A frontrunner seems to have finally emerged in the race to bring the first CAR-T cell therapy to market. For several months the two chief competitors and their drug candidates have been Novartis’ tisagenlecleucel-T and Kite Pharma’s axicabtagene ciloleucel, both developed for treating cancers of the blood. However, earlier this month a meeting of the FDA’s Oncologic Drugs Advisory Committee was convened to discuss Novartis’ application. The outcome of the meeting was a unanimous vote that Novartis’ therapy merited approval. With an overwhelmingly positive response by the Committee, it would appear that the very first CAR-T cell therapy is on the verge of being approved by the FDA and offered widely as a cancer treatment.
This milestone is surely an enormous one for next-generation cancer treatment and personalized medicine, even though the promise of CAR-T cell therapy has been known for some time now. In multiple clinical trials CAR-T cell-based cancer therapies have demonstrated an astounding ability to rein in even the most aggressive varieties of blood cancer, giving hope to patients for whom traditional treatments like chemotherapy and radiation has failed. A trial of Novartis’ tisagenlecleucel-T revealed that, three months after administration of the therapy, 82% of patients showed complete remission or complete remission with incomplete blood count. A trial of Kite Pharma’s axicabtagene ciloleucel left 36% of patients in complete response—meaning that there was no evidence of remaining cancer in the body—six months after one round of treatment.
But while the past few months may have felt like a sprint for the finish line, it’s important to remember that the race for CAR-T cell therapy approval has been a marathon that is, in fact, still ongoing in many ways.
The long road to CAR-T cell therapy
Although CAR-T cells may seem a relatively recent entrant to the world of cancer therapy, the drug candidates that have been submitted for FDA approval actually represent the product of nearly 40 years of research. Thus, only very recently in their long history, have CAR-T cells shown the potential to break out of the realm of experimental treatment and into the market for approved therapies.
The idea for chimeric antigen receptors T cells, or CAR-T cells, has been in the works since the 1980s when immunologist Zelig Eshhar of Israel’s Weizmann Institute of Science developed the concept of genetically modifying the immune system’s T cells in ways that would cause them to identify and destroy cancerous cells.
While the earliest attempted CAR-T cells were not very effective at mounting an immune response against tumor cells, successive attempts have shown an increasing level of intricacy and effectiveness. Today’s CAR-T cells are designed not only with anti-tumor activity in mind, but also persistence and memory of the therapeutic T cells as well as control over and prevention of toxicity. Even as Novartis’ therapy inches closer to FDA approval, other companies and research groups are developing a diverse array of sophisticated CAR-T cell technologies that together have the potential to completely change the face of cancer treatment.
Thus, even though Novartis’ product seems to be situated to reach the market first, the best outcome we could hope for is for all racers to reach the finish line.
With a variety of cancers being addressed and a number of different tools being built into CAR-T cells across the industry landscape, a higher number of successful players means a greater number of options for treating the disease. Novartis and Juno Therapeutics, for example, are both developing products that will be used to treat leukemia, a cancer of the leukocytes. Kite Pharma’s candidate is aimed at treating non-Hodgkin lymphoma, a cancer of the white blood cells.
Other groups are reaching beyond blood cancers and tackling the trickier hurdle of solid tumors, including the recent collaboration between Intrexon, ZIOPHARM Oncology, and Merck. Cell Design Labs, the exclusive licensee of essential CAR-T cell patents from UCSF, is advancing a line of therapy known as CellBot™ that allows for more complex control over a patient treatment.
With so many innovators marching their CAR-T cell therapies toward the market, Novartis’ imminent approval is not only a win in its own right but also a signal that a new and exciting era of cancer treatment is forthcoming.
Be sure to join us at SynBioBeta SF 2017 in October where you will hear more about developments such as these during the Health and Drug Discovery Session.
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