Future directions in chimeric antigen receptor T cell therapy

Curr Opin Pediatr. 2017 Feb;29(1):27-33. doi: 10.1097/MOP.0000000000000436.


Purpose of review: The impact of immunotherapy has grown exponentially in the past 5 years. Principle illustrations are encouraging results with engineered T cells expressing a chimeric antigen receptor (CAR). This experimental therapy is developing simultaneously in pediatric and adult clinical trials, making this field particularly relevant and exciting for pediatric oncologists.

Recent findings: CAR-modified T cells targeting CD19 have produced dramatic antitumor responses in patients with relapsed/refractory B cell acute lymphoblastic leukemia. Clinical trials from several institutions, in both children and adults, using distinct CAR T cell products have demonstrated similar high complete remission rates of 61-93%, with durable remissions observed. Although the development of CARs for other malignancies has lagged behind, research into novel approaches to overcome inherent challenges is promising.

Summary: Clinical trials of CAR-modified T cells have produced unprecedented results and are anticipated to have a broader impact as this approach expands into other indications, including other cancers and frontline therapy. The potential for long-term disease control, if fully realized, will have a transformative impact on the field.

Publication types

  • Review
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Antigens, CD19 / immunology*
  • Biomarkers, Tumor / immunology*
  • Cell Engineering
  • Cell- and Tissue-Based Therapy / methods*
  • Humans
  • Immunotherapy / methods*
  • Precursor Cell Lymphoblastic Leukemia-Lymphoma / immunology
  • Precursor Cell Lymphoblastic Leukemia-Lymphoma / therapy*
  • Receptors, Antigen, T-Cell / therapeutic use*
  • T-Lymphocytes / immunology*


  • Antigens, CD19
  • Biomarkers, Tumor
  • Receptors, Antigen, T-Cell