A look to the future: Prediction, prevention, and cure including islet transplantation and stem cell therapy

Anna Casu, Massimo Trucco, Massimo Pietropaolo

Research output: Contribution to journalArticlepeer-review


There is common agreement indicating that the occurrence of multiple antibodies against islet autoantigens serves as a surrogate marker of disease in primary or secondary intervention strategies aimed at halting the disease process. To date, a number of intervention strategies are in the pipeline and some of them seem promising. These therapies include anti-CD3 humanized monoclonal antibody; antilymphocyte serum; and a number of antigen-specific therapies, such as oral insulin. The DPT-1 has recently performed a subgroup analysis suggesting potential benefit of oral insulin for relatives with high insulin autoantibody titers. A trial conducted by Neurocrine using an altered insulin peptide ligand of insulin B:9-23 is currently underway in humans in which the peptide is delivered without the use of an adjuvant or other immunomodulation. Many of these antigen-specific therapies for T1DM and other autoimmune diseases have not been approved. There is both a growing effort and a large opportunity for exploring new specific strategies alone or in combination with immunomodulation. It is possible that gene-engineered cell therapeutics if combined with immunotherapy may effectively replace the pancreatic β-cell loss in T1DM. The hope to induce pancreatic islet regeneration and, ultimately, to transplant insulin-producing cells with a sustained secretagogue capacity propels confidence that the cure of T1DM is within reach.

Original languageEnglish
Pages (from-to)1779-1804
Number of pages26
JournalPediatric Clinics of North America
Issue number6
Publication statusPublished - Dec 2005

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health


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