New cell-based immunotherapy drugs are changing the way certain cancers are treated at a small number of FDA-approved centers, with the possibility of becoming a first-line therapy.
A type of immunotherapy called Chimeric Antigen Receptor (CAR) T-cell therapy, or CAR T, has the potential to rapidly change cancer treatment.
But for now, it's limited to two types of cancers—certain types of leukemia and lymphoma—and is available at a limited number of FDA-approved treatment centers.
Yet some experts think it has the potential to replace chemotherapy and stem-cell transplants altogether, which would dramatically change how cancer is treated, and thus, how cancer centers operate.
In CAR T therapy, doctors extract and modify a patient's T-cells so they produce a protein that recognizes another protein on the surface of cancer cells, allowing the T-cells to attack cancer cells.
City of Hope, a Duarte, California–based research and treatment hospital, is one of 49 comprehensive cancer centers as recognized by the National Cancer Institute, and is one of only a few centers approved by the FDA to offer the CAR T drugs Kymriah and Yescarta.
Based on interviews with Harlan Levine, MD, president of strategy and business ventures with City of Hope, and Michael Caligiuri, MD, president of City of Hope National Medical Center, here are four factors that will play into how CAR T therapy could affect the standard of cancer treatment.
1. Using a Patient's Own Immune System
"In a broad context of using a person's immune system to fight cancer, it's cell-based therapy, and we've been doing that for multiple decades," says Levine.
"CAR T therapy is taking antibodies that recognize the tumor antigen and hooking them up to a virus, which infects the T-cell, and then infusing those T-cells back in the patient," says Caligiuri. "You get a twofer: a T-cell that knows where to go and can kill a multidrug-resistant tumor as well as a virgin tumor. This takes away the shield and it comes in after other therapies haven't worked."
2. High Treatment Prices Will Come Down
"Prices for treatment are in the seven-figure range, but over time, prices really drop when you consider the stuff you're avoiding having to do," says Caligiuri, referring to treatments that may not be necessary anymore. "As the safety profile improves, the more patients you treat, the more comfortable you get with the anticipated and unanticipated side effects, and that ultimately improves outcomes."
"As we get more experience, we'll be more comfortable with the treatment and its role in the overall care of the patient," says Levine. "We talk a lot about value-based care these days. When we talk about value the tendency is to go to price, but with a patient, it's outcomes."
"As we learn more about CAR T therapy, we may move it up earlier in the treatment protocol to become a first line of treatment. The instinct with other conditions, like hypertension or diabetes, is what's called step therapy. That makes sense for conditions where you have time to find the right regimen, but [with] cancer, your best chance of a cure is your first chance," says Levine.
3. Potential for Expansion Limited
"This is a very expensive and highly technological treatment," says Caligiuri. "Are health systems willing to make the appropriate investments to do this really well? This won't be needed at every hospital. For the foreseeable future, it should be done at a center of excellence and, from the research point of view, it makes sense to pursue this in an academic environment.
"Leading cancer institutions will continue to invest in this area as part of a broader advance in oncology, but this is an early form of gene therapy. Until it becomes more commoditized, it won't spread far beyond centers of excellence. Only tens of centers are now getting product sent to them from the pharma companies," says Caligiuri.
"People underestimate the sophistication of this therapy," says Levine. "It involves not only extraction of cells and reengineering with the antibody to make it effective, but also the care and treatment of the patient. This is a safe place to come because of our 40-year experience with team-based care. If we move too fast, we'll end up with complications that are avoidable, and may undermine progress we're trying to make in the field."
4. Changing Standards of Care?
"Before this is exported, there needs to be an understanding of the breadth and depth of the side effects, some of which are fatal," says Caligiuri. "I would envision colleagues coming to City of Hope and other approved centers to learn how to manage [side effects] before doing this in their own institution."
"The treatments to date have shown a remarkable curative potential, which is an important part of value-based care when you have a drug that's really remarkable," says Levine. "It will go broader, and the fraction of patients who are cured will increase as we move this further toward front-of-the-line therapy."
"Over time, the sky's the limit for a multidisciplinary approach," says Caligiuri. "We have 14 active trials right now, but it's difficult to say when other such CAR T therapies will be considered so efficacious that they will be part of the paradigm. It's contingent upon facilities to grow the virus, to grow cells, and teams that can handle the volumes, as well as funding sources."
Philip Betbeze is the senior leadership editor at HealthLeaders.