Time to really usher in medicine for a new era

A “new era” in medicine does not come along very often. We had one, perhaps, in 1854 when John Snow effectively invented the practical applications of epidemiology by removing the handle from London’s Broad Street water pump. We had one about 50 years prior when Edward Jenner discovered the prevention of smallpox with vaccination. We had one when the value to public health of basic sanitation was first sorted out; we had one when micronutrients were discovered to cure and prevent various deficiency syndromes; and we had one when Alexander Fleming and a bit of serendipity combined to discover penicillin, and usher in the antibiotic age.

And we had one on July 13, 2017, at least according to the New York Times, which used just that language to report the provisional approval by a FDA panel of a breakthrough therapy for leukemia involving genetic engineering. The approval is provisional because the recommendations of FDA expert panels, unanimously in favor of the new “drug” called tisagenlecleucel (good luck) in this case, require formal action by the agency. Such action, however, rarely departs from panel recommendations, and certainly not in the case of a unanimous decision.

The new therapy is perhaps the epitome of “personalized” medicine. It extracts a variety of white blood cells called T cells from an individual patient, and engineers a genetic adjustment so that these T cells can recognize the patient’s own malignant B cells as foreign and attack them. The immune system is in effect reprogrammed to seek out and destroy the leukemia cells.

Predictably, the costs involved in extracting one person’s cells, reprogramming them with genetic engineering techniques, and infusing them back into that one person are potentially quite staggering. The amped-up T cells work only for their owner; there is no scalability. How ironic, and opportune, that this stunning but costly advance should arrive even as the basic mechanisms of health care coverage roil our Congress and our society.

Now is just the time for every parent and grandparent, whatever their political leanings, to ask: Would they want access to this product of human ingenuity if ever their beloved child faced all but certain death without it? Would they want access regardless of their ability to pay? Would they want that child forever after subject to potential unin-surability because of that pre-existing condition?

There are all sorts of ways to reduce the costs and improve the outcomes of our so-called “health care” system quite massively; denying a desperate 6-year-old life-saving access to the progress born of humanity’s relentless resourcefulness should not figure among them.

Those better ways include universal access, and universal coverage for essential, and preventive care. Those better ways include a fundamental shift from just “disease” care, to a genuine focus on “health” care, with lifestyle as the preferred medicine. Those better ways include confronting the hypocrisies of a culture that laments diseases in children, but propagates them for profit.

My particular field — lifestyle medicine — is in the vanguard of all such efforts, with attendant benefits for the planet, too, that no other branch of medicine tends even to mention. I am proud and inspired to be involved in so timely and propitious an effort. The reality, though, is that while lifestyle can often be the very best of medicine, there are times when some other medicine emphatically is. Most of us have experience with that; I certainly do. We are not obligated to choose.

Let’s use lifestyle to promote health and prevent disease as only it can; and let’s use it as we can to treat and reverse disease, too. But then let’s celebrate the stunning scientific advances that empower us to treat those who get sick anyway, as some inevitably will. Bad things can happen to people who do everything right, and well, just as gales can cause the best of ships and crews to founder. When that happens, the rest of us muster the resources for a rescue mission to save all who can be saved. Medicine, more than one kind of medicine, should be used to do the same.

I applaud the insights and toil, inspiration and perspiration that brought us tisagenlecleucel and that will bring us related advances. I call upon us as a society to do what’s necessary to honor this endowment of the human mind and ensure it is never denied to a child in peril. It’s a new era in medicine. But more importantly, it’s just another day that 52 young people will live to see.