A study just reported at the Heart Failure 2013 Congress in Lisbon, Portugal indicates that co-enzyme Q10 (CoQ10) is effective in congestive heart failure, improving both function and survival. This is of considerable importance to the use of CoQ10, of potentially great significance for the treatment of heart failure, and of far more profound relevance to the entire endeavor of biomedical advance.
Congestive heart failure is an increasingly prevalent condition around the world as the chronic disease epidemiology of modern living takes over the globe, and populations live longer. The most common variety of CHF, as it is known, involves weakening of the muscle in the heart's left ventricle following bouts of ischemia and/or infarction (heart attack). Areas of heart muscle stop pumping because they are starved for oxygen, and the heart becomes a less efficient pump overall.
The consequences of this are just what they would be when any pump stops working right: whatever it is pumping backs up and pools. In the case of the left ventricle, that substance is blood, and it backs up first into the lungs. This causes shortness of breath, limits exercise tolerance, and can make it hard to lie down flat. But it can also lead to something called acute pulmonary edema, when the volume of fluid in the lungs creates severe breathlessness. Essentially, this is drowning, but the fluid is coming from within, rather than without.
Heart failure is, as noted, increasingly prevalent. When severe, it can limit function to the point of making walking nearly impossible. It waxes and wanes with diet, activity, stress, and more in an often unpredictable pattern that results in frequent need for hospitalization, and very high costs. And over a span of decades, very few medications have been identified that work well to treat CHF, and far fewer that lower the associated mortality risk.
Which brings us back to CoQ10. The new study showed it did both, and that's just about astounding. Co-enzyme Q10, also known as ubiquinol, is a compound found widely in plants, but in very low concentrations. Our bodies need it, using it to facilitate the vital, energy-generating process of electron transport in our mitochondria, the power-plants residing in our cells.
We can make CoQ10, but not with great efficiency, and get much of what we need from food. Levels in our bodies rise and fall with dietary intake, and with other exposures. For example, statin drugs used routinely to treat high cholesterol deplete CoQ10 levels. In my personal clinical experience, and the common clinical experience, supplementation of CoQ10 often ameliorates the muscle soreness that is rate-limiting for use of statins.
Proponents of CoQ10, and there have long been many particularly in the natural medicine world, have asserted its value in treating high cholesterol, high blood pressure, periodontal disease, heart failure, low energy, and more. One always worries when a medical remedy starts sounding like a Ginsu knife: "It slices, it dices, it feeds your fish, it bathes your children...!" But actually, the mechanism of action of CoQ10 is so very near the bedrock of our metabolism, it makes sense that it would affect every organ system, and have implications for almost every condition. As an example, every condition is worsened by a deficiency of oxygen, and improved when we have enough. The notion of a legitimately, universally relevant "remedy" is not entirely unprecedented.
The current study, which thus far has only been presented at a conference, albeit a prestigious one, and not yet published in full in the peer-reviewed literature, appears to be a game-changer. The trial was conducted in nine countries, enrolled hundreds of patients, and followed them for two years. It had all the right bells and whistles, too: It was randomized, double-blinded, and placebo-controlled.
The punch line was refreshingly straightforward: The CoQ10 group had half the mortality rate, and half as many adverse cardiac events, as the placebo group. Both findings were of clear statistical significance. That they were of stunning clinical significance is all but self-evident, but it embellishes the case to note that a drug to lower mortality in congestive heart failure has not been identified in over a decade.
What makes this study truly astonishing, and causes it to reverberate through modern medicine far beyond the topics of either CoQ10 or heart failure, is that the very possibility of such an effect was overtly dismissed that same decade ago. In 2000, a paper in the Annals of Internal Medicine purported to show that co-enzyme Q10 did not work for heart failure. An accompanying editorial went further, declaring the co-enzyme Q10 hypothesis dead.
But this was a study of just several dozen patients, followed for a few months. In a tale I've told before, this was in contrast to a study of the patented drug, Carvedilol, published around the same time and demonstrating its value in heart failure. The study of Carvedilol enrolled thousands, and lasted years.
And so that 2000 study of CoQ10 did not generate meaningful evidence of absent effects; rather, it left us with a rather meaningless absence of evidence. I have long argued, including at an Institute of Medicine summit, that just such scenarios require us to think of medical evidence more fluidly than just "yes" or "no." Absence of evidence is not evidence of absence, and thinking it is mistakes the subtleties of incremental advance and shades of gray for the black-and-white of a light switch. Science doesn't work that way.
But there is a very vocal group, self-proclaimed as guardians of science, who argue that it does. For my defense of such notions as the possibility that CoQ10 might work for heart failure after all, I have been feathered, if not tarred, in cyberspace. All water off a duck's back in light of the new study.
I hasten to add I did not believe that CoQ10 worked for heart failure in the absence of good evidence. I just believed we had an absence of good evidence. I believed we had a good Q, and no clear A.
One would like to think that the Q&A that drives biomedical advance is about what matters most. But the long and tortuous timeline associated with the attachment of the right A to CoQ suggests it has a lot to do with patents, and profits, and what pays the most. CoQ10 is all but ubiquitous in plants, and you can't patent parsley. We talk a lot about evidence-based medicine, but the playing field for generating evidence is far from level. Patent-holders perennially have the home-field advantage.
That CoQ10 almost certainly works for heart failure is important to the clinical use of CoQ10, and important to patients and doctors dealing with heart failure. But it is perhaps even more important to all of medicine that CoQ10 is being shown to work more than a decade after the very possibility of it doing so was ruled out. Biomedical advance is forestalled, and the human condition compromised, when absence of evidence is not only tolerated, but mistaken for evidence of absence.
We can avoid the repetition of that mistake by keeping our minds open. Not so open our brains fall out, of course, but carefully open just the same. For we now have an exceptional medical story to prove a rule: A closed mind is a menace to every other organ.
David L. Katz, MD, FACP, MPH, FACPM, is an internationally renowned authority on nutrition, weight management, and the prevention of chronic disease, and an internationally recognized leader in integrative medicine and patient-centered care. He is a board certified specialist in both Internal Medicine, and Preventive Medicine/Public Health, and Associate Professor (adjunct) in Public Health Practice at the Yale University School of Medicine. He is the Director and founder (1998) of Yale University's Prevention Research Center; Director and founder of the Integrative Medicine Center at Griffin Hospital (2000) in Derby, Conn.; founder and president of the non-profit Turn the Tide Foundation; and formerly the Director of Medical Studies in Public Health at the Yale School of Medicine for eight years. This post originally appeared on his blog at The Huffington Post.