In the third of a front page series in the The New York Times published July 31st – August 2nd, entitled “Setting the Body’s ‘Serial Killers’ Loose on Cancer,” science writer Andrew Pollack reported on Dr. Steven Rosenberg at the National Cancer Institute, a research physician who has committed half a century to the promise of immunotherapy. The article also highlighted the CAR-T immunotherapy treatment pioneered by Scientific Advisory Council members Dr. Carl June at the University of Pennsylvania and Dr. Michel Sadelain at Memorial Sloan-Kettering Cancer Center and referenced ACGT co-founders Edward and Barbara Netter.
ACGT has funded the research into CAR-T therapy from inception and we have seen incredible progress in refining this science as a whole new way of treating cancer, and the vast majority of patients enrolled in trials are experiencing long-lasting remissions.
What exactly is CAR-T cell therapy and how does it work?
Following is a simple summary of a complex process, and the Times printed a very useful diagram in Part I.
T-cells are a type of white blood cell whose sole function is to identify and destroy cells that threaten the body’s health. They are also known as killer cells, an apt description. Each T-cell has claw-like receptors that can lock onto antigens – protein fragments on the surface of abnormal cells. In a very simple blood draw, a patient’s own cells are extracted and then modified to target cancer cells before reinserting into the patient to awaken the immune system. The great beauty of this science is that those T-cells now know exactly where to attack. They go after the cancer – and only the cancer – so side effects are generally few, and mild, nothing like the toxic effects of chemotherapy drugs. And the treatment is usually one or two days, not weeks or months.
Of course there is a snag in the approach – nothing in medical science happens overnight. Cancer cells have what are called Checkpoint Inhibitors that defend against any interference, even from the immune system. So T-cells have to bypass those inhibitors and that’s the more delicate part of the process. When this doesn’t work, side effects can be severe – in fact, the immune system can turn on itself, known as an autoimmune response, which can be severe, and the treatment may not work. The future of CAR-T relies on shutting down checkpoints and there is a lot of optimism that this will be accomplished sooner than later.
CAR-T has been proven extremely effective with leukemia and lymphomas and other blood cancers, and some pediatric types as well. Further study and human trials are necessary to improve the effectiveness of the treatment on solid cancers, and ACGT scientists are working to make this happen.
Promise of Cell and Gene Therapies for Cancer
The real future, and the great promise of cell therapies like CAR-T, is to match the cancer profile to the treatment design and tremendous progress is being made toward this end. In this way, the checkpoints on each cancer in each individual patient will be more easily identified, and neutralized, and the treatment will be more reliably effective.
Immunotherapy is the future and is happening right now and ACGT is still the only non-profit in the nation dedicated exclusively to cell and gene therapies for cancer. Stay tuned!