Groundbreaking gene therapy for cancer approved for use in US – but it's expensive
The FDA has just approved Kymriah, a new form of genetically modified T-cell immunotherapy for treating young patients with a type of blood and bone marrow cancer. This is the first gene therapy of its kind to be approved for use in the United States, but the treatment is not without controversy as it has the potential for severe side effects as well as an extraordinarily prohibitive price tag.
It's not hyperbolic to say that the development of CAR-T immunotherapy has been a revolution in cancer research over the past few years. The therapy involves harvesting a patient's T-cells and then genetically modifying them to hold potent molecules called chimeric antigen receptors (CARs). These CAR-T cells are then reintroduced into the patient and act as cancer hunters, targeting and killing specific tumor cells.
Over the last few years, CAR-T cell research has exploded, and there are currently almost 300 clinical trials underway experimenting with the treatment. Many previous trials have offered stunning results, with sustained remissions in anywhere from 60 to 90 percent of patients.
But the treatment is not without significant risk. The engineered T-cells can lead to what is called cytokine release syndrome, a life-threatening condition that is triggered by an overactive immune system. Several recent patient deaths in CAR-T clinical trials have raised serious questions over the safety of the treatment.
The FDA has recognized these dangers in approving the Kymriah treatment, noting that the approval is only directed at patients that have not responded to other, more common, initial treatments. Despite the potential of major side effects, clinical trials of Kymriah have displayed overall remission rates of up to 83 percent.
The other big controversy surrounding CAR-T therapy is the significant cost. These kinds of personalized therapies are currently incredibly labor-intensive to produce. Novartis, the company behind Kymriah, claims it takes around 22 days to produce an individual treatment for a patient.
This of course results in a very expensive drug price. In the case of Kymriah the initial cost is being quoted at US$475,000 per treatment. While this is less than initial estimates of over $600,000 per treatment, some are rightfully calling this price exorbitant and prohibitive.
"While Novartis' decision to set a price at $475,000 per treatment may be seen by some as restraint, we believe it is excessive," says David Mitchell, Founder and President of Patients For Affordable Drugs. "Novartis should not get credit for bringing a $475,000 drug to market and claiming they could have charged people a lot more."
The complexity of the treatment, involving the harvesting the patient cells and the subsequent genetic editing, is undoubtedly time-consuming and labor-intensive, so it remains to be seen how widely this kind of customized gene therapy could be rolled out.
CAR-T cell therapy is indisputably a revolution in cancer treatment, but much more research needs to be done before it's labeled the breakthrough we've all been waiting for. And even if we continue to get positive clinical trial results, the lingering issue of cost remains. Is this going to be a cancer cure only available to the rich?