摘要
On August 30, 2017, the US Food and Drug Administration (FDA) released a news statement on its website titled ‘‘FDA approval brings first gene therapy to the United States”[1]. The article disclosed the use of FDA-approved Kymriah (tisagenlecleucel), a cell-based gene therapy developed by Novartis Pharmaceuticals Corporation, for certain pediatric and young adult patients with refractory or relapse acute lymphoblastic leukemia (ALL). ALL is a cancer of the bone marrow and blood, which progresses quickly and is the most common childhood cancer in the United States. Kymriah, which is a genetically modified autologous T cell immunotherapy, is approved for use in those up to 25 years of age with B cell precursor ALL. The individual patient’s own T cells are genetically modified with a new gene that contains a specific protein (chimeric antigen receptor, CAR) directing the T cells to target and kill leukemia cells with a specific antigen (cluster of differentiation 19, i.e. CD19) on the surface. In fact, Kymriah represents a kind of adoptive cellular immunotherapy using geneengineered T cells.“We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer,” said FDA Commissioner Scott Gottlieb, MD. From a historic point of view, cell therapy—or, more accurately, cellbased gene therapy—represents an important marker indicating that pharmacological disease intervention has entered a new era. The first era of disease intervention by drugs was based on chemical medicine, and the second era was based on biological drugs. Now the third era has arrived: cell therapy. This article outlines the historical progress and characteristics of three eras of pharmacological disease intervention, and analyzes the prospects for cell therapy.
