The rise, fall, and resurgence of immunotherapy in type 1 diabetes

Moufida Ben Nasr, Francesca D'Addio, Vera Usuelli, Sara Tezza, Reza Abdi, Paolo Fiorina

Research output: Contribution to journalArticle


Despite considerable effort to halt or delay destruction of β-cells in autoimmune type 1 diabetes (T1D), success remains elusive. Over the last decade, we have seen a proliferation of knowledge on the pathogenesis of T1D that emerged from studies performed in non-obese diabetic (NOD) mice. However, while results of these preclinical studies appeared to hold great promise and boosted patients' hopes, none of these approaches, once tested in clinical settings, induced remission of autoimmune diabetes in individuals with T1D. The primary obstacles to translation reside in the differences between the human and murine autoimmune responses and in the contribution of many environmental factors associated with the onset of disease. Moreover, inaccurate dosing as well as inappropriate timing and uncertain length of drug exposure have played a central role in the negative outcomes of such therapeutic interventions. In this review, we summarize the most important approaches tested thus far in T1D, beginning with the most successful preclinical studies in NOD mice and ending with the latest disappointing clinical trials in humans. Finally, we highlight recent stem cell-based trials, for which expectations in the scientific community and among individuals with T1D are high.

Original languageEnglish
Pages (from-to)31-38
Number of pages8
JournalPharmacological Research
Publication statusPublished - Aug 1 2015



  • B cells
  • Hematopoietic stem cells
  • Immunosuppression
  • Immunotherapy
  • NOD mice
  • T cells
  • Type 1 diabetes

ASJC Scopus subject areas

  • Pharmacology

Cite this

Ben Nasr, M., D'Addio, F., Usuelli, V., Tezza, S., Abdi, R., & Fiorina, P. (2015). The rise, fall, and resurgence of immunotherapy in type 1 diabetes. Pharmacological Research, 98, 31-38.