We have no idea why Tylenol works. Acetaminophen, the active drug in Tylenol, was the happy consequence of guess and check (and not without some grave errors along either). The mechanism that makes acetaminophen effective is unclear to us.
It seems odd that something we use for common aches and pains is a complete mystery to scientists. But Tylenol is not the only drug we haven’t the slightest as to why it works. This happens with: penicillin on infections, lithium for bipolar disorder, many drugs that affect our brain. Beyond this, older drugs are often rebranded for new purposes when the side effects are considered useful solutions to other problems. Thalidomide, for example, was originally on the market for reducing morning sickness. Then someone realized it would better serve as a treatment to cancer and leprosy (don’t ask me how). Or one great factoid to (never) use at a party: Viagra. It was first sold as a medicine for blood pressure but then, you know, we realized it helped with erectile disfunction.
How is this possible? Well, functionally specialized research (finding a cure or treatment to a specific disease, for example) often leads to researchers trying different chemical reactions or drugs and seeing what works.
“But the dirty secret of modern medicine may be that the most logically alluring method for solving diseases – steadily accruing knowledge about human biology that we can act on – hasn’t always been the best [fastest] way to hit home runs. An older method of just slinging possible drugs at cells, animals — or in some cases, people — and watching what works has often been the more productive starting point, with understanding to come later.” Carolyn Y. Johnson
There are a lot of medications that we don’t understand. More than this, there are a lot of diseases we don’t understand (at the molecular and molecular pathways level). We approach the unknown with a “throw everything at it and see what sticks” mentality.
Why does this matter?
Knowledge is power. Understanding the mechanisms of something matters. Imagine this: you are playing a game of chess. As the game progresses, you realize the strategy of your opponent. Because you know their next moves (functionally how they work) you are able to block their next moves, making them no longer a threat – or at least an effective one – to your king.
Now imagine, you never figured out the strategy of your opponent. You decided to randomly move your pieces on the chess board to see what happened.
Now you are choosing your “move” (what medicine to use or develop) against your opponent (let’s say pancreatic cancer) to beat them (it). Instead of making random moves (like testing out random drugs and chemicals on it to see the effect), let’s say you knew the in’s and out’s of your opponent. You know all the molecular pathways which cause the mutation of the cell to reproduce unchecked. Then you might be able to design a drug or use an existing drug to block certain processes from occurring.
So why doesn’t this currently happen?
Old habits tend to die hard. Researchers receive grants for specific interests, of which donors or philanthropists have connections to said disease. It would be rare to see someone receive grants for studying “cancer” rather than a specific type of cancer. Beyond this, medicine may be one of those incidences where more effective production doesn’t come out of specialization (contrary to Adam Smith). At least not yet.
There is a need for what is referred to as “basic research“. Basic research is studying the foundations of life – like cells, or newly discovered molecules, or little-understaood processes. It is research performed without obvious or immediate benefit, meaning you aren’t researching with known obvious value to the knowledge sought, but simply to understand.
What should we do?
My prescription for improving global health is to direct further funding to basic research. This can be achieved through government spending (such as grants) and tax incentives to those who fund basic research. This would yield more basic research and thus fundamental knowledge of the world around us.
It is an odd prescription (I admit) to push for more basic research, which by definition has unknown benefits. However, basic research has led to amazing discoveries and application in furthering health and science. Two really great examples: the discovery of DNA and the discovery of neurotransmitters, which lead to treatments in cancer and antidepressants, respectively.
This has the potential to greatly improve or lead to (new) medicines for a wide variety of diseases. It also has the potential to allow us to understand medicines which are currently mysteries.
In Epic Measures, J. Smith shows the heroic measures (of Chris Murray) to understand disease and health on a global-scale. The Global Burden of Disease Study not only adjusted previously misrepresented numbers of disease, but it also included injury and disability to give a more complete picture of the health concerns that people face globally.
It is now, after such amazing progress on the global-scale, that we need to examine and explore the micro-level of our world (and of biology) to allow us to better treat (and cure) different ailments which affect us. DALYs, the measurement used to index injury and disability with mortality, have paved the way to understanding what affects who and by how much. BUT what is missing is how does it manage to effect them.