Multiple Myeloma

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CAR T-Cell Therapy’s Current and Future Success

Dr. Nina Shah UCSF Medical Center

CAR T-Cell Therapy's Promise

  • 80% of patients have a response to CAR T-cell therapy after other treatments have failed
  • CAR T-cell therapies are generally well tolerated with some patients experiencing no side effects
  • Researchers hope the next step is to begin using this therapy in even earlier stages of treatment.

There are many different therapies for multiple myeloma including chemotherapy, radiation, treatment with corticosteroids and bone marrow transplants. Unfortunately, many times after these therapies the cancer returns. One of the newest and most promising treatments is CAR T-cell therapy, a type of therapy that works by super-charging the body’s own immune response. The treatment has received FDA approval for patients whose multiple myeloma has returned after at least four other treatments have failed.

What is CAR T-Cell Therapy?

Multiple myeloma is a cancer of the white blood cells called plasma cells. When healthy, these cells are part of the body’s immune system that help recognize and attack germs by making antibodies. CAR T-cell therapy filters patients’ blood to remove a type of immune cell called T-cells. These cells are sent to a laboratory where they are genetically engineered to become more efficient cancer killers.

“CAR T-cell therapy represents a new modality of treatment for multiple myeloma,” says Dr. Nina Shah, a hematologist with the University of California San Francisco Medical Center. “In the past we’ve used things like chemotherapy or antibodies, but this is the first live drug, using your own cells engineered to attack your myeloma.”

The Promising Response Rate

What’s exciting about this treatment is that clinical trials have shown response rates of between 80 and 100 percent. “It’s really unprecedented. This is something we had never seen before for patients who have had six or seven prior lines of therapy,” says Dr. Shah. Oncologists are hoping that CAR T-cell therapy can eventually be used not only for multiple myeloma that has returned after previous treatments—called relapsed or refractory myeloma—but earlier in treatment as more data accumulates from clinical trials.

Dr. Shah says that many patients are concerned about side effects from this new treatment, but that she’s found CAR T-cell therapies are pretty well tolerated. “I’ve even had patients who didn’t have any side effects,” she says. Currently scientists aren’t sure whether the side effects correlate with how well the treatment is working, so it’s difficult to tell patients whether their side effects—or lack of them—are a good or bad thing. “All I can say is that every patient is different, and every patient has a different course,” says Dr. Shah.

When side effects do occur, the mildest one is fatigue. “That’s very normal, and it usually resolves in the first month,” says Dr. Shah. One of the more serious side effects is called cytokine release syndrome (CRS). CRS occurs when the CAR-T cell therapy causes a large, rapid release of cytokines—small proteins that are part of the immune system—into the circulation. That release can cause a variety of reactions. The flu-like symptoms of CRS can include headache, fever, chills, scratchy throat, nausea, vomiting, diarrhea, muscle or joint pain and lack of energy. In addition, CRS can cause shortness of breath, low blood pressure, and fast heart rate. Most people who develop CRS will have mild to moderate symptoms, but sometimes CRS can be serious and even life-threatening.

Fortunately, doctors are able to control this side effect. “We used to really worry about it a lot, but now we know that it can be controlled with a drug called tocilizumab (common brand name Actemra), which starts to bring down the reaction,” explains Dr. Shah. “And we know that receiving this drug tocilizumab probably doesn’t change the outcome of your CAR T-cell therapy.”

CAR T-Cell Therapy’s Future

Data shows that the quality of life for people who have undergone CAR T-cell therapy “actually improves when we talk about pain, fatigue, and emotional and social functioning. And so whether or not you experience side effects, we hope that this therapy will improve your quality of life,” says Dr. Shah.

Considering its impressive success rate, the fact that side effects can be controlled, and data showing that patients’ quality of life improves, CAR T-cell therapy has doctors enthusiastic about using it for their patients with relapsed or refractory myeloma. “We certainly are excited to use this new tool that we have to hopefully give people a longer chance to live,” says Dr. Shah.

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Dr. Nina Shah is a hematologist who specializes in the treatment of multiple myeloma, a type of cancer affecting the blood marrow. She treats patients at the Hematology and Blood and Marrow Transplant Clinic. Read More