Transduction, s'il vous plait?

As most of you are aware, I work for the National Center for Advancing Translational Science (NCATS) here in Rockville, MD. We often talk about translational research at the office - how it works, how we can improve on it, and how we can use it to make people healthier. But what is translational research? The vast majority of biomedical research in the United States is known as basic research. This research seeks to understand the fundamental mechanisms that govern life - how genes are regulated, what proteins influence biochemical pathways in the body, and so on and so forth. I think that this research needs relabeling, as basic seems to imply a lack of complexity (at least, that's what it sounds like to me!). The word fundamental would probably be more apt here, as this research is absolutely critical to applied research. Applie research uses the information gathered in basic research to create new therapies for humans, such as new medical devices or new drugs. The process by which knowledge is transformed from basic to applied research is known as translation, which is the challenging topic that my institute focuses on.

This is certainly easier said than done. As you can imagine, a vast amount of basic research is performed around the world every year, yet the rate at which we develop new therapies is much lower. 200 billion USD was spent on research and development last year ($90 billion in the US) alone in the life sciences, while thousands of scientists work around the clock to figure out new ways to tackle problems in human health. Do you know how many drugs were approved by the FDA last year? 41. Forty. One. This is actually much higher than the 21 per year that the FDA averaged over the past decade.

Okay, let me be a bit transparent here. That figure is of novel drugs approved, which means that the drugs were completely brand-spankin' new. This would be in contrast to repurposed drugs, which are drugs that have already been developed by can now be used for another disease. But even if that number of drugs were in the hundreds, it is still abysmally low relative to the amount of money that we put into research. What gives?

Have you ever volunteered for a walk to help fund medical research? Maybe you've donated some money here and there to foundations promising to accelerate research. If you've been in either of those scenarios, I'm sure you've felt frustrated that it seems like the amount of energy and money that we spend trying to cure diseases usually results in very limited results.

In the end, it boils down to one thing: it's really really really really really really x 1000 difficult to move an idea found in the laboratory to the clinic. So difficult in fact, that my institute was founded on the very premise of solving this as a systematic problem. Once we can solve this problem, we will be able to utilize the vast wealth of basic research that scientists around the world have spent their entire careers on. As the Isaac Newton once said, "If I have seen further it is by standing on the shoulders of giants".

Before I let you go, I'm going to spoil the ending a little. Despite the sort of dreary tone this blog post holds, I am infinitely optimistic that we will only continue to improve the way in which we discovery new drugs and develop new medical interventions. Costs will go down, precision will go up, and most importantly, the quality of life for sick patients around the world will soar. But before we can cross the quagmire that is translation, we're going to need some supplies. What makes translation so difficult? Where do we need to improve on? This is what I'll be discussing in the next few blog posts, so stay tuned for more!