What to Know About Kymriah for Leukemia and Lymphoma

Immunotherapy has become a game-changer in the treatment of cancer. It works by harnessing your immune system to help detect and fight cancer cells. Today, there are several types of immunotherapy medications that cancer specialists can choose from. 

Chimeric antigen receptor (CAR) T-cell therapy is one of these options. It works by using your T cells, a type of white blood cell, to fight cancer. CAR T-cell therapy can be helpful in treating certain types of cancer even after other treatments haven’t worked. 

Kymriah (tisagenlecleucel) is a type of CAR T-cell therapy. In fact, it was the first CAR T-cell therapy to obtain FDA approval. But it’s a complex medication, and there are several things to know about it. Here, we’ll discuss how Kymriah works, how effective it is, and its possible side effects.

What is Kymriah?

Kymriah is a medication that’s unique to you. This is because it’s made using your own T cells. It is used for a couple types of cancer:

• Non-Hodgkin lymphoma (NHL): Kymriah treats adults with non-Hodgkin lymphoma (NHL) that has come back (relapsed cancer) or has stopped responding to other treatments (refractory cancer). It’s most effective against certain types of NHL, namely follicular lymphoma and diffuse large B-cell lymphoma (DLBCL).

• Acute lymphoblastic leukemia (ALL): Kymriah treats relapsed or refractory ALL in people ages 25 or younger.

Because Kymriah is personalized to you, it works differently than treatments like chemotherapy and stem cell transplants. More on this next.

How does Kymriah work for leukemia and lymphoma?

CAR T-cell therapies such as Kymriah modify your immune cells to allow them to target and destroy cancer cells. Kymriah is a type of gene therapy medication because it genetically alters your T cells to fight your cancer.

Your healthcare provider will collect your T cells to get the process started. These cells are taken from your body in a process called leukapheresis. This is similar to donating blood, except that the machine taking your blood keeps only the white blood cells and returns the rest of your blood to your body. Once your T cells are secured, they’re sent to a lab and transformed into Kymriah.

At the lab, your T cells are modified to detect and kill cancerous B cells. The genetic modification allows them to recognize CD19, an antigen (protein) that’s found on cancerous B cells in NHL and ALL.

It often takes labs about a month to make Kymriah. After it’s made, the finished product is sent back to your healthcare provider so they can give it to you.

How is Kymriah given when treating leukemia and lymphoma?

Before you receive Kymriah, your cancer specialist will give you a short course of chemotherapy to prepare your body for the treatment. This is also known as a preparative regimen. It’s meant to destroy your original T cells so Kymriah can step in and do its job. It usually consists of two chemotherapy medications — fludarabine and cyclophosphamide.

A few days after chemotherapy, Kymriah is infused into a vein over about 1 hour. It’s usually only given a single time. 

How effective is Kymriah when treating leukemia and lymphoma?

Most of what we know about Kymriah’s effectiveness comes from clinical trials. 

Kymriah was initially studied in 75 people ages 25 and younger who had relapsed or refractory ALL. In the study, the overall remission rate within 3 months was 81%. This means that 81% of people who received Kymriah saw a disappearance of all signs of their cancer, or experienced a decrease in the amount of cancer in their body.

Kymriah was also studied as a treatment for adults with relapsed or refractory DLBCL. Of the 93 adults that received Kymriah, 40% had a complete response. This means that 40% of the people saw a disappearance of all signs of cancer in their body.

What are the potential side effects of Kymriah?

Even though Kymriah works by targeting cancerous B cells, it can also affect healthy cells. This can lead to side effects.

Common side effects

A number of Kymriah’s side effects are fairly common. They include:

• Infection, which may stem from Kymriah or chemotherapy

• Diarrhea

• Nausea and vomiting

• Fatigue

• Low blood pressure

• Bleeding 

• Headache

To lower your risk of infection after receiving Kymriah, try to wash your hands regularly and avoid crowded places. And if you experience a fever, chills, or new cough, let your healthcare provider know right away. These are possible symptoms of an infection that should be addressed as soon as possible.

