ABSTRACT
Introduction
Medicine stands at the threshold of a new era heralded by the vast potential of cell engineering. Like advances made possible by genetic engineering, current prospects for purposeful control of cell functions through cell engineering may bring breakthroughs in the treatment of previously intractable diseases.
Areas covered
Engineering of cytotoxic T cells for expression of chimeric antigen receptors (CARs) instructs them to attack and destroy malignant cells and thus provides an exciting new approach in oncology. A decade of practical experience and first-in-human trials encourage the search for new and broader uses of CAR technology, including in autoimmune diseases.
Expert opinion
Systemic lupus erythematosus is an example of a broader category of autoimmune diseases, for which cell engineering will provide a powerful new therapeutic approach. This article describes different types of CAR T cell strategies that will provide new treatment options for patients with autoimmune diseases and replace conventional therapies.
Article highlights
Autoimmune diseases are common and often life-threatening but generally lack effective treatments,
B cells are at the center of autoimmune pathology in systemic lupus erythematosus and related diseases, but attempts to treat lupus with recombinant antibodies to CD20, a B cell marker, did not meet expectations.
Cytotoxic T cells can be engineered to express chimeric antigen receptors (CARs) to CD19 on B cells, thus yielding sustained and highly effective cell-based immunotherapy that provides an improved approach to B cell depletion.
Anti-CD19 CAR T cells are only one example of CAR T cells that show efficacy in animal models of autoimmune disease, and anti-CD19 CAR T cells were remarkably successful in their first use in a lupus patient.
Scenarios are plausible and predictable, in which cell engineering will provide long-sought cures for severe, treatment-refractory autoimmune diseases.
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Declaration of interests
The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.
Reviewer disclosures
Peer reviewers on this manuscript have no relevant financial relationships or otherwise to disclose.