CAR-T Cell Immunotherapy: Could it Be a Revolution in the Making?

It takes a lot to call something a revolution in medicine. One required aspect is the strategy’s basis on pivotal scientific advances or technology. Another is a biological effect that is distinct and never before seen. The most important, of course, is its impact on people’s lives — particularly, a cure for illness or a major extension of life. A revolution also implies a sweeping cataclysmic change, one that is disruptive and boldly states that things will no longer be the same.

At CURE® magazine — in recent issues and those to come — we have been covering the story of chimeric antigen receptor (CAR)-T cells from many angles and in the context of several diseases. This issue’s report focuses on the modality’s still-investigational application in multiple myeloma. An accompanying illustration explains the elegant technology and its effect on cancer in the body — almost like something out of the 1966 movie (for those who remember it) “Fantastic Voyage,” in which a team of specialists is miniaturized to enter a patient’s body to repair brain damage.

The last decade has witnessed transformative changes in multiple myeloma, with a shift away from chemotherapy-based treatment and stem cell transplants (which have, in fact, produced some long-term remissions) and toward an array of biological therapies. Although generally not curative, these treatments clearly extend remission and life. The rapid rise of immunotherapy for many diseases is fundamentally different in that it aims to use the body’s own healing powers — its immune system — in an ongoing manner, even after treatment is complete.

Cancer cells are immunologically similar to normal ones and can evolve mechanisms to evade immune surveillance. In contrast, in CAR therapy, genetic engineering generates a customized T cell that recognizes a distinct protein on cancer cells, bypassing the need for the immune system to take its usual multitude of steps to fight cancer. Also, these immune cells can stay in circulation for years, ready to recognize and attack dormant cancer cells that may re-emerge over time. In cases of myeloma, as our feature article describes, this technology is showing remarkable early results.

I have to admit that I am not quite ready to label CAR-T cells a revolution, but this highly innovative platform is certainly a candidate for this as we await its impact in many diseases — not only its long-term effects and ability to really cure cancers but also its safety record. After all, the first two CAR-T products were approved in September and October 2017.

Time will tell, but we should be grateful to the many scientists and clinicians who invented and translated this technology to patient care, as well as to the brave patients who participated in clinical trials to get us to this promising point.

DEBU TRIPATHY, M.D.

Editor-in-Chief Professor of Medicine Chair, Department of Breast Medical Oncology The University of Texas MD Anderson Cancer Center