You should also let your provider know if you experience nausea or vomiting after receiving Kymriah. They can recommend anti-nausea medications, such as ondansetron (Zofran) or prochlorperazine, for relief. You can also try eating smaller meals and staying well hydrated. Over-the-counter (OTC) antidiarrheals, such as loperamide (Imodium A-D), can help manage diarrhea.

Another side effect called cytokine release syndrome (CRS) is also possible. CRS is an inflammatory response that can happen from medications like Kymriah. Most people only experience mild CRS, but it can sometimes become severe.

Serious side effects

CRS and neurological problems are the main serious side effects to watch out for with Kymriah. Kymriah even has boxed warnings for these risks — the FDA’s most serious medication warning. 

Signs and symptoms of severe CRS include:

• Difficulty breathing

• Fever

• Chills

• Confusion

• Nausea and vomiting

• Diarrhea

• Muscle or joint pain

• Low blood pressure

Signs and symptoms of neurological side effects include:

• Changes in awareness or consciousness 

• Confusion

• Feeling agitated

• Seizures

• Loss of balance

• Difficulty speaking and understanding

Other risks are possible, too:

• Allergic reactions, including anaphylaxis. This may include symptoms like difficulty breathing, low blood pressure, or swelling.

• Serious infections, which can be life-threatening. Tell your healthcare provider immediately if you develop fever, chills, body aches, or symptoms of an infection.

• Hypogammaglobulinemia. This is when the level of immunoglobulins in your body are low. This can raise your risk of infections.

• Cancer. Secondary cancers, or the recurrence of your cancer, can occur after receiving Kymriah.

• Coordination problems. Kymriah may cause temporary memory and coordination problems. This can temporarily affect your ability to drive. You should not drive or operate other heavy machinery for 2 months after you get Kymriah.

Because of side effects like these, Kymriah is only available through a Risk Evaluation and Mitigation Strategy (REMS) program called the Kymriah REMS. Your cancer specialist can tell you more about this program.

How to save on Kymriah

Without any insurance coverage or financial assistance, Kymriah has a high sticker price. But there are ways you can save.

Kymriah may be covered under your medical benefit if you have health insurance. Novartis, the manufacturer of Kymriah, also has a financial assistance program called Kymriah Cares that offers assistance to eligible recipients. For more information, call 1-844-459-6742 or visit Kymriah Cares online. 

Are there any alternatives to Kymriah?

Yes. Even though Kymriah was the first CAR T-cell therapy to be approved, other options are now available. This includes Yescarta (axicabtagene ciloleucel) and Breyanzi (lisocabtagene maraleucel).

Yescarta and Breyanzi are both approved to treat adults with relapsed or refractory large B-cell lymphoma. Yescarta is also approved to treat adults with relapsed or refractory follicular lymphoma.

Kymriah vs. Yescarta and Breyanzi

There aren’t any head-to-head studies that directly compare one CAR-T cell medication to another. But their approved uses help set them apart. Breyanzi and Yescarta may be considered earlier on in your journey with lymphoma:

• Breyanzi and Yescarta are approved to treat DLBCL after receiving only one line of prior treatment.

• Kymriah is approved to treat people with relapsed or refractory DLBCL after receiving two or more lines of prior treatment.

Why is there this distinction? Clinical trials showed that Breyanzi and Yescarta — when used as second-line treatments — helped people stay cancer free for longer than people who received standard care. This was not the case for Kymriah.

Why trust our experts?

Written by:

Sonja Jacobsen, PharmD, BCPS, BCOP

Sonja Jacobsen, PharmD, BCPS, BCOP,  is a clinical oncology pharmacy specialist currently practicing in Seattle. She has been practicing as a pharmacist since 2015 and is licensed to practice in Washington state and North Carolina.

Edited by:

Joshua Murdock, PharmD, BCBBS

Joshua Murdock, PharmD, BCBBS, is a licensed pharmacist in Arizona, Colorado, and Rhode Island. He has worked in the pharmacy industry for more than 10 years and currently serves as a pharmacy editor for GoodRx.

Reviewed by:

Sophie Vergnaud, MD

Sophie Vergnaud, MD, is the Senior Medical Director for GoodRx Health. An experienced and dedicated pulmonologist and hospitalist, she spent a decade practicing and teaching clinical medicine at academic hospitals throughout London before transitioning to a career in health education and health technology.

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