Frumento Davide, Ben Nasr Moufida, El Essawy Basset, D'Addio Francesca, Zuccotti Gian Vincenzo, Fiorina Paolo
DITID, San Raffaele Hospital, Milan, Italy.
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.
J Endocrinol Invest. 2017 Aug;40(8):803-814. doi: 10.1007/s40618-017-0641-y. Epub 2017 Mar 4.
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.
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.
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.
尽管已经测试了许多方法来克服1型糖尿病(T1D)患者中观察到的胰腺β细胞破坏所导致的胰岛素依赖,但医学研究在很大程度上未能阻止T1D的发病或逆转病情。
在这项工作中,将研究免疫疗法的最新进展,并对该领域最重要的成果进行批判性讨论。特别是,我们将专注于临床方面,从而避免在非肥胖糖尿病(NOD)小鼠中进行的繁琐的临床前工作,而这些工作在临床应用中效果不佳。
迄今为止,干细胞疗法取得了最有希望的结果,而免疫消融和标准免疫抑制剂并未维持临床前研究结果所预期的效果。下一步将是制定一种可行且安全的临床方法,以治愈数以千计受T1D影响的患者。