CAR T-Cell Therapy

The FDA approved in August 2017 the use of a new type of immunotherapy, called CAR T-cell therapy, for the treatment of B-cell acute lymphoblastic leukemia (ALL) in children and young adults. Cincinnati Children’s is one of the first pediatric hospitals certified to provide this groundbreaking treatment.

What Is CAR T-Cell Therapy?

CAR T-cell therapy, short for chimeric antigen receptor T-cell therapy, uses a patient’s own immune system to stop or slow aggressive and treatment-resistant cancers.

How Does It Work?

The therapy involves collecting T cells from a patient’s blood. T cells are infection-fighting cells in our bodies. The T cells are sent to a laboratory to be engineered to become cancer fighters. The T cells are reinfused into the patient. The engineered anti-cancer T cells then attack a protein called CD19 on leukemia cells.

Who Can Benefit from CAR T-Cell Therapy?

Patients up to 25 years of age with either refractory (difficult to treat) or second or greater relapse of B-cell ALL. There were previously no curative options in cases where chemotherapy or transplant didn’t work. Clinical trials have indicated that CAR T-cell therapy has the potential to provide long-term remission and even a cure for B-cell ALL.

Why Come to Cincinnati Children’s for Treatment?

Cincinnati Children’s has been conducting clinical trials related to ALL, the most common cancer in children, for some time, giving our care teams the necessary experience to care for these patients. We have multiple clinical trials for children with relapsed or refractory ALL, including CAR-T therapy and other immunotherapies. We are also now among a small number of places approved as a treatment site for the drug tisagenlecleucel (brand name Kymriah), which is used in CAR T-cell therapy.

Cincinnati Children’s recently received approval to use Kymriah for our patients with relapsed or refractory large B-cell lymphoma, such as diffuse large B-cell lymphoma (DLBCL).

What Is Kymriah?

Kymriah is a one-time treatment that has shown an 83 percent remission rate for B-cell acute lymphoblastic leukemia (B-ALL) in clinical trials with patients who do not respond to traditional treatments.

While this treatment can have significant side effects, it offers hope for patients that had none before. This therapy will continue to be modified for better, more robust treatment that will save the lives of those patients with relapsed cancer.

What Is Yescarta?

At Cincinnati Children’s, we are also testing other forms of anti-leukemia CAR-T cell therapies. Yescarta (axicabtagene ciloleucel) has been approved by the FDA for the treatment of adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL), primary mediastinal large B-cell lymphoma (PMBCL), high-grade B-cell lymphoma, and DLBCL arising from follicular lymphoma. 

We offer the Kite Yescarta KTE-C19 CAR T-cell therapy, examining its activity in children and young adults with relapsed / refractory B-precursor acute lymphoblastic leukemia (the ZUMA-4 trial). 

In adults, Yescarta KTE-C19 demonstrated a 71 percent rate of complete remission, with 100 percent of responders having no detectable minimal residual disease, including patients with high tumor burden and high-risk genetic abnormalities.

What You Need to Know

Visit our blog.

A pediatric oncologist at Cincinnati Children’s shares four things parents must know about CAR T-cell therapy.

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Clinical Trial

Learn more about our clinical trials.

We continue to study the use of CAR T-cell therapy in relapsed / refractory B-precursor acute lymphoblastic leukemia.

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