CAR T-Cell Therapy

What is CAR T-cell therapy?

CAR T-cell therapy is a specialized type of immunotherapy that uses your child’s own immune cells to find and destroy cancer cells.  

The immune system protects the body from infections and disease. It recognizes and attacks germs such as bacteria and viruses. It also helps find and remove unhealthy or abnormal cells, such as cancer cells.  

The immune system includes many types of cells. One type is the T cell, a white blood cell that helps recognize and attack abnormal cells. 

CAR stands for chimeric antigen receptor. A CAR is a special type of receptor made in the laboratory and added to your child’s T cells. The changed cells, called CAR T cells, are given back to your child through an infusion. The CAR helps the T cells better target and fight cancer cells.  

Once in the body, CAR T cells can continue to grow and multiply in your child’s body as they work to do their job of helping your child’s immune system recognize and fight the cancer cells.  

How CAR T-cell therapy works

CAR T cells are specially designed in a laboratory to recognize specific proteins on the surface of cancer cells, called antigens. When the CAR T cells recognize the antigen, they attach to the cancer cell and destroy it. This therapy helps your immune system better recognize and fight cancer. 

Conditions treated with CAR T-cell therapy

CAR T-cell therapies are often used to treat certain blood cancers, including B and T acute lymphoblastic leukemia (ALL), lymphoblastic lymphoma, and acute myeloid leukemia (AML). 

Researchers are also studying CAR T-cell therapy for certain solid tumors and brain tumors, as well as other non-cancer conditions, including autoimmune diseases such as lupus. 

Kymriah® (tisagenlecleucel) is currently the only commercially available CAR T-cell therapy FDA-approved for use in children. Some CAR T-cell therapies may only be available in a clinical trial. 

What to expect during CAR T-cell therapy

1. Collect T cells (apheresis) 

Apheresis is a procedure to remove a part of the blood. Blood will be collected from an IV in your child’s arm or central line.   

An apheresis machine takes blood from a vein and separates it into parts. The white blood cells are removed from the blood and stored. The rest of the blood is returned to the body through a vein. Only a small amount of blood is out of the body at any time.  

The lab counts the white blood cells after the procedure to see if there is enough. Apheresis usually takes 3-6 hours, and counting takes another 1-2 hours. Apheresis can be repeated the next day or another time if more cells are needed.  

2. Make CAR T cells in the lab

Your child’s T cells are sent to a specialized laboratory, where the cells are changed by adding a chimeric antigen receptor (CAR). These modified cells, called CAR T cells, are designed to recognize your child’s cancer. The CAR T cells are grown in the lab until there are enough for treatment. This process usually takes several weeks. 

In some cases, the laboratory may not be able to make the CAR T cells. This can happen for many reasons, and it is not caused by anything your child or family did. If this happens, your care team will discuss next steps with you.  

3. Prepare for CAR T-cell therapy   

While waiting for CAR T-cell therapy, your child might get other treatments such as chemotherapy, radiation, or surgery. This is sometimes known as bridging therapy because they help control the cancer while CAR T cells are being made. Your care team will work with you to make a treatment plan based on your child’s needs. 

Many patients may need to be admitted to the hospital for CAR T-cell therapy, especially those treated in a clinical trial.  

4. Receive CAR T cells 

In most cases, CAR T cells are given by infusion. Before the CAR T-cell infusion, patients usually get several days of lymphodepleting chemotherapy. This will temporarily decrease your child’s immune cells so that the CAR T cells grow and work better.  

The CAR T cells are then given to your child by an infusion through an IV or a central line. It is not painful. A nurse will monitor your child during the infusion. The infusion usually takes less than an hour. A parent or family caregiver may stay with your child during the infusion. Your child will be closely monitored for several hours after the infusion to watch for any immediate side effects.  

Side effects of CAR T-cell therapy

Apheresis side effects  

Your child might have some side effects during or after apheresis. Side effects may include: 

• Bleeding or bruising 
• Feeling dizzy or lightheaded 
• Feeling anxious or cold 
• Nausea 
• Numbness or tingling 
• Muscle cramps 

Apheresis can temporarily increase your child’s risk of bleeding. During apheresis, your child will receive medicines to prevent blood clots. The risk of bleeding is even higher if your child takes certain medications before or after the procedure. These medicines include aspirin and NSAIDs, such as ibuprofen and naproxen. Avoid aspirin, NSAIDs, and medicines that contain them for 1 week before apheresis and for 2 weeks afterward. Children under 18 should not take aspirin or any medicines that contain aspirin.  

Anti-clotting medicine used during apheresis can also affect the way your child’s body uses calcium. If this happens, your child may be given calcium to help with side effects. 

Apheresis can lower the number of red blood cells and platelets. Your child might need a blood transfusion if this happens. 

Chemotherapy side effects  

Your child may have side effects from lymphodepleting chemotherapy given before CAR T-cell infusion. Symptoms can vary based on the type and dose of chemotherapy. Side effects may include: 

• Nausea or vomiting 
• Feeling tired 
• Low blood counts 
• Increased risk of infection 

Talk to your care team about what side effects to expect and ways to help manage them if they occur. 

