Pediatric Cellular Therapy Program

Immunotherapy is an exciting new form of cancer treatment that harnesses a body's own immune system to kill cancer cells. The Pediatric Cellular Therapy Program, located in NewYork-Presbyterian Morgan Stanley Children's Hospital, is one of the first to be certified in CAR T-cell therapy for children and adolescents with recurrent or persistent acute lymphoblastic leukemia (ALL). 

We offer the FDA-approved CAR T-cell therapy, KYMRIAH, to patients up to 25 years old with ALL that has continued to grow or come back despite two or more regimens of treatment. 

We are also conducting clinical trials to evaluate other uses of CAR T-cell therapy, including:

• treatment of children and adolescents with non-Hodgkin lymphoma
• use of T-cells to prevent and fight viral infections in children undergoing bone marrow or solid organ transplantation

Our physicians have years of experience with CAR T-cell therapy in clinical trials. They know how to give it safely and manage any side effects. They also have expertise caring for patients with multiple health problems and complex medical needs.

What is CAR T-cell therapy?

T-cells are immune cells in the blood that fight viruses and cancer. But sometimes, cancer cells find ways to defeat, trick, or overwhelm the body's T-cells, allowing the cancer to grow. CAR T-cell therapy is a type of immunotherapy in which a patient's own T-cells are modified to successfully attack and kill cancer cells. 

With CAR T-cell therapy, we remove T-cells from your blood, add genetic material in a lab, and then grow more of the modified cells. These modified cells contain special protein receptors on their surface (chimeric antigen receptors, or CAR), which help them to recognize and kill cancer cells. We then inject millions of CAR T-cells back into the body, where they act as "killer cells" by agressively seeking out and destroying cancer cells.

Who can benefit from CAR T-cell therapy?

CAR T-cell therapy has great potential for cancer treatment, and clinical trials are looking at its use for many types of cancer in children and adults. 

Currently, CAR T-cell therapy is approved for children and adolescents with recurrent or persistent ALL. Most people with ALL respond well to standard chemotherapy, but about 15 percent of cases either don't respond or respond only temporarily to chemotherapy. Those people, along with chidren and adolescents whose disease has returned after a bone marrow transplant, may benefit from CAR T-cell therapy. 

Your care team at Columbia and NewYork-Presbyterian will evaluate your child and work with your family to decide if CAR T-cell therapy is the right treatment for your child.

What should I expect from CAR T-cell therapy?

There are several steps in the treatment process. We will begin by introducing your family to your immunotherapy care team at Morgan Stanley Children's Hospital, which may include an interventional radiologist, anesthesiologist, apheresis technician, nurse practitioner, and social worker. 

1. The first step in the process involves collecting T-cells. T-cell collection takes place through a process called apheresis. First, your child's veins will be evaluated. If they are too small or delicate to accomodate the needle used for apheresis, you will be referred to an interventional radiologist, who will place a central line, also called a catheter, to facilitate apheresis. This involves a short procedure in an operating room, under anesthesia.

During apherisis, you will be able to sit with your child in a comfortable room, with care team members present at all times to answer your questions. Apheresis takes between three and five hours. Blood is drawn, T-cells are extracted, and the blood is then returned to the body. 

2. The T-cells are processed in the lab. Your child may get regular chemotherapy treatment while CAR T-cells are being engineered.

3. The CAR T-cells are reintroduced to the body. The CAR T-cell infusion itself takes only a few minutes, but the entire process involves a longer stay in the hospital. Before the infusion, your child will receive a series of preparation chemotherapy treatments. Following the infusion, your child will be closely monitored for side effects. We will also do some tests to help us understand the response to the therapy before your child goes home.

4. We will continue to monitor your child closely through outpatient visits.

What are the side effects from CAR T-cell therapy? 

Most complications from CAR T-cell therapy occur within the first four weeks. We monitor your child closely during this period. 

Common short-term side effects include sleepiness, confusion, weakness, dizziness, problems with coordination, and seizures.

Some children experience more serious side effects, such as high fever or sudden blood pressure drop. Children with serious side effects are admitted into our pediatric intensive care unit (PICU), where our physicians and staff are well-equipped to handle the effects of this therapy. 

Research and Clinical Trials

Current cellular therapy studies at our center include:

• AAAS3695:  Zuma-4: Study Evaluating KTE-C19 in Pediatric and Adolescent Subjects with Relapsed/Refractory B-precursor Acute Lymphoblastic Leukemia and non-Hodgkin lymphoma.
• AAAS44110: A Phase II Trial of Tisagenlecleucel in First-line High-risk Pediatric and Young Adult Patients with B-cell Acute Lymphoblastic Leukemia Who Are Minimal Residual Disease Positive at the End of Consolidation  Therapy
• AAAR8071: Antiviral Cellular Therapy for Enhancing T-cell Reconstitution Before or After Hematopoietic Stem Cell Transplantation (ACES) PBMTC SUP1701.
• Multicenter, Open-Label, Phase 3 Trial of Allogeneic Epstein-Barr Virus Cytotoxic T Lymphocytes  for Allogeneic Hematopoietic Cell Transplant  Patients with EBV-Associated Post-Transplant Lymphoproliferative Disease after Failure of Rituximab 
• ANHL1522: A Pilot Study of Rituximab  and Third Party Latent Membrane Protein specific Cytotoxic T-Lymphocytes in Pediatric Solid Organ Recipients with EBV-Positive CD20-Positive Post-Transplant Lymphoproliferative Disease
• Multicenter, Open Label, Phase 3 Study of Tabelecleucel for Solid Organ Transplant Subjects with Epstein-Barr Virus-Associated Post-Transplant Lymphoproliferative Disease after Failure of Rituximab or Rituximab and Chemotherapy (ALLELE Study)