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CAR T Cell Therapy in Acute Lymphoblastic Leukemia and Potential for Chronic Lymphocytic Leukemia

  • Leukemia (JP Dutcher, Section Editor)
  • Published:
Current Treatment Options in Oncology Aims and scope Submit manuscript

Opinion statement

Adoptive transfer of autologous T cells engineered to express a chimeric antigen receptor (CAR) represents a powerful targeted immunotherapy that has shown great promise in some of the most refractory leukemias. CAR-modified T cells directed against CD19 have led the way, setting a high standard with remission rates as high as 90 % in clinical trials for relapsed/refractory acute lymphoblastic leukemia (ALL). Yet, the first demonstration of efficacy was in another disease, chronic lymphocytic leukemia (CLL), in which CD19-targeted CAR T cells eradicated bulky, highly refractory disease. Despite early encouraging results, clinical trials in CLL have yielded lower response rates, revealing disease-specific differences in response in this form of immunotherapy. Ongoing research focused on identifying and overcoming these limitations, promises to improve response rates. Beyond the induction of remission, the transformative impact of engineered T cell therapy lies in its potential for long-term disease control. With longer follow-up and durable T cell persistence now reported, we are closer to answering the question of whether sustained remissions are possible with CAR T cell monotherapy. As might be expected with a highly effective therapy using a single mechanism of action, escape pathways have emerged. Combinatorial approaches are needed to anticipate and prevent this mode of relapse. Lastly, toxicity management is vital to ensure the safety of this exciting cancer immunotherapy.

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Author information

Authors and Affiliations

  1. Department of Medicine, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA

    Nathan Singh MD & Noelle V. Frey MD, MSCE

  2. Division of Oncology, The Children’s Hospital of Philadelphia, Department of Pediatrics, University of Pennsylvania Perelman School of Medicine, 3012 CTRB, 3501 Civic Center Blvd., Philadelphia, PA, 19104, USA

    Stephan A. Grupp MD, PhD & Shannon L Maude MD, PhD

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  1. Nathan Singh MD
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  2. Noelle V. Frey MD, MSCE
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  3. Stephan A. Grupp MD, PhD
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  4. Shannon L Maude MD, PhD
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Corresponding author

Correspondence to Shannon L Maude MD, PhD.

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Conflict of Interest

Nathan Singh declares that he has no conflict of interest.

Noelle V. Frey has received research funding through grants from Novartis.

Stephan A. Grupp has received research funding through grants and has served as a consultant for Novartis and has a patent pending for toxicity management in ALL, which is managed under policies of the University of Pennsylvania.

Shannon L. Maude has received research funding through grants and has served as a consultant for Novartis.

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This article reviews reports of human studies, but does not contain the primary research.

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This article is a part of Topical Collection on Leukemia

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Singh, N., Frey, N.V., Grupp, S.A. et al. CAR T Cell Therapy in Acute Lymphoblastic Leukemia and Potential for Chronic Lymphocytic Leukemia. Curr. Treat. Options in Oncol. 17, 28 (2016). https://doi.org/10.1007/s11864-016-0406-4

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  • DOI: https://doi.org/10.1007/s11864-016-0406-4

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