everything he can.

    In other cases, SBM might not meet that emotional need. There might really be nothing to do, or it might not be communicated effectively. If the emotional need to feel as though one has done everything is not being met through SBM, and it can be met by doing something that is not harmful, I don’t think that recognition of the emotional need is in itself rejection of SBM.

    A brief blog post I wrote last year:

    http://www.alisoncummins.com/2009/03/10/crimes-against-the-present/

    “Mark brought our excellent neighbour over to the house yesterday to give her a tour and show her how to water the plants while we’re gone. She’s been preoccupied lately with her brother, whose health is not good these days. He has cancer which has progressed and metastasized to his brain. Our neighbour described in detail the support she is offering him: potent vegetable juices to boost his immune system; coaching to boost his morale. “You’re only fifty-seven! You don’t want to die now. Just think, you’re about to enjoy your retirement! Fight! Live!”

    Oh dear. Magical thinking. Ineffective remedies. And badgering the poor man in his last days. Can’t she just let him die in peace?

    And then I thought: good thing he’s not my brother. I’d probably be muttering “What, not dead yet? What are you waiting for? Look, it’s in your brain, no point in hanging on now.” At least my neighbour’s brother knows he is loved.”

  39. “ON the other hand, I see no problem with the following scenario: [...] The clinical trials had not been done, but there was a plausible mechanism and the patient was in a serious clinical condition on a respirator with nothing to lose.”

    I do see a problem with that scenario. He should be in a clinical trial. If it’s plausible and doctors are willing to try it out, then they should be willing to conduct a trial. That whole registry thing.

  40. BillyJoe says:

    Alison,

    Sorry, I tried to find a link to that MS story. He was a young lad who rapidly progressed from paralyis of his leg muscles to respiratory failure as a result of paralysis of his respiratory muscles. He was in intensive care on a respirator. There had been some indiviual case reports of some benefit from stem cells. Because the lad was dying and no clincally proven treatment was available, and failing any consensus through a neuologial case conference, his treating neurologist, after discussion with the family, decided to ablate the patients bone marrow and inplant some of the patient’s own stem cells.

    The procedure was plausible: The immune system causes MS by reacting against and destroying the patient’s own myelin sheath. So, it makes sense to get rid of the defective immune system and start afresh with a new immune system derived from the patient’s own stem cells.

    He is now able to breathe on his own and walk unassisted and back at hime with his parents. And the change in the brain scans is dramatic.

    Of course, because of the significant risks of bone marrow ablation this should be carried out only in severe, rapidly progressive cases like his. This would more or less make clinical trials unrealistic. A case series, where well defined patients did obviously better than the natural history would predict, would be more realistic way to confirm the benefits of the procedure. After all, after that dramatic response to an eminently plausible procedure, how could you divide your subsequent patients randomly into treatment and control groups especially considering they would be arriving at intervals measured in years?

  41. BillyJoe says:

    …and, I’m sorry, I don’t believe in being wishy-washy about the use of alternative medicine. When there is no plasuible mechanism and no evidence of benefit – except to the practitioners who promote it and to the cause of alternative medicne in general – then to use it is worse than doing nothing.

    I don’t think meeting the emotional needs of the patient is a good excuse. There are surely better ways to fulfil the emotional needs of patients than kidding them with implausible untested or refuted alternative medicine rituals and nostrums.

  42. BillyJoe,

    The last time my sister came for a family visit she mentioned a patient she’d left behind. She was elderly, had had a stroke, and was unconscious and dying. My sister left a note in the patient’s file that the family was permitted to use traditional remedies.

    If even my painfully EBM sister allows family to try cupping her patients in the hospital for the sake of the family’s peace of mind, then I think you’re being more catholic than the pope. In this situation nobody had abandoned EBM: the patient was in the hospital (doctor and family in agreement that this was appropriate) and being manged with EBM. The cupping or moxibustion or whatever the family were using was not preventing anyone from receiving care.

    There are surely better ways to fulfil the emotional needs of patients than kidding them with implausible untested or refuted alternative medicine rituals and nostrums.

    Who’s kidding who? You think my neighbour should have been forbidden to offer vegetable juice to her brother? Or to try to boost his morale? Or pray with him?

    windriven isn’t asking a doctor to prescribe banana skins and goat saliva. windriven is saying that as a distraught friend, the emotional context could make banana skins and goat saliva seem surprisingly appealing.

    It’s patients and their families who get to define their emotional needs, and they are the ones who get to decide when they’ve been met. You might have opinions about what a rational, healthy person’s emotional needs should be and about proper ways to meet them, but real people don’t necessarily ask your permission first.

    As I said upthread, SBM often (my guess is usually or almost always, but I don’t have the data) does meet the emotional needs. There may be times when it can’t.

  43. BillyJoe says:

    Alison, I think we are talking about different situations here. You are talking about preventing others from using altmed for their diseases. I was talking about SBM supporters showing true lack of conviction by resorting to unproven and implausible altmed nostrums when the disease actually hits them or their loved ones. I was responding to this quote from Windriven:

    But had the disease taken longer I would have happily tried fermented banana skins mixed with goat saliva if a one-eyed snake handler from redneck junction claimed it might work.

