Stopping diabetes in its tracks: autologous non-myeloablative stem cell transplantation.
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.
Fousteri
Georgia
G
La Jolla Institute for Allergy and Immunology, 9420 Athena Circle, La Jolla, CA 92037, USA. fousteri@liai.org
Hayek
Alberto
A
von Herrath
Matthias
M
D001323:Autoantibodies / Q000627:therapeutic use; D002096:C-Peptide / Q000097:blood; D017321:Clinical Trials, Phase I as Topic; D017322:Clinical Trials, Phase II as Topic; D003131:Combined Modality Therapy; D003520:Cyclophosphamide / Q000627:therapeutic use; D003922:Diabetes Mellitus, Type 1 / Q000628:therapy*; D005500:Follow-Up Studies; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D008954:Models, Biological; D013234:Stem Cells / Q000166:cytology*; D050378:T-Lymphocytes, Regulatory / Q000276:immunology; D013997:Time Factors; D019172:Transplantation Conditioning; D014182:Transplantation, Autologous; D016896:Treatment Outcome
D001323:Autoantibodies; D002096:C-Peptide; D007166:Immunosuppressive Agents; C006606:anti-thyroglobulin; D003520:Cyclophosphamide