A recent FDA advisory panel recommended the approval of 2 new agents in a novel class of cholesterol lowering drugs known as PCSK-9 inhibitors. What makes this remarkable is that these drugs illustrate all the promise and pitfalls of modern pharmaceutical development.
First, a little science. The target of the new drugs – a protein named proprotein convertase subtilisin/kexin type 9 (PCSK-9) – was discovered in 2001. Two years later, investigators reported that “gain-of-function” mutations in the gene that codes for PCSK-9 were associated with familial hypercholesterolemia and high rates of atherosclerotic vascular disease. Mutations of the gene that led to reductions in the function of PCSK-9 were associated with low LDL-cholesterol levels, and a lower incidence of vascular disease. That made the compelling case that PCSK-9 had a counter-regulatory function in LDL-cholesterol metabolism, so that interfering with its function would lead to lower cholesterol levels.
Continue reading The New Paradigm
I have written previously about the potential impact of mobile apps and ubiquitous computing on health and healthcare delivery, but I admit I did not see this one coming. The current issue of The New England Journal of Medicine has a report from a research group in Sweden that developed a system – and tested it in a randomized controlled trial – to use smartphones to alert CPR-trained bystanders when there was a nearby cardiac arrest. This figure from the paper describes how it works:
Continue reading Crowdsourcing CPR
A recent piece in the New York Times profiled a young man with a remarkable medical history, and an equally remarkable approach to sharing it. I think it raises some profound issues regarding the self-monitoring movement and the “ownership” of patients’ health information, both of which have the potential to change our traditional practices in a big way.
The guy – Steven Keating – is not your average Joe. He is a graduate student at MIT who trained as a mechanical engineer and is working in the cutting-edge MIT Media Lab. He also had a brain tumor the size of a tennis ball. His website hosts all of his medical records, including his pre- and post-op brain scans and, believe it or not, a video of his tumor resection surgery.
Continue reading Whose Record Is It?
If you were the right age to have been watching television in the mid-1970s, you probably remember “The 6 Million Dollar Man.” The show was about an astronaut who is critically injured in a test-mission gone bad, and is “rebuilt” with bionic (nuclear powered!) limbs and sensors to be “better than he was.” The campy intro, complete with scenes from the operating room, is, of course available on YouTube. Continue reading Better than new
I felt a little sad when I read the “perspective” piece in the New England Journal of Medicine this week about the introduction of point-of-care ultrasound in medical education. Continue reading Death of the stethoscope?
I recently read two books that both have a powerful “wow” factor, which has kept them on my mind. In thinking about them some more, it also seems that there are some connections to medicine that could also be quite profound. Continue reading Globalization and Extinction
For years, I have been hearing about how new technology will transform every day clinical practice, and I have been looking forward to it. Who wouldn’t want to be able to understand better the basis of human disease based on the “new taxonomy” of precision medicine? Or offer personalized therapeutics based on full genome sequencing? Or have the ability to predict better which patient will decompensate based on advanced analytics? And yet… most of us are pretty much doing what we have always done – diagnosing disease based on signs and symptoms, prescribing drugs based on their likelihood of efficacy in a population that more or less looks like the patient in front of us, and waiting for patients to decompensate and then reacting to it. Yes, we are doing all this while using (struggling with?) an EMR, but still, the basics are all pretty much the same. Continue reading The Future Arrived for Me Last Week
I think I am like many practicing physicians in my “love-hate” relationship with clinical practice guidelines. On the one hand, it is often helpful to look up a set of evidence-based recommendations on a particular clinical issue, and I feel particularly fortunate that the American College of Cardiology and the American Heart Association have collaborated to produce high quality guidelines on a wide-range of subjects relevant to my practice. On the other hand, I am well aware of the shortcomings of practice guidelines, including the limitations of the underlying evidence base, the challenge of synthesizing the available evidence into guidelines, and the often limited applicability of recommendations to clinical practice. Continue reading Practice Guideline Overload