    As I said, it just easy to be sceptical and pro SBM when it’s someone else with the disease, but when you are the victim and you blithely accept the next unproven implausible altmed nostrum that presents itself, what’s the point in all your scepticism, if you respond just like everyone else?

  44. Robmx says:

    Someone asked for more info on how I started and why I started on DCA.

    I am like Obama who said to a meeting of the AMA. “I do what you folks tell me to”

    Never thought I would be self medicating. Medical science has saved my life at least two times and I never thought to question doctors much having never taken a course in biology.

    But over the years before having cancer I developed an attitude to chemo that gave me pause when my oncologist told me that I had metastatic colon cancer and wanted to give me a port the same day. I asked for a second opinion. He suggested someone and the delay in getting that opinion, from April till September 2009 was when I started DCA.

    My father had prostate cancer and was progressing in 2007. I had had a colon resection in December 2006 and was thought to be cured and not given chemo then. But while waiting for that pronouncement I was avidly looking for answers to my colon cancer and discovered DCA that was in the news in January of 2007.

    I would not have bought DCA though I was intrigued by it for my fathers sake. Someone sent me 50 grams as a sample. It lay around for two years while I debated with my siblings about giving it to my Dad. He died without my giving it to him.

    So when I was diagnosed last year with metastatic colon cancer and the wait for a second opinion took months I asked my doctor if I should use DCA. Against his advice I did anyway.

    And I keep using it only because the CT scans show either reductions or stable disease. As I told my oncologist last time after my April 5th CT scan. “If you had come in the room and announced that my tumor had grown I would want to be on chemo this afternoon”.

    So the tension rises as my next CT scan looms middle of June, she extended the interval to 2.5 months from 2 months.

    Other things I am interested in, Reolysin, G-202, BBI608, JX-594 all in clinical trials. Difference is I can’t get my hands on them.

    Very interesting thing this cancer. Can’t help but thinking that we are missing something. No matter what the FDA does there are going to be very larger numbers of people self medicating in the world.

    Seems that this chaotic ongoing clinical trial involving millions of people worldwide could be better controlled and mined for data if someone could come up with say an Internet based data collection and analysis system. A system that could also impose more order.

    As it is cancer patients in clinical trials and in chemo treatment are self medicating like CRAZY anyway and their oncologist don’t know what there are taking though they all know it is going on. There is nothing pristine about anything going on now even in the FDA USA.

    A doctor asked me if I was in a clinical trial if I would still take DCA on the side. I told him no that I was interested in finding out what the results of the agents being tried and thought it would be unethical anyway to secretly be sabotaging the trials results. He said in response that people were doing it anyway in trials all the time.

    It would be better in my opinion if we could entice everyone to be totally honest about what they were doing and then take the data and use super computers to extract the real info.

    For example I take 200 mg of Metformin a drug used by diabetics. I take it because diabetics who have been taking Metformin for years have been in a clinical trial they were unaware of.

    Someone noticed in massaging the data that diabetics who used Metformin were less likely to get cancer and when treated for cancer were less likely to suffer a reversal of a remission. Then in vitro they discovered that Metformin kills cancer and cancer stem cells.

    Metformin seems to work on the metabolic side of cancer like DCA.

    Also take 4000 iu of D3. Someone noticed in the data that people who take a lot of D3 seem to have less cancer. Sloan Kettering Cancer Center is about to start a trial of D3 to see if it slows or stops the progression of colon cancer in high doses.

    Wish I had taken biology.

  45. BillyJoe says:

    Taking everything and anything that has ever been shown perhaps, maybe, to help cancer is clasping at straws. Somewhere someone thinks they have shown X helps kill cancer cells. There may be N different Xs. Are you going to take all N treatments? N could be 100. Most people attracted to this type of misguided activity usually just pick the first one or two that they come across. Luck of the draw. But why those two? Why not the other 98 unproven treatments?

  46. “As I said, it just easy to be sceptical and pro SBM when it’s someone else with the disease, but when you are the victim and you blithely accept the next unproven implausible altmed nostrum that presents itself, what’s the point in all your scepticism, if you respond just like everyone else?”

    I don’t think it’s an all or nothing proposition. Many people consult altmed for any complaint. Have a cold? Altmed, Kids acting up? Altmed. Have a cat scratch that is red and puffy with a line running up your arm…put witchhazel on it. (That’s an old friend of mine.) For them altmed is a lifestyle choice.

    These people are very distinct from those who generally seek conventional medicine but are in desperate straights. Often the second group are people much closer to the “since it can’t get any worse, maybe this could help.” end of the spectrum.

  47. BillyJoe says:

    I was talking about posters here who claim to be sceptical and pro SBM. It seems, for some, their scepticism and pro SBM stance lasts as long as their next serious illness. Then they’re quite willing to try unproven and implausible alternative medical “treatments”. Excuse me, then, if I smell a lack of conviction, or even hypocracy, in their stance.

  48. mdcatdad says:

    And how do proponents of this “miracle” explain why doctors do not take it when they have cancer or do not provide it to family members and friends.

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