Informed Consent

“Informed consent involves three components: information, comprehension and free will. Individuals vary in their ability to comprehend information relevant to their consent, which minimally includes the nature of the procedure and the implications of their participation in a research study. Thus, providing necessary information and subsequently establishing comprehension are both essential aspects of obtaining consent. The final component of informed consent is free will.”
[Nature Methods 6(2):111 (2009)]

My View on Alternative Medical Therapies

I'm sure that you've noticed that there is a strong rise in the availability (and marketing) of alternative medical therapies. This is generally a good thing. Quite often Western medical practice advances incrementally, slowly improving, building upon historical foundations, studies and existing gold standards. This is also a good thing; especially in terms of safety and the ethics of providing the best possible care. However, this also means that there are generally no big leaps forward in medical therapies. This is obviously a bad thing. I argue that the recent spike in interest in alternative therapies is healthy, by their very existence as competing alternatives, to shake up thinking, to get researchers, clinicians and companies to consider completely different paradigms. It's really the same argument economists use when describing the benefits of a competitive marketplace for any "product". Eventually, the best of the alternative techniques -- the ones that can be picked apart, understood from a basic science perspective, tweaked, tested thoroughly via clinical trials -- should be eventually integrated into standard Western medical practice.

Although I fully support fostering alternative therapies and learning everything we can from them, what gets me somewhat angry is when these therapies are marketed in such a way to lead potential patients to believe that they have been rigorously tested, and are thus on par in safety and efficacy with the standard or equivalent Western therapies. Complete understanding of the mechanism of a therapy is probably setting the bar a bit too high (e.g., we didn't understand how penicillin worked when it was first disseminated; we just knew it didn't harm us in large doses and that it did a good job at killing bacterial infections). But, at the very least, if there has not been any legitimately run clinical trials (i.e., double-blind) that have been overseen by a neutral and expert third-party body (e.g., committee of experts in the field, FDA, etc.), those therapies should be marketed as "try at your own risk" with the caveat that "it may or may not be as effective as the current gold standard" for that particular disease. Not informing patients in this manner is unethical. Even the fact that we don't fully understand the mechanism of action should be disclosed, regardless of how well tested a treatment is. On this note, I am fervent in my belief that the same should be done with the Western therapies. If a drug has gone through clinical trials and been FDA approved, but we don't really know why it works, it should be disclosed. Full disclosure should be the norm.

I realize that I've probably already struck a nerve with some who will respond that it's an unfair financial burden to put on many treatments, which by their ancient nature are quite often unpatentable, and in any case which have been used for generations throughout recorded human history. To those I'll simply reply that it's true that history of successes is indeed a good sign, it certainly gives credence to a potential therapy, but how complete and unbiased are those ancient records, often not even written down, but passed down orally? For example, for one ancient therapy there could have been for every 9 successes, one fatal failure. Would this failure be attributed to the therapy? Would the story make it to the historical records? And what about optimum dosage, was that studied and recorded? Sometimes yes, but quite often not. In fact, for plant-based products, even if the traditional therapy called for the administration of "one crushed leaf", for example, simply living in a different region might have provided a different effective dosage of the active ingredient, by the very nature of the different microclimates affecting the growth and development of that plant. Wouldn't it be useful to know what the optimum, let alone safe, dosage should be? And isn't it our duty to ensure, by modern standards of measurement and recording, that the success rate of the therapy is acceptable? Just knowing if there was a 1 in 10 failure rate for a particular therapy would be useful. Even if this is worse than the existing gold standard Western treatment (assuming there is one), it would be disclosed and then it would be up to the patient to decide, quite similar to how a patient is quoted the odds of complications prior to signing off on having a surgical procedure.

