June 22, 2023 — CAR-T cell therapy is a comparatively recent and intensely effective treatment for blood cancers akin to leukemia. That is, for those who can get it. For many, the drugs are too expensive without insurance coverage, and treatment requires access to the few hospitals that supply the therapy.
The hurdles are particularly unequal since the drugs are currently among the many first successful treatment options, says Dr. Sairah Ahmed, a lymphoma and myeloma specialist on the University of Texas MD Anderson Cancer Center.
CAR-T cells are described by the National Cancer Institute as “living drugs.” They involve collecting the T cells of a patient's immune system and modifying them within the laboratory to bind to and kill cancer cells. Large quantities of those cells are produced after which infused into the patient. The infusion process takes just an hour. Beforehand, the patient receives chemotherapy to weaken their immune system and prepare it to receive and multiply the CAR-T cells.
Ahmed – who leads the CAR T cell therapy program at MD Anderson – said every CAR T-cell products can cost $500,000 out of pocket without insurance or Medicare. Additional costs include the crucial Chemotherapy, together with the burden of possible negative effects.
However, there are programs that may also help. According to the MD Anderson websitePatients can avoid unnecessary bills by getting approval from their insurance company before a scan. In addition, MD Anderson offers patients who're U.S. residents and Texas residents and fall into the category of low-income or limited-asset individuals offers free care. In Financial year 2021MD Anderson provided $317.5 million for such treatments for greater than 77,000 patients.
Only about 100 cancer clinics within the US are equipped to supply the treatment, making access and travel costs difficult for a lot of patients. A CAR-T therapy patient would also should stay on the CAR-T cell center after the cells have been infused.
“There may be resources available for certain groups to help offset some of these costs,” Ahmed said. “But ultimately, it is the patient's family that has to bear these costs.”
“I think there are several socioeconomic barriers. And I think there are some centers that have the resources to help patients with some of those costs,” she said. “But even in the best resource-rich setting, we certainly don't have the ability to completely mitigate those costs for a patient.”
In 2015, MD Anderson launched its first clinical trial of CAR-T cell therapy and treated lots of of patients. The FDA approved CAR-T cell therapy for leukemia in 2017 with the primary product, called Kymriah, from pharmaceutical company Novartis. Since then, the FDA has approved five more CAR-T therapies, all designed to treat blood cancers, from various types of leukemia to lymphoma and, most recently, multiple myeloma.
Jeremiah Bergeron, a CAR T-cell therapy nurse practitioner, said the success rate for patients achieving remission is 60%. Side effects, which will be severe, range from fever to neurological changes. In many cases, he said, the patient can develop cytokine release syndrome when the newly engineered cells infused into the patient's body attack their very own cells, potentially causing fever, nausea, headache, rash, rapid heartbeat, low blood pressure and difficulty respiratory.
“We do [take] conservative measures, but when it starts to [cause] If you're short of breath, we'll give you oxygen. We'll give you medications that can slow down CAR T,” Bergeron said.
“Exciting times”
Initially, a patient would have to undergo two rounds of more traditional cancer therapy before being approved for CAR-T therapy. But for the past two years, Ahmed said, a patient who has received only one prior treatment can also consider CAR-T cell therapy.
“In patients whose disease has relapsed inside 12 months, CAR-T cell therapy is the popular treatment modality since it is a curative-intent treatment,” she said. Ongoing clinical trials of CAR-T therapy as a first-line treatment for large cell lymphoma have also expanded the pool of eligible patients.
Several factors determine the side effects and adverse reactions experienced by patients taking CAR-T therapy. Ahmed said these include the patient's age and health status prior to therapy.
Sherry Adkins, a nurse at MD Anderson, developed an app called CARTOX that rates the severity of a patient's side effects and links them to the best treatment.
Ahmed said doctors consider various factors and give patients differentiated recommendations based on their risks. “So perhaps finding ways to cut back toxicity is a way forward, since it looks just like the treatment remains to be very effective: It works well,” she said. “We just have to in some way make it work without having so many negative effects.”
“You wish to have something right in front of you and quickly type within the symptoms after which see what it's best to do,” Ahmed said. “It's really helpful.”
She believes there are several potential approaches to improving CAR T cell therapy in the future, including exploring other antigens, using one patient's T cells to help another, and sequencing CAR T with chemotherapy or other treatments.
“I believe there's still loads of room for innovation and for further steps,” Ahmed said. “But you recognize, it's a extremely exciting time to be a cell therapy doctor immediately.”