Future of Healthcare is Personalized Medicine

(Diplomatic Courier, July 08, 2013)

By Sen. Bill Frist, M.D.

It is a common refrain that America has the best health care in the world, but our people are far from the healthiest. We spend twice as much as any other nation on health services, yet rank dismally, behind more than twenty other countries in basic health metrics like infant mortality and life expectancy. We have more MRI machines, heart transplants, new drug patents, Nobel Prize winners than any country in the world, and yet people in Greece, Israel, and Jordan live longer. How can this be?

The New York Times will tell you that the lack of a true healthcare system—preferably a nationalized and universal system—causes these failures. If only we had a true, equitable and universal system, such as Britain’s or France’s, we could skyrocket up the rankings. This is simply not true.

Data have shown that when all factors are taken into account health services only account for roughly 10 percent of determining how long we live. The two major factors are personal behavior, accounting for 40 percent, and genetics, accounting for 30 percent (social circumstances and environmental exposure round out the rest at 15 percent and 5 percent, respectively). So how do we harness the true drivers of mortality? How do we live healthier, create a more targeted and personalized approach to medicine, all while simultaneously cutting costs and eliminating waste?

The answer lies in what I call “New Medicine.” I believe that we are currently at an inflection point in medicine. We are poised to capitalize on decades of innovation, tying together disparate fields including genetics, social networks, supercomputing, the internet, stem cells, cutting edge imaging and sensors, and pharmaceuticals. These forces, unleashed in a dynamic, coordinated fashion can usher in this era of “New Medicine” that treats each patient as an individual, not the average; eliminates waste and adverse side effects; and maximizes the outcome of diagnosis and treatment for you specifically, not just for most people or the population in general.

We have been building towards this inflection point for decades with seemingly unrelated advances such as the advent of cell phones in the 1970s, the discovery of the double helix model of DNA in 1953, or the first MRI on a human being in 1977.

I have written in several forums about some of these advances, most notably pharmacogenomics and the rise of consumerism in healthcare, but today I focus on one breakthrough of incredible potential.

Stem cells hold the power to be a major pillar of the “New Medicine.” Scientists have speculated for years about the awesome potential of stem cells. Conditions as wide ranging as diabetes, spinal cord injuries, burns, limb amputations, heart disease, and neurological disorders can all likely be treated by stem cells. But what makes embryonic stem cells so special?

Embryonic stem cells are special for two reasons; first they produce exact copies forever and second they can grow into specialized tissue, which fully formed (or “adult embryonic stem cells”) human cells cannot, or at least were not thought able to. Embryonic stem cells are harvested from a five day old embryo, taking the inner cell mass of the blastocyst, generating pluripotent embryonic stem cells. These cells can be differentiated into heart cells, brain cells—basically any cell in the human body.

But there are two problems with embryonic stem cells. First is the ethical issue. It requires the destruction of embryos, the destruction of something that, left to nature, would become human life. Second, it is “non-self.” Even though these cells can become specialized into any type of cell, they do not come from you and your body knows this. But this approach has yielded remarkable scientific breakthroughs. We have cloned frogs and sheep. I had the great pleasure of visiting Dolly in Scotland, the first cloned sheep, which many may not know was actually named after Dolly Parton, the famous Tennessean entertainer. However, for reasons we don’t fully understand, humans cannot be cloned using this same method.

Thus, the real breakthrough came using a radically different approach. In 2012, the Nobel Prize for Medicine and Physiology went to Dr. Shinya Yamanaka (along with Sir John B. Gurdon). Yamanaka induced skin cells to become “Pluripotent Stem Cells” (iPS). These “iPS cells” have all the special properties of the magical stem cells. In the simplest terms, Yamanaka was able to take normal, adult skin cells and transform them, using four master genes, into pluripotent stem cells, just as capable of transforming into any cell type, but without the use, and consequent destruction of an embryo. Not only does this solve an incredibly complex moral issue, it also bypasses the issue of embryonic stem cells being foreign or “non-self” to the eventual patient or user. Doctors can simply use the patient’s own skin cells to create the iPS cells needed to treat him.

The first real application of this breakthrough is in the form of regenerative medicine. As a transplant surgeon, I have performed hundreds of operations to extend the life of patients by giving them a new heart or lung. This complex procedure meant that I would get on a plane, fly to the organ donor, remove the donor heart and fly back, transplanting it into my patient, all in a matter of hours. The patient receives years, even decades, of life with family and friends. I have many patients who I transplanted more than 25 years ago. But the heart transplant patient has traded a fatal disease for a chronic disease, which requires daily management. The heart a surgeon transplants is “non-self” and thus the body continually attempts to reject it. On top of having just undergone one of the most traumatic operations a human body can endure, the patient must then take multiple immunosuppressant medicines to keep his own body from killing his new heart. Consequently, pneumonia, or even a common cold, becomes deadly.

But this operation may well become a thing of the past, like the iron lung. In the not too distant future, we will take a patient who needs a heart transplant, perform a skin biopsy, create patient-specific iPS cells, grow them into healthy heart cells and then transplant, not a separate heart, but merely the genetically matched healthy heart cells. The body, recognizing its own genetic material, would find no cause for rejection and this operation can be done with a needle, not a bonesaw. There is also no shortage of organ donors or patients dying while waiting for just the right match.

This example represents just one of hundreds of applications. In fact, the first clinical trials for macular degeneration (blindness) will occur this year.

The second application of Yamanaka’s breakthrough is the huge potential for drug discovery. Take Alzheimer’s Disease for an example. Up to now, all drugs have failed. But what if we could test a new drug specifically on your brain cells to see if it works. Using this technology we can. We simply take adult skin cells, revert them to iPS cells in a test tube and then again into brain cells and then test their response to a potential drug in a dish. Now we can literally conduct “clinical trials in a dish.” It’s a new world.

The Gladstone Institute, where Dr. Yamanaka works, has already made cells for Alzheimer’s, Parkinson’s, Huntington’s, and cardiac disorders. With the ability to test drugs on actual cells of a specific patient, we can take the guesswork out of efficacy and toxicity of drugs. This should, and will, lead to a major rejuvenation of the pharmaceutical industry and more targeted, specific, and effective care for patients.

“New Medicine” is coming. Don’t be left behind.

Bill Frist is a transplant surgeon and former Senate Majority Leader (R-Tennessee).

This article was originally published in the special annual G8 Summit 2013 edition and The Official ICC G20 Advisory Group Publication. Published with permission.