Davide Frumento1, Moufida Ben Nasr2,3, Basset El Essawy4, Francesca D'Addio1,2, Gian Vincenzo Zuccotti5, Paolo Fiorina6,7. 1. DITID, San Raffaele Hospital, Milan, Italy. 2. International Center for T1D, Pediatric Clinical Research Center Fondazione Romeo e Enrica Invernizzi, Department of Biomedical and Clinical Science L. Sacco, University of Milan, Milan, Italy. 3. Nephrology Division, Boston Children's Hospital, Harvard Medical School, Enders Building 5th floor Room EN511, 300 Longwood Ave, Boston, MA, USA. 4. Medicine, Al-Azhar University, Cairo, Egypt. 5. Pediatric Clinical Research Center Fondazione Romeo ed Enrica Invernizzi, Department of Biomedical and Clinical Science L. Sacco, University of Milan, Milan, Italy. 6. International Center for T1D, Pediatric Clinical Research Center Fondazione Romeo e Enrica Invernizzi, Department of Biomedical and Clinical Science L. Sacco, University of Milan, Milan, Italy. paolo.fiorina@childrens.harvard.edu. 7. Nephrology Division, Boston Children's Hospital, Harvard Medical School, Enders Building 5th floor Room EN511, 300 Longwood Ave, Boston, MA, USA. paolo.fiorina@childrens.harvard.edu.
Abstract
INTRODUCTION: Although many approaches have been tested to overcome the insulin dependence caused by the pancreatic β-cells destruction observed in individuals affected by type 1 diabetes (T1D), medical research has largely failed to halt the onset or to reverse T1D. METHODS: In this work, the state of the art of immunotherapy will be examined, and the most important achievement in the field will be critically discussed. Particularly, we will focus on the clinical aspect, thus avoiding the tedious preclinical work done in NOD mice, which has been so poorly translated to the bedside. CONCLUSIONS: Stem cell therapies achieved thus this far the most promising results, while immune ablation and standard immunosuppressants did not maintain the premises of preclinical results. The next step will be to generate a feasible and safe clinical approach in order to cure the thousands of patients affected by T1D.
INTRODUCTION: Although many approaches have been tested to overcome the insulin dependence caused by the pancreatic β-cells destruction observed in individuals affected by type 1 diabetes (T1D), medical research has largely failed to halt the onset or to reverse T1D. METHODS: In this work, the state of the art of immunotherapy will be examined, and the most important achievement in the field will be critically discussed. Particularly, we will focus on the clinical aspect, thus avoiding the tedious preclinical work done in NOD mice, which has been so poorly translated to the bedside. CONCLUSIONS: Stem cell therapies achieved thus this far the most promising results, while immune ablation and standard immunosuppressants did not maintain the premises of preclinical results. The next step will be to generate a feasible and safe clinical approach in order to cure the thousands of patients affected by T1D.
Entities:
Keywords:
Clinical; Immunotherapy; Trials; Type 1 diabetes
Authors: Antoinette Moran; Brian Bundy; Dorothy J Becker; Linda A DiMeglio; Stephen E Gitelman; Robin Goland; Carla J Greenbaum; Kevan C Herold; Jennifer B Marks; Philip Raskin; Srinath Sanda; Desmond Schatz; Diane K Wherrett; Darrell M Wilson; Jeffrey P Krischer; Jay S Skyler; Linda Pickersgill; Eelco de Koning; Anette-G Ziegler; Bernhard Böehm; Klaus Badenhoop; Nanette Schloot; Jens Friis Bak; Paolo Pozzilli; Didac Mauricio; Marc Y Donath; Luis Castaño; Ana Wägner; Hans Henrik Lervang; Hans Perrild; Thomas Mandrup-Poulsen Journal: Lancet Date: 2013-04-05 Impact factor: 79.321
Authors: V V Zhukouskaya; C Eller-Vainicher; A P Shepelkevich; Y Dydyshko; E Cairoli; I Chiodini Journal: J Endocrinol Invest Date: 2015-04-12 Impact factor: 4.256
Authors: Kevan C Herold; Stephen Gitelman; Carla Greenbaum; Jennifer Puck; William Hagopian; Peter Gottlieb; Peter Sayre; Peter Bianchine; Emelita Wong; Vicki Seyfert-Margolis; Kasia Bourcier; Jeffrey A Bluestone Journal: Clin Immunol Date: 2009-05-14 Impact factor: 3.969
Authors: Sonja Kleffel; Andrea Vergani; Sara Tezza; Moufida Ben Nasr; Monika A Niewczas; Susan Wong; Roberto Bassi; Francesca D'Addio; Tobias Schatton; Reza Abdi; Mark Atkinson; Mohamed H Sayegh; Li Wen; Clive H Wasserfall; Kevin C O'Connor; Paolo Fiorina Journal: Diabetes Date: 2014-09-03 Impact factor: 9.461
Authors: Anne M Pesenacker; Virginia Chen; Jana Gillies; Cate Speake; Ashish K Marwaha; Annika Sun; Samuel Chow; Rusung Tan; Thomas Elliott; Jan P Dutz; Scott J Tebbutt; Megan K Levings Journal: JCI Insight Date: 2019-03-21
Authors: Sunil M Kurian; Kevin Ferreri; Chia-Hao Wang; Ivan Todorov; Ismail H Al-Abdullah; Jeffrey Rawson; Yoko Mullen; Daniel R Salomon; Fouad Kandeel Journal: PLoS One Date: 2017-10-02 Impact factor: 3.240
Authors: Seung-Soo Kim; Adam D Hudgins; Jiping Yang; Yizhou Zhu; Zhidong Tu; Michael G Rosenfeld; Teresa P DiLorenzo; Yousin Suh Journal: PLoS One Date: 2021-09-16 Impact factor: 3.240