Fousteri Georgia, Hayek Alberto, von Herrath Matthias
La Jolla Institute for Allergy and Immunology, 9420 Athena Circle, La Jolla, CA 92037, USA.
Regen Med. 2007 Sep;2(5):845-51. doi: 10.2217/17460751.2.5.845.
A recent report in this year's April issue of Journal of the American Medical Association describes an unprecedented success in delaying insulin dependence in patients with recent-onset Type 1 diabetes after non-myeloablative immune suppression with cyclophosphamide and antithymocyte globulin followed by autologous stem cell transplantation. In this study, 14 out of 15 patients became insulin-independent, which lasted up to 35 months. Concomitantly, C-peptide levels increased substantially compared with preintervention values. Treatment of autoimmune disorders, and in particular Type 1 diabetes, constitutes a complex balancing act between suppressing autoaggressive responses strongly and permanently enough, while circumventing much-feared long-term side effects from chronic immunosuppression. This clinical Phase I/II trial is relevant to fine-tuning interventive protocols and contributing to the further development of suitable combination therapies to prevent and treat Type 1 diabetes.
今年4月号的《美国医学会杂志》上最近发表的一篇报告描述了一项前所未有的成功案例,即对近期发病的1型糖尿病患者进行非清髓性免疫抑制,使用环磷酰胺和抗胸腺细胞球蛋白,随后进行自体干细胞移植,成功延缓了胰岛素依赖。在这项研究中,15名患者中有14名不再依赖胰岛素,这一情况持续了长达35个月。与此同时,与干预前的值相比,C肽水平大幅上升。自身免疫性疾病的治疗,尤其是1型糖尿病的治疗,是一种复杂的平衡行为,既要足够强烈和持久地抑制自身攻击性反应,又要避免慢性免疫抑制带来的令人担忧的长期副作用。这项临床I/II期试验对于微调干预方案以及推动预防和治疗1型糖尿病的合适联合疗法的进一步发展具有重要意义。