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Most Recent Clinical Advances in CAR T Cell and Gene Therapy 2017/2018

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Cell and Gene Therapies

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Abstract

Adoptive cell therapy with gene-engineered T cells bearing antitumor-reactive T-cell receptor or chimeric antigen receptor (CAR) is a promising and rapidly evolving field of translational medicine. This approach has delivered exciting responses for some patients with lymphoid hematologic neoplasms, leading to recent US Food and Drug Administration approvals. Hematopoietic stem cellular gene therapy has also shown promising advances, with durable and potentially curative clinical benefit and without the potential toxicities of allogeneic hematopoietic cell transplant. However, for both of these novel strategies, many questions remain unanswered. Compared to synthetic viral gene addition therapy (e.g., CAR T-cell engineering), translation of gene-editing technologies to patient care is in its infancy. Multiple clinical trials are ongoing or expected to open for CAR T cell and inherited monogenic disorders (Gardner et al., Blood 129:3322–3331, 2017) (refer to subsequent disease-specific chapters in the book). In this chapter, we will highlight the most recent and clinically relevant developments in the arena of gene-modified T-cell-based therapies and hematopoietic stem cellular gene therapy specifically focusing on hematologic disorders. We will conclude the chapter by summarizing the apparent challenges and directions for the future.

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Notes

  1. 1.

    https://www.fda.gov/downloads/BiologicsBloodVaccines/CellularGeneTherapyProducts/ApprovedProducts/UCM573941.pdf. Accessed July 4, 2018.

  2. 2.

    https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm581216.htm. Accessed July 4, 2018.

  3. 3.

    https://www.fda.gov/drugs/informationondrugs/approveddrugs/ucm606540.htm. Accessed July 4, 2018.

  4. 4.

    https://www.cancer.gov/publications/dictionaries/cancer-drug/def/792630. Accessed July 4, 2018.

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Conflict of Interest

Syed A. Abutalib—None.

Saar I Gill—Research funding (Novartis); Equity (Carisma Therapeutics); Scientific Advisory Board (Carisma Therapeutics, Extellia Therapeutics).

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Authors and Affiliations

  1. Cancer Treatment Centers of America, Zion, IL, USA

    Syed A. Abutalib

  2. Chicago Medical School, Rosalind Franklin University of Medicine and Science, North Chicago, IL, USA

    Syed A. Abutalib

  3. Division of Hematology-Oncology, Department of Medicine, University of Pennsylvania, Philadelphia, PA, USA

    Saar I. Gill

  4. Smilow Translational Research Center, Philadelphia, PA, USA

    Saar I. Gill

Authors
  1. Syed A. Abutalib
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Editors and Affiliations

  1. Adult Bone Marrow Transplant Service, Memorial Sloan Kettering Cancer Center, New York, NY, USA

    Miguel-Angel Perales

  2. Cancer Treatment Centers of America, Deerfield, IL, USA

    Syed A. Abutalib

  3. Center for Cancer and Immunology Research Children’s National and The George Washington University, Division Allergy and Immunology, Washington, DC, USA

    Catherine Bollard

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Abutalib, S.A., Gill, S.I. (2019). Most Recent Clinical Advances in CAR T Cell and Gene Therapy 2017/2018. In: Perales, MA., Abutalib, S., Bollard, C. (eds) Cell and Gene Therapies. Advances and Controversies in Hematopoietic Transplantation and Cell Therapy. Springer, Cham. https://doi.org/10.1007/978-3-319-54368-0_2

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  • DOI: https://doi.org/10.1007/978-3-319-54368-0_2

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