Monday, April 16, 2012

Insight on Research

Every so often you will come across a depressing article about someone (usually a young child or an adolescent who is the star student and athlete, yadda yadda yadda) who is fighting a debilitating disease. The disease is usually progressive, and typically very aggressive or rapid in its progression and with a poor prognosis. Everyone is clamoring about how sad it is (which it is) and how everyone should pray, and that they hope they find a cure soon. Likewise, I typically see donation stations for various foundations that donate money to research asking for $1 here or $3 there to be donated to researching the cure for disease X. Then I'll hear groups (usually right wing groups) complaining about how millions of dollars are poured into researching these diseases and yet we have had no cures or new medications, and so they whine and complain about how it's wasting their tax dollars and we could use the money better elsewhere. In all of these situations however, people need a much better understanding of scientific research, if for no other reason than to prevent them from getting upset or having false hope. Realistically, a cure is not going to be discovered overnight and save the life of featured new story child, nor is a cure/medication going to be discovered suddenly, a mission accomplished flag waved, and research money allocated elsewhere preventing the complaints of the economic conservatives. You may have heard it before elsewhere, but for any drug, the time from research to market is approximately 20 years. In those 20 years, let look at the breakdown of the process. The money given to the research is used to buy equipment, reagents, and pay the salaries of the interns, students, technicians, and professors researching. A discovery might be made in-vitro at first, after which it is then tested in an animal model (rats, mice, etc.). Animal studies take time, and a lot of money, so again, we use some research bucks and the clock ticks by. If the treatment or drug seems to work with some statistical significance in helping animals with similar symptoms/disease characteristics (this is key, because a mouse with disease X or similar symptoms is after all a mouse, and does not guarantee that the same therapy will work in humans) then the research will progress to clinical trials in humans. This is where you'll begin to hear advertisements on the radio for an easy $3200 if you'll only let someone inject you with something and stay overnight for 4 days. Additionally, human clinical trials are broken into 3 phases. At first, a phase I clinical trial is performed to determine the metabolic and pharmacological actions of the drug/treatment, its side effects at different dosages, and to see what early signs of effectiveness are evident. If everything appears to be working, the study is progressed to Phase II where additional trials evaluate how effective the treatment is for a particular indication or patients with a given disease to see what the short-term side effects and risks are. If the drug/treatment seems safe and still somewhat effective, then the study is progressed to Phase III, where expanded tests are performed to further evaluate the effectiveness of the treatment and to better determine the benefit versus risk of the treatment so that the treatment has a good indication of appropriate use for physicians. If everything is still fantastic, then we have the treatment evaluated and approved by various regulatory bodies like the FDA, and the treatment is eventually brought to market. This is all in a perfect world where nothing goes wrong. In reality, a treatment or drug will often not function the same in animal models as it does in cell culture or other in-vitro studies. It may work wonderfully in animals but not be efficient at all in humans, or worse, cause a myriad of side effects not observed in animals due to the physiological differences of humans. The treatment may fail at Phase I due to complications or study participant toxicity and fatalities. It may fail at Phase II due to a lack of effectiveness or an excessive risk to the patient. In the end, it is exactly what it appears to be at face value: a study; an experiment; a scientific venture. Science is precise, and exact, but it does not carry a 100% success rate. It is a field of educated guesses and trial and error. Breakthroughs are not made overnight, and even a 50% success rate can be the best you can hope for at a given time. The sad truth is that real science does not work like it does on shows like CSI where analysis and solutions to problems are found in 5 minutes or by the end of the 1 hour program. Experiments take time, failure, re-analysis, and new attempts. New treatments and cures will be found eventually, but it will take time, testing, regulatory approval, and more time to bring to market. Disease are constantly evolving, and with them so must treatments; a cure or treatment for one type of a disease may not work for a second type of the disease, no matter how closely related the symptoms are. There will most likely never be a cure-all for cancer or other debilitating diseases, but rather better treatments and earlier detection techniques that enable earlier interventions. The sooner the general population realizes this and tempers their hopes with a healthy does of realism and understanding of the nature of scientific research the better. Does this mean we should fund these areas less? Of course not. We should fund them more so that breakthroughs can be made faster. But faster means 17 years instead of 20; not a cure you can pick up from Walgreens in 6 months.


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