Finally, I don't think all alternative therapies are of equal value. In fact, I have some fairly strong opinions on the main categories of therapies:

• Botanical Medicine: These are quite exciting in their potential, given that there are so many possible plant-derived substances that have not yet been thoroughly tested. Those that have been shown historically to have some success (e.g., those past down through indigenous medical traditions) should be the first ones we explore. Just to name one example, we should study graviola (Annona Muricata) more seriously.
• Acupuncture and Healing Touch: Though our understanding is still lacking, both in terms of basic science at the molecular level, but also at the level of physiological mechanisms related to the nervous system, there is growing evidence through clinical trials that the effects seen are more than the simple placebo effect.
• Homeopathy: This is the category that disturbs me. Bluntly, I think it is complete fantasy. That is not to say that it can't have benefits, just that I'm convinced that any benefits are completely due to the placebo effect alone. The main problem I have in giving any serious credence to this branch of therapies is that they advocate the administration of "homeopathic dilutions" of various substances (specific to the disease being treated), dilutions that are supposed to be so dilute that they are, in fact, impossible to create. For example, a common therapy level advocated is 1 molecule of active ingredient for every 10^60 molecules of water. (For a sense of scale, 10^60 is roughly the number of stars thought to be in our universe, cubed!) Assuming that one could create such a dilute solution, the probability that the patient drinking a 1L bottle of this solution (which is a lot more than is typically prescribed; often a few spoonfuls per day) would get anything but pure water would be infinitesimally small (i.e., only one in hundreds of billions of these bottles would contain one molecule of the active ingredient). Of course, homeopathic practitioners claim that it's not the actual presence of the molecule that counts, rather that it came in contact with the water (or other diluent, such as alcohol or sugar) and, thus, changed the properties of the water. However, this later claim has been disproved time and time again (although there are scientific papers published on this, the BBC did an entertaining documentary on this topic); water with nothing in it, even if it at one time came in contact with another substance, is just water, with the properties of water.
• Placebo Effect: Though I knocked any effect seen in homeopathy as being "simply" due to the placebo effect, that is not to imply that this effect itself is actually simple, as it is rather quite fascinating! To think that by believing something, we can affect change and healing in our bodies is quite mind boggling. It's quite exciting to think of the possibility that with our brain we might be able to control very specific functions, such as those related to one particular type of cells in our immune system. Though the data is scant, to say the least, there definitely needs to be more people studying this avenue of therapy, perhaps to cultivate it as an adjuvant strategy to complement other therapies. Often, in medicine, the placebo effect is seen in a negative light, but we need to stop thinking that way!

Gene Therapy 101

In the media these days we often hear of "gene therapy" and its great promise for the future of medical treatment. I have never taken the time to look up how it actually works until now.

----- [ From the Human Genome Project ] -----
Gene therapy is a novel approach to treat, cure, or ultimately prevent disease by changing the expression of a person's genes. The field is still in its infancy, and current gene therapy is still primarily experimental, with most clinical trials only in the early stages.

Gene therapy can be targeted to somatic (body) or germ (egg and sperm) cells. In somatic gene therapy the recipient's effective genome is changed, but the change is not passed along to the next generation. In germline gene therapy, the parents egg and sperm cells are changed with the goal of passing on the changes to their offspring. Germline gene therapy does not seem to be actively investigated, although a lot of discussion is being conducted about its value and desirability.
----- [ end quote ] -----

A common gene therapy strategy is to deliver "good" DNA material to cells via a carrier virus. A commonly used virus is the adeno-associated virus, which does not cause any known diseases or trigger immune responses such as inflammation. In the lab, most of the virus’s own DNA is removed and replaced with therapeutic DNA. Then it’s injected into the patient’s tissue, where it does what it does best: infect cells. Because the virus is coated with specific marker molecules, the DNA material carried by the virus eventually gets into the cell nuclei. From there it is expressed like any other DNA in a cell nucleus. None of the "bad" DNA is replaced or changed by this method, a common misconception for those unfamiliar with gene therapy (view a Macromedia Flash animation of gene therapy by this method here, courtesy of MIT Technology Review).

In most diseases the root problem is either a lack of production of a particular protein (perhaps due to a specific gene not being turned on), or a particular protein being produced incorrectly (perhaps due to incorrect DNA coding). In both these cases, with the type of gene therapy outlined above, the newly added DNA material will supplement the cell's genome with an independent piece that correctly codes for the production of the protein in question. An example of a disease that could be potentially cured by gene therapy strategies is cystic fibrosis. People who suffer from cystic fibrosis produce a faulty cellular transport protein called cystic fibrosis transmembrane conductance regulator, which results in the build-up of mucous in their lungs.