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Expert Discusses Approval of First CAR T-Cell Therapy

Danielle Bucco
Published: Monday, Sep 18, 2017

James L. Ferrara, MD
James L. Ferrara, MD
The FDA’s recent approval of tisagenlecleucel (Kymriah) as the first chimeric antigen receptor (CAR) T-cell Therapy, marks a new era in oncology.

Ferrara, a professor of hematology and medical oncology at the Icahn School of Medicine at Mount Sinai, discussed the significance of the FDA approving tisagenlecleucel as the first CAR T-cell therapy.

OncLive: Can you please provide some background on this CAR T-cell therapy?

Ferrara: This is a therapy for relapsed leukemia. If a type of leukemia does not respond to chemotherapy, which happens around 25% of the time, it’s very unlikely to completely respond to chemotherapy again. When we treat leukemia, it goes through different stages, like intensification and then maintenance. We use these different chemotherapy strategies to have many ways of poisoning the cancer cell, but cancer cells can become resistant to chemotherapy. Usually, the only therapy that works is bone marrow transplantation from a different donor where you have this part of immunotherapy called graft-versus-leukemia effects. If you relapse after bone marrow transplantation, you’re very likely to die because nothing has worked. 

The therapy is complex and expensive but it is a huge advance that has taken decades of researchers investigating what the breaks are, how to take them off, how to make the target, and how to genetically engineer lymphocytes so that they don’t become cancerous themselves. It’s very high-tech, but the results are astonishing for these patients who have no options because their cancer has become resistant.

What are your thoughts on the cost of the therapy?

Since the cost comes to around half a million dollars, it appears that only the elite will be able to afford it and that is not a good solution. I wish there was an answer to this issue. Everybody worries about the cost, and it is such a big advance that many can benefit from.

Now that this has been approved, does this pave the way for other CAR T-cell therapies?

Absolutely. As soon as researchers heard about the initial results 3 or 4 years ago, there is a big race going on to find the right target for these therapies. This therapy targets a protein called CD19, which is not specific to lymphocytic leukemia cells, as it’s on many B-lymphocytes, which makes antibodies.

One negative side effect of this therapy is the patient loses their B cells that express CD19. The good news is that since it is only on B cells, we can replace the antibodies through a therapy called gamma globulin. These patients need to get an infusion once a month for the rest of their lives to help their immune systems by giving them antibodies but many patients do fine with this kind of therapy.

Where do you believe the next area of research should be?

It is likely that this therapy will extend to other subsets of leukemia and lymphoma. Solid tumors are more difficult because the protein targets are often expressed to some extent on other normal tissues, making it important to find the right target that has limited expression.

When the cancer cells are attacked, it causes a chain reaction between the target and the therapy itself which leaves cytokines, causing cytokine release syndrome and that is a problem that researchers are attempting to address. Those patients can get very sick and the practitioners must be alert to this because a patient can rapidly have a toxicity.


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Community Practice Connections™: 22nd Annual International Congress on Hematologic Malignancies®: Focus on Leukemias, Lymphomas and MyelomaMay 30, 20192.0
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