Oncology Researchers Investigating Treating Pediatric Cancer With CAR-T Therapy

An innovative form of cancer treatment for patients as old as 25 with the most prevalent kind of pediatric leukemia has more than doubled the survival rate, with 82% of patients going into remission. Doctors find that 66% of patients receiving this treatment are relapse-free after 18 months.

Approved by the U.S. Food and Drug Association in 2017, the drug KYMRIAH is a form of chimeric antigen receptor (CAR) T-cell therapy.

The FDA approves it for use on patients diagnosed with B-cell precursor acute lymphoblastic leukemia (also known as “ALL”), which is the most common kind of pediatric leukemia, according to the University of Texas Southwestern Medical Center. Doctors will use the therapy when other types of treatment have not been effective.

Before researchers developed chimeric antigen receptor therapy, the survival rate for ALL was 20 to 30%.

The Texas medical center is one of 13 centers in the U.S. that took part in a clinical trial for KYMRIAH in 2015, testing the drug for safety and feasibility. They also are participating in ongoing work to optimize the therapy for adults of all ages, which is now available for patients just up to 25 years old.

About B-cell Precursor Acute Lymphoblastic Leukemia

ALL is a rare form of cancer, accounting for less than half of one percent of all cancer cases in the U.S., per the University of Texas. The majority (some 80%) of patients are children.

About 85 to 90% of children diagnosed with pediatric ALL respond to standard chemotherapy treatment.

For the remaining 10-15% of young patients whose chemotherapy does not help or whose cancer has come back, an option is to seek treatment at UT Medical Center. Doctors currently treat about 30 patients every year with innovative immunotherapy treatment. The human immune system sometimes needs a boost from medical science, especially in the face of rare and severe diseases.

KYMRIAH trials can help patients when traditional methods don’t yield success. The idea is that cancer cells can adapt, learning how to avoid chemotherapy. But when you help train the body’s immune system, only leukemia is destroyed and not healthy tissue.

Unfortunately, damage to normal tissue is often an uncomfortable side effect of chemotherapy today. So for patients that have not gone into remission with standard care, CAR-T therapy may be an appropriate approach.

About CAR-T Therapy

With chimeric antigen receptor T-cell therapy, doctors harness the patient’s own immune system T-cells. They modify a section of a patient’s T-cells, teaching it how to find what’s known as the CD19 protein inside the B-cells afflicted with cancer. Once the T-cells detect a cancerous B-cell, it destroys it.

The work with car-T therapy doesn’t end with B-cell Precursor Acute Lymphoblastic Leukemia. At UT Southwestern, researchers are also turning their attention to using it to fight pediatric acute myeloid leukemia, also known as AML.

The team is anticipating going into clinical trials to test the therapy on another ailment. Presently, UT Southwestern scientists are involved in testing CAR T-cell therapy with clinical trials for adult patients with multiple myeloma that relapsed.

Current Therapy Is Approved for Patients Who Have not Responded to Traditional, Standard Care for B-cell Precursor Acute Lymphoblastic Leukemia

While the results of the research are encouraging, it’s important for parents and other interested parties to keep in mind that the CAR-T therapy at the University of Texas Southwestern Medical Center is currently approved only for patients who have not responded to the current standard of care therapy.

With an eye to the future, UT researchers are advising parents with eligible children to consult with their child’s oncologist and therapy team so they can determine the most appropriate course of action.

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