Chimeric Antigen Receptor (CAR) T-cell Therapy For Pediatric Acute Lymphoblastic Lymphoma – Some Resources

This post contains links to an Indianapolis Star article,  Riley Hospital for Children uses what could be revolutionary cancer therapy on first patient,on a revolutionary new cancer treatment therapy that Riley Children’s Hospital is using, Resource (1) below:

18 year old Kilee Sweat was diagnosed with Acute Lymphoblastic Leukemia in 2012.

Pediatric Acute Lymphoblastic Lymphoma (2) is the most common cancer of childhood and causes 3,000 new cases a year (accounting for one quarter of all pediatric cancers each year).

90% of patients with the disease are cured following grueling cancer chemotherapy.

Kilee was one of the 10% who are not cured with current firstline therapy.

For these patients, additional therapy is needed and the prognosis for cure may not be as favorable.

One of those additional therapies is bone marrow transplant.

But Kylee chose to undergo CAR T-cell therapy, a new therapy just approved by the FDA in 2017.

Resources (2), (3), and (4) below provide more information from the National Cancer Institute on the revolutionary Chimeric Antigen Receptor (CAR) T-cell Therapy For Cancer that Riley Hospital is using.

The Indianapolis Star article points out that the CAR T-cell Therapy [See Resources (3), (4), and (5)] has significant drawbacks as well as many potential benefits:

Kymriah [Tisagenlecleucel], Novartis’s name for the CAR-T cell therapy it offers, costs $475,000 for leukemia and $373,000 for lymphoma. The $475,000 covers the cost of the drug alone; the subsequent hospital stay is additional.

Plus, the majority of those who receive this therapy experience a side effect known as cytokine release syndrome in which the body floods with cytokines, immune substances that under other circumstances can be beneficial. In large amounts they can throw the body out of whack and may eventually lead to high fevers and shock. The therapy may also lead to neurotoxicity, which can range from mild confusion to life-threatening seizures. In about 5 to 10 percent of children who receive this therapy, these side effects will prove fatal, Skiles said.

“The really hard part about this and the reason why nobody is jumping up and down to say let’s give this medicine to everybody is because about 70 percent of kids who get this drug will wind up in the ICU,” Skiles said. “I think that’s why people are hesitant to say let’s make this a prime time, first-line drug because it’s not an insignificant amount of toxicity but it’s only toxicity for the first month or two instead of toxicity for three years and the rest of your life.”

Nor can doctors promise that this therapy will offer a cure. A New England Journal of Medicine study earlier this year found that of 75 patients studied, 81 percent saw remission of their cancer at three months. The overall survival rate was 90 percent at six months. At the one year mark, those numbers dipped to 76 percent.


(1) Riley Hospital for Children uses what could be revolutionary cancer therapy on first patient from The Indianapolis Star, November 25, 2018.

(2) Pediatric Acute Lymphoblastic Leukemia Updated: Sep 05, 2017 Author: Vikramjit S Kanwar, MBBS, MBA, MRCP(UK), FAAP from

(3) Definition of CAR T-cell therapy from NCI Dictionary of Cancer Terms

(4) CAR T Cells: Engineering Patients’ Immune Cells to Treat Their Cancers from the National Cancer Institute.

(5) NCI Dictionaries

NCI Dictionary of Cancer Terms
A dictionary of cancer and biomedical terms defined in non-technical language. Terms and definitions are reviewed by a multidisciplinary panel of reviewers and new terms are added each month.

NCI Dictionary of Genetics Terms
A dictionary of more than 150 genetics-related terms written for healthcare professionals. This resource was developed to support the comprehensive, evidence-based, peer-reviewed PDQ cancer genetics information summaries.

NCI Drug Dictionary
The NCI Drug Dictionary contains technical definitions and synonyms for drugs/agents used to treat patients with cancer or conditions related to cancer. Each drug entry includes links to check for clinical trials listed in NCI’s List of Cancer Clinical Trials.

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