Stopping diabetes in its tracks: autologous non-myeloablative stem cell transplantation

Regen Med. 2007 Sep;2(5):845-51. doi: 10.2217/17460751.2.5.845.

Abstract

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.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Autoantibodies / therapeutic use
  • C-Peptide / blood
  • Clinical Trials, Phase I as Topic
  • Clinical Trials, Phase II as Topic
  • Combined Modality Therapy
  • Cyclophosphamide / therapeutic use
  • Diabetes Mellitus, Type 1 / therapy*
  • Follow-Up Studies
  • Hematopoietic Stem Cell Transplantation*
  • Humans
  • Immunosuppressive Agents / therapeutic use
  • Models, Biological
  • Stem Cells / cytology*
  • T-Lymphocytes, Regulatory / immunology
  • Time Factors
  • Transplantation Conditioning
  • Transplantation, Autologous
  • Treatment Outcome

Substances

  • Autoantibodies
  • C-Peptide
  • Immunosuppressive Agents
  • anti-thyroglobulin
  • Cyclophosphamide