Cytokine release syndrome (CRS)

Cytokine release syndrome (CRS) is a group of symptoms that can develop as a side effect of CAR T-cell therapy and some antibody therapies. The syndrome occurs when immune cells are activated and release large amounts of cytokines, causing inflammation in the whole body. In severe cases, CRS can cause breathing problems, low blood pressure, and damage to organs.  

CRS can develop quickly. Watch for signs and symptoms of CRS, such as: 

• Fever 
• Trouble breathing 
• Low blood pressure   
• Headache 
• Body aches 
• Tiredness 
• Rapid heartbeat 

CRS usually develops within the first few days after the infusion of CAR T cells and is only a risk during the initial weeks after treatment. It often begins with fever and flu-like symptoms, but CRS can worsen quickly and cause serious illness.  

If you notice any of these symptoms after CAR T-cell therapy, contact your care team right away. It is very important to seek care to prevent the CRS from getting worse. Always follow your care team’s instructions about when to seek emergency help. 

Treatment for CRS focuses on supportive care and managing symptoms. Your child may need steroid medicines or medicines that lower the immune response. 

Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANS) 

Immune effector cell-associated neurotoxicity syndrome (ICANS) is a side effect that can happen after CAR T-cell therapy. ICANS affects the brain and nervous system and can cause changes in thinking, speech, movement, or alertness. It often occurs after or at the same time as cytokine release syndrome (CRS), but it can also happen on its own.  

Most cases of ICANS are mild and improve with treatment, but severe cases can be life-threatening if not treated promptly. 

ICANS can develop quickly. Watch for signs and symptoms such as: 

• Confusion or trouble thinking clearly  
• Problems speaking or finding words  
• Sleepiness or difficulty staying awake  
• Decreased alertness or loss of consciousness  
• Seizures  
• Muscle weakness  
• Headache  
• Tremors or shaking  
• Changes in handwriting  
• Problems with balance or coordination

ICANS usually develops within 3–10 days after CAR T-cell therapy, although it can occur earlier or up to a few months after infusion. Symptoms may start gradually but can worsen quickly. 

If you notice any of these symptoms after CAR T-cell therapy, contact your care team right away. It is very important to seek care as soon as symptoms begin. Always follow your care team's instructions about when to seek emergency help. 

ICANS is usually managed with monitoring, supportive care, steroid medicines, medicines to prevent seizures, and treatment of symptoms.  

Local inflammation

CAR T-cell therapy may also cause inflammation and swelling at or around the tumor. For patients with brain tumors, this can cause new or worsening neurological symptoms. In patients with other solid tumors, symptoms may depend on where the tumor is located. 

Your child’s care team will watch closely for these problems. 

Infection

CAR T-cell therapy can weaken the immune system and increase the risk of infection. Chemotherapy given before CAR T-cell therapy can lower the number of infection-fighting blood cells. CAR T cells may also affect healthy immune cells that help the body fight infection. Some medicines used to manage CAR T-cell side effects can weaken the immune system further.   

Take steps to prevent infection, and watch for signs of infection or illness. Contact your care team right away if your child has a fever or other signs of infection.  

After CAR T-cell therapy

Watch for side effects, including symptoms of cytokine release syndrome and neurologic changes (ICANS).  

• Contact your care team right away if your child shows any symptoms or if you have any other concerns.  
• You will get a Patient Wallet Card with information on the side effects of CAR T-cell therapy. Keep this card with you at all times. Be sure that all health care providers know that your child received CAR T-cell therapy or show them this card.  
• Your child must stay within 2 hours of the hospital for at least 4 weeks after the infusion, unless otherwise instructed by your care team. 
• Do not let your child drive or do dangerous activities or jobs for at least 8 weeks after receiving CAR T-cell therapy. 

After CAR T-cell therapy, your child will have regular outpatient clinic visits and lab tests to check their progress. Your care team will monitor your child’s cancer to see if the treatment is working. Typically, this involves undergoing repeat testing to monitor cancer levels in the body, which occurs about 1 month after the CAR T-cell infusion.  

Your care team will discuss the specifics of this evaluation with you and your child, including when and how often they will occur. 

Always follow the instructions given by your care team. Talk to your doctor or pharmacist if you have questions or concerns. 

Questions to ask your care team

• How does CAR T-cell therapy work? 
• Is my child a good candidate for CAR T-cell therapy? 
• Can CAR T-cell therapy cure my child’s cancer? 
• What other treatments could help treat my child’s cancer? 
• What are the potential benefits of CAR T-cell therapy compared to other treatments? 
• What are the short-term and long-term risks or side effects? 
• How long does the whole process take, including recovery time? 
• What kind of follow-up care and monitoring will my child need? 
• How long will my child need to stay near the hospital after treatment? 
• What symptoms should make me call the care team right away? 

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