When a new type of immunotherapy called CAR T-cell therapy was approved by the US Food and Drug Administration in 2017, it gave patients who had exhausted other treatment options hope where there was none before.
The treatment, which genetically alters a patient's own immune cells to fight cancer, is intended for patients who have not responded to standard therapies, such as chemotherapy or bone marrow transplantation, or whose cancer has returned after receiving standard treatments.
The therapy is currently approved by the FDA to treat some blood cancers, including multiple myeloma, certain subtypes of non-Hodgkin's lymphomas, and the most common childhood cancer: acute lymphoblastic leukemia (ALL). More recently, the FDA has approved the first cell therapy approaches for metastatic melanoma and a rare type of sarcoma.
"There is no question that using a patient's own immune system to fight cancer was one of the major medical breakthroughs of the 21st century," said Dr. Ran Reshef, director of the cellular therapy program at NewYork-Presbyterian/Columbia University Irving Medical Center and a professor of medicine at Columbia's Vagelos College of Physicians and Surgeons. "CAR T-cell therapy addresses the major challenge with immunotherapy by finding a way to harness the immune system by either genetic modification or other methods so it can do a better job of fighting cancer."
NewYork Presbyterian's Health Matters spoke with Reshef about what to expect during CAR T-cell therapy, its potential benefits and side effects, and what questions to ask your doctor before receiving treatment.
What is CAR T-cell therapy?
CAR T-cell therapy is a type of immunotherapy that has had a tremendous impact on how we treat certain types of cancer and holds great promise for treating other cancer types and even autoimmune diseases.
It involves collecting types of white blood cells like lymphocytes, also called T cells, from cancer patients, and genetically modifying them to produce new CAR (chimeric antigen receptor) proteins that can detect specific antigens on cancer cells. I like to think of it as arming T cells to be able to recognize cancer. Once these newly armed T cells are manufactured, we infuse them back into the same patient so these cells can start attacking cancer cells. It's a living drug, where the new T cells not only attack the cancer but also continue to multiply and persist in the patient.
Why is CAR T-cell therapy considered to be so promising?
These re-engineered T cells are tremendously potent. The term serial killers is the best way to describe them. These modified T cells kill one cancer cell, then move on to kill the next, and the next, to the very last one. In some cases, these cells may remain in the body for years and offer protection against cancer recurrence. As a result, some patients who were once considered incurable have gone into complete remission now known to last more than a decade and possibly indefinitely.
What's the process like for patients?
The first step is the cell collection, where a medical device called an apheresis machine collects the patient's blood, skims off the lymphocytes, and then returns the blood to the patient. The T cells are then shipped to the pharmaceutical company's manufacturing facility to be genetically modified and multiplied.
Two to four weeks later, the new T cells are ready for infusion into the patient, who will receive a form of preparatory chemotherapy. This is meant to reduce their own lymphocytes and make room for the new "super potent" T cells to establish themselves. The patient will be closely monitored for side effects for several weeks after the treatment. If all goes well, the T cells will attack the cancer cells successfully, and the patient will achieve remission.
What are the possible side effects?
A small minority of patients experience serious side effects that require urgent management, occasionally in intensive care. This occurs when the new T cells cause an aggressive inflammatory response in the patient. That's the reason that CAR T cells are administered either inpatient or outpatient with daily monitoring during the first seven to 10 days. One side effect can be cytokine release syndrome (CRS), which causes:
- Fever
- Malaise and fatigue
- Shortness of breath
- A drop in blood pressure and oxygenation, among other symptoms
The inflammatory response can also affect the nervous system and cause a complication called immune effector cell-associated neurotoxicity syndrome (ICANS), which can result in:
- Tremors
- Headaches
- Drowsiness
- Difficulty speaking
- Disorientation
- Seizures and brain swelling in rare and severe cases
This is why CAR T-cell therapy is administered only in specialized centers. The good news is that the side effects occur within days from the infusion and, given that CAR T cells are designed to be a one-time treatment, the overwhelming majority of patients completely recover from these side effects and can return to their regular activities within a few weeks.
Where do you see CAR T-cell therapy going next?
We are still learning the potential of CAR T-cell therapy, and clinical trials are underway using it to treat a number of other cancers, including solid tumors, as well as autoimmune diseases like lupus, scleroderma, and multiple sclerosis.
What advancements in cell therapy are you particularly excited about?
At NewYork-Presbyterian and Columbia, we have a highly developed program with a decade of experience in administering CAR T-cells and other cell therapies. Beyond the FDA-approved products, we have a large portfolio of clinical trials that address the needs of patients, for whom FDA-approved products are not yet available. These trials include experimental treatments for various solid tumors and autoimmune diseases, and, in the future, we anticipate expanding our trials for additional conditions.
Learn more about Cell Therapy: Diagnosis & Treatment | NewYork-Presbyterian.
This sponsored post was supplied by NewYork-Presbyterian.
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