Before I begin, I will be clear that this is a personal post and not on behalf of my current employer or any company, including those mentioned explicitly or implicitly. I have never worked with any of the companies mentioned in this post.
In a world where we incessantly muse about the future of technology and healthcare, we don’t always celebrate when that future finally arrives. Maybe we should.
We have a penchant to memorialize dates of disasters. We all know them. They become ingrained in our day-to-day conversations, and they often are remembered for generations. We do the same with dates of large military victories: half memorializing the losses that have preceded them and half celebrating the completion of a large and arduous campaign. We are even, as a society, fairly vigilant at recording the dates of successful milestones of broader technological achievements (e.g., the moon landing). In contrast, we are tremendously poor at recognizing milestones that mark a slow (or even fast) progression to new paradigms in human achievement.
On August 30th, 2017 we had such a day—and, though many noticed it, I can’t help but think that we have officially taken a giant leap towards the future of conquering cancer, and it has passed with not nearly the amount of fanfare it deserved. When it comes to the public healthcare discourse, we seem to be laser focused on the list price of novel innovative therapies (not what they actually cost or the vast amounts of time and money that have gone into making them) and far less focused on the massive leaps forward that some therapies represent—not only to healthcare, but also to individual people, their families, and more broadly humanity.
In the 1940s, chemotherapy came into its own with the first clinical use of nitrogen mustards and antifolates. Before chemotherapies ever extended a life, they already had the baggage of a dark past: nitrogen mustards were first investigated because of the lethal effects of mustard gas on solders. However, in a therapeutic setting, nitrogen mustards were mainly constrained to the treatment of blood cancers. It wasn’t until the mid/late 1960s that combination therapies, taxanes, camptothecins, platinum-based therapies, etc., were proven to treat other cancer types and started to become standards of care. We now view chemotherapies as mainstays in many cancer treatment algorithms. Of course, humanity didn’t stop there. Non-specific chemotherapies were later followed by hormonal therapies and more complex targeted therapies, including biologics and immunotherapies.
This evolution of oncology treatments has helped millions, but outcomes show that there is still a long road ahead. On August 30th, we saw the latest major leap forward when the FDA approved the first ever gene therapy in the United States. The therapy is only approved for a narrow population: a subpopulation of those with a specific bone marrow/blood cancer. Not to downplay the importance of the therapy to the applicable patient populations and their families, but this step forward is so much bigger than just this subgroup. (Nevertheless, it should be noted that this therapy is a meaningful advancement for this group, with promising clinical data and a new treatment option for a patient population wherein very few options previously existed.)
This gene therapy is the first of a new type of T-cell immunotherapy. Without getting too far into the scientific details, the therapy takes the patient’s own blood (specifically, the T-cells in that blood) and genetically modifies them to target and kill leukemia cells, based on the presence of a specific protein on the leukemia cell’s surface. The modification of the T-cells is done through inserting a gene that encodes a specific protein (a “chimeric antigen receptor” or CAR). The genetically-modified cells are called “CAR T-cells”, and they are infused back into the patient’s body.
There are known side effects of CAR T cell therapy [in this case, a boxed warning for cytokine release syndrome (CRS), neurological events, and other severe side effects, including infection, hypotension, acute kidney injury, fever, hypoxia, etc.], some of which are life threatening (though some of these can be muted through other drugs), and hospitals will be required to be specially certified prior to using it. But REMS programs and post-market observational study aside, CAR T therapies represent a new, and seemingly efficacious, tool for the treatment of cancers.
August 30th, 2017 represents a first step in CAR T cell therapy application. We’ve officially added a new tool to the belts of oncologists. In the years ahead, we’ll see more dollars devoted to the technology and bringing therapies to market (the $11 billion dollar purchase of Kite Pharma by Gilead earlier this week reinforces this). We will also hopefully overcome hurdles—the largest being that we are still likely years away from successfully treating solid tumours with CAR T cell therapies. Still, yesterday was big. It was important. It should be celebrated and remembered, not just as a milestone, but also for being the first step in what will likely be a revolution in oncology treatment.
We often use the “fight” analogy when we talk about patients and cancer, but less so when we talk about the clinical path to approving a drug or a new class of therapies… maybe that’s wrong. We remember the dates that we win big battles. This approval represents a significant first win at the end of a long, expensive, and arduous journey; as such, this date should be remembered.
In an age where more press is given to incremental improvements to the iPhone than leaps forward in oncology treatments—and healthcare dialogue is more focused on cost than innovation—we should remember the days that will change lives. They are not just moments in time that mark the beginnings of exciting trends— they represent millions of moments that will be given back to children, patients, and loved ones for decades to come.