Medicine is always evolving. But the synergy between the scientific and technological revolutions of the past decade has propelled medical knowledge into the future at an explosive speed. Diseases and drugs are now understood on a molecular level, promising the advance of personalized diagnostics and treatments. In "The Cure in the Code: How 20th Century Law Is Undermining 21st Century Medicine," Peter Huber, senior fellow at the Manhattan Institute for Policy Research, explains the possibilities and argues that public policy needs to be updated to keep up with growing capabilities. Huber spoke with U.S. News about recent discoveries and why Washington hasn't adapted. Excerpts:
How is this era of medical science different?
We can now see diseases and their causes right down to the molecular level. And that's really important because it's down in that level that drugs operate, and we now have very good tools for designing drugs precisely targeted to specific molecules. That gives us a completely new perspective on what the diseases are, whether they're spawned by our own chemistry or by infectious microbes. And it gives us a very systematic way of designing drugs to control those diseases molecule by molecule.
When did this revolution begin?
You can pick your dates, but the [Food and Drug Administration] licensed the first drug that was designed specifically to target a molecule in the late 1970s. It's much more recently, however, that we developed this explosive power to really read all the code. The sequencing of the first human genome was completed 10 years ago. The cost of technologies for reading all the genetic code has been plummeting, the power increasing very rapidly. We're getting very good at designing diagnostic sniffers, if you will. The kind of stuff we have in the pregnancy dip stick we can now do for essentially any protein that's found in our body.
What's one example of technology and science working together?
Breast cancer used to be viewed pretty much as a single disease – it's got a set of clinical symptoms, and it would be diagnosed that way. [There were] various untargeted drugs, and success rates weren't particularly high. We now know that breast cancer is at least 10 distinct diseases. They're characterized by different molecular profiles, and we've got a bunch of targeted drugs. Some breast cancers are treated with estrogen; the majority are actually treated with estrogen blockers, the exact opposite drug. One of those drugs, tamoxifen, depends on how it's metabolized by the patient's liver – and we have genetic markers on who's got the right liver to metabolize that drug. It's a precision medicine now, and you actually understand what you're doing and why you're doing it.
How close are we to personalized medicine?
We are getting better and better at precisely profiling the molecular structure of a disease. Do we have all the biomarkers for all the diseases? No, but we're close. If we find the specific molecular profile for one type of cancer, can we actually hit the particular molecules that define the different varieties of that cancer? No, [but] we're close. And that's one of the reasons I have a book out because we could have many more drugs, [and] we could be getting them out there faster.
Are existing laws hindering this process?
It's implementation that's been the challenge. The FDA is still largely stuck in trial protocols that do not let us exploit fully our ability to tailor drugs precisely to the patient's molecular profiles. The only way you can find out how a drug is going to interact with different profiles is actually to prescribe it to the patients. The FDA is still very much anchored in what are called randomized blind protocols. There's no learn-as-you-go process in the early trials, where you learn about the different ways that different patients can respond to the same drug and then refine the way you prescribe that drug. That's one obstacle.