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Three things you need to know about CAR T-cell therapy

CAR T-cell therapy
CAR T-cell therapy may transform some cancers from acute diseases with poor outcomes to chronic illnesses that may be managed with more positive results.

Twenty years ago, the U.S. Food and Drug Administration (FDA) ushered in a new era of cancer care when it approved imatinib (Gleevac®) to treat chronic myelogenous leukemia (CML. "It revolutionized the way physicians and scientists began to think about how to treat cancer," Louis DeGennaro, President and CEO of The Leukemia & Lymphoma Society, says in a recent USA Today article marking the approval’s anniversary. "It was the birth of precision medicine."

A decade later, the FDA approved ipilimumab (Yervoy®), the first checkpoint inhibitor to help the immune system better recognize and attack cancer, marking the beginning of the age of immunotherapy

Today, it’s chimeric antigen receptor (CAR) T-cell therapy’s turn to take center stage. In the four years since the FDA approved tisagenlecleucel (Kymriah) to treat acute lymphoblastic leukemia (ALL), the groundbreaking cancer therapy has gained momentum, with four new drug approvals to treat numerous blood cancers. Jeffrey Schriber, MD, Director of Hematologic Malignancies at Cancer Treatment Centers of America® (CTCA), Phoenix, says access to CAR T-cell therapy may transform some cancers from acute diseases with poor outcomes to chronic illnesses that may be managed with more positive results.

“It’s a great time to be someone who studies and treats patients with these therapies,” he says. “I’m so gratified that there are options to help these patients.”

Dr. Schriber likens CAR T-cell therapy to using a code inserted into the human body to clear out cancer cells. For the patient, it starts with a blood draw, after which white blood cells are captured through a process called leukapheresis. The cells are sent to a laboratory, where T-cells (white blood cells that are integral to the immune system) are reengineered to enable them to fight against the cancer, then infused back into the patient.

CAR T-cell therapy may be an appropriate treatment regimen for some patients diagnosed with several types of blood cancers, including:

  • Acute lymphoblastic leukemia
  • Multiple myeloma
  • Types of non-Hodgkin lymphoma, such as:
    • Mantle cell lymphoma
    • Diffuse large B-cell lymphoma
    • Follicular lymphoma
    • Mediastinal large B-cell lymphoma
    • High-grade B-cell lymphoma

And yet, for all their promise, CAR T-cell therapies have limitations and challenges: They currently are only approved for liquid cancers, they’re very expensive, and some patients may experience severe side effects.

Still, CAR T-cell therapy offers hope for many patients with cancers that once were more difficult to treat. Here are three things cancer patients and their families need to know about this innovative therapy:

Only five CAR T-cell therapies have been approved so far

While dozens of targeted therapy and chemotherapy drugs have been approved to treat cancer, the FDA has approved five CAR T-cell therapies:

  • Tisagenlecleucel (Kymriah®), which the FDA approved in August 2017, is used to treat patients 25 and younger with B-cell precursor acute lymphoblastic leukemia that has relapsed or is resistant to treatment.
  • Axicabtagene ciloleucel (Yescarta®), which the FDA approved in October 2017 for some adults with certain types of large B-cell lymphoma who have relapsed or haven’t responded to other treatments. Large B-cell lymphoma is the most common type of non-Hodgkin lymphoma (NHL) in adults, according to the Lymphoma Research Foundation. Approximately 72,000 new cases of NHL are diagnosed each year, and large B-cell lymphoma makes up about one in three of these cases.
  • Brexucabtagene autoleucel (Tecartus), which the FDA approved in July 2020 for patients with mantle cell lymphoma (MCL) that doesn’t respond to other treatments or has recurred. A rare, fast-growing cancer of the blood, MCL has typically spread by the time a patient is diagnosed.
  • Lisocabtagene maraleucel (Breyanzi), which the FDA approved in February, is used to treat adult patients with certain types of large B-cell lymphoma.
  • Idecabtagene vicleucel (Abecma®), which the FDA approved in March to treat adults with multiple myeloma, was the first CAR T-cell therapy approved to treat multiple myeloma.

CAR T-cell therapy may cause serious side effects

Patients should know that they may experience serious side effects in response to this therapy. One patient described the experience with cytokine release syndrome, also called a cytokine storm or cytostorm, as “the worst flu of [my] life.” A cytokine storm occurs when the body releases too many cytokines, which are the small proteins powering the growth and activity of immune cells and blood cells. A patient may experience chills, fever, low blood pressure and even death, though the latter is rare.  

In rare cases, patients may experience neurologic effects called immune effector cell-associated neurotoxicity syndrome (ICANS). It can be a scary experience, says Dr. Schriber. The patient may not even recognize family or friends. It’s not clear why the syndrome occurs, but steroids may be used to treat it.

In recognition of the side effects experienced by some patients who receive CAR T-cell therapy, researchers at the National Cancer Institute made subtle changes to the CAR T-cell design. A January 2020 study published in Nature Medicine revealed that the new therapy caused far fewer neurologic side effects and worked just as well as the original therapy.

CAR T-cell therapies are expensive

Yescarta and Tecartus each costs $373,000, while Kymriah comes with a price tag of $475,000. But the total cost of care if a patient suffers from severe side effects may top $1 million.

The federal government and states that administer Medicaid programs are stepping in to help. The U.S. Centers for Medicare & Medicaid Services, which oversees programs such as Medicare, Medicaid and the Children’s Health Insurance Program, announced in August 2019 that it would cover patients at facilities that use the Kymriah and Yescarta products and agree to adhere to strict protocols.

“This therapy is changing the face of cancer,” says Dr. Schriber, who stresses that, due to the complexity involved, patients should seek out a facility that has deep expertise in treating cancer patients with CAR T-cell therapy.

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