The Teplizumab Saga: The Challenge of Not Getting Lost in Clinical Translation.
| Autor: | Chatenoud L; Université Paris Cité, CNRS, INSERM, Institut Necker Enfants Malades-INEM, Paris 75015, France lucienne.chatenoud@inserm.fr kevan.herold@yale.edu jbluestone@sonomabio.com., Herold KC; Departments of Immunobiology and Internal Medicine, Yale University, New Haven, Connecticut 06511, USA lucienne.chatenoud@inserm.fr kevan.herold@yale.edu jbluestone@sonomabio.com., Bach JF; Université Paris Cité, CNRS, INSERM, Institut Necker Enfants Malades-INEM, Paris 75015, France., Bluestone JA; Sonoma Biotherapeutics, South San Francisco, California 09080, USA lucienne.chatenoud@inserm.fr kevan.herold@yale.edu jbluestone@sonomabio.com. |
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| Jazyk: | angličtina |
| Zdroj: | Cold Spring Harbor perspectives in medicine [Cold Spring Harb Perspect Med] 2024 Sep 16. Date of Electronic Publication: 2024 Sep 16. |
| DOI: | 10.1101/cshperspect.a041600 |
| Abstrakt: | In November 2022, teplizumab became the first drug approved to delay the course of any autoimmune disease and to change the course of type 1 diabetes (T1D) since the discovery of insulin. The path to its approval took more than 30 years with both successes and failures along the way that would have normally led to its abandonment in other circumstances. Development of the drug was based on studies in preclinical models and parallels efforts in transplantation. From a series of innovative adaptations in response to issues related to adverse events and immunogenicity, humanized Fc receptors (FcR) nonbinding antibodies were developed with improved clinical outcomes and safety as well as new mechanisms. Importantly, as a result of these developments, teplizumab has been able to achieve efficacy over extended periods of time without global immune suppression. The approval of teplizumab represents a significant first step toward achieving escape from T1D and, in the future, reversal of the disease. (Copyright © 2024 Cold Spring Harbor Laboratory Press; all rights reserved.) |
| Databáze: | MEDLINE |
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