June 22, 2023 – CAR T-cell therapy is a relatively new and extremely effective treatment for blood cancers such as leukemia. That is, if you can get it. For many, the drugs are too expensive to afford without insurance coverage, and treatment requires access to the few hospitals that offer the therapy.
Barriers are especially unequal because the drugs are now in “the frontline setting” of successful treatment options, said Sairah Ahmed, MD, a specialist in lymphoma and myeloma at the MD Anderson Cancer Center at the University of Texas.
CAR T cells are described by the National Cancer Institute as a "living drug," where a patient's own immune system T cells are collected and reengineered in a lab to bind to cancer cells and kill them. Large numbers of the cells are created and then infused back into the patient. The infusion process takes only an hour, prior to which the patient receives chemotherapy to weaken their immune system to prepare for the intake and proliferation of CAR T cells.
Ahmed – who directs the CAR T-cell therapy program at MD Anderson – said each CAR T-cell product can cost $500,000 out of pocket without insurance or Medicare. Other costs include the necessary chemotherapy, along with the burden of potential side effects.
There are programs to help, though. According to the MD Anderson website, patients can avoid unnecessary bills through authorization from their insurance company before getting a scan. In addition, for patients who are U.S. citizens and a legal residents Texas, and who fit into the low-income or limited financial assets category, MD Anderson provides uncompensated care. In fiscal year 2021, MD Anderson provided $317.5 million in such care for more than 77,000 patients.
Only around 100 cancer facilities in the United States are equipped to use the treatment, making access and travel costs challenging for many patients. A CAR T therapy patient would also have to stay in the CAR T-cell center after they have the cells infused.
“There may be some kind of resources for certain groups to be able to mitigate some of that cost,” Ahmed said. “But at the end of the day, it is the patient’s family who’s picking that up.”
“I think there are multiple barriers, socio-economically. And I think there are some centers that have the resources to help patients with some of those costs,” she said. “But certainly, even in the best kind of resource-rich environment, we’re not able to completely mitigate that cost to a patient.”
In 2015, MD Anderson launched its first CAR T-cell therapy clinical trial and cared for hundreds of patients. The FDA approved CAR T-cell therapy for leukemia in 2017 with the first product called Kymriah, from the drug company Novartis. Since then, the FDA has approved five other CAR T therapies, all designed to treat blood cancers, from different forms of leukemia to lymphomas and, most recently, multiple myeloma.
Jeremiah Bergeron, a CAR T-cell therapy nurse, said the success rate for patients achieving remission is 60%. The side effects, which can be severe, range from fever to neurological changes. In many cases, he said, the patient can have cytokine release syndrome, when the reengineered cells infused into the patient’s body attack their own cells, potentially causing fever, nausea, headaches, a rash, a rapid heartbeat, low blood pressure, and trouble breathing.
“We do [take] conservative measures, but if it starts to [cause] shortness of breath, we will put you on oxygen. We will give you medication so that it can be used to slow down CAR T,” Bergeron said.
Initially, a patient would have to go through two rounds of more traditional cancer therapy before being approved for CAR T therapy. But in the last 2 years, Ahmed said, a patient who has only had one previous treatment can consider CAR T-cell therapy treatment.
“For patients who have disease that relapsed within 12 months, CAR T-cell therapy is the preferred choice of treatment, and it is a curative intent treatment” she said. Ongoing clinical trials using CAR T therapy as the first treatment for large-cell lymphoma have also widened the populations of eligible patients.
Various things determine the side effects and negative reactions of patients using CAR T therapy. Ahmed said those include the patient's age and their health before getting the therapy.
MD Anderson advanced practice nurse Sherry Adkins created an app called CARTOX that works to grade how severe a patient’s side effects are and link that to the best treatment.
Ahmed said doctors look at various factors and make nuanced recommendations for patients based on their risks. "So potentially finding ways to decrease toxicity is a way forward because it looks like the treatment is still really efficacious: It works well,” she said. "We just have to kind of make it work without having so many side effects.”
“You want to have something that is right in front of your face and be able to quickly plug in what the symptoms are and then what you should do,” Ahmed said. “So that’s really instrumental.”
She thinks there are several potential avenues for refining CAR T-cell therapy in the future, including research on targeting other antigens, using one patient’s T cells to help another, and sequencing CAR T with chemotherapy or other treatments.
“I think there's still a lot of room for innovation, and for kind of next steps,” Ahmed said. “But you know, this is a really exciting time to be a cellular therapy doctor right now.”