Target: Type 1 Diabetes

Prevalence and Unmet Medical Need

  • 18,000 children under 20 in U.S. with new onset T1D per year1
  • 3% annual growth rate worldwide2
  • Autoimmune destruction of insulin-producing (beta cells)
  • No curative treatments for T1D, only lifelong insulin therapy
  • Diabetes is leading cause of kidney failure, new cases of adult blindness, and non-traumatic lower-limb amputations

Type 1 Diabetes Program Development

Type 1 diabetes (also known as insulin dependent diabetes or juvenile diabetes) is caused by the autoimmune destruction of insulin-producing beta cells of the pancreas.

We have established a collaboration with UCSF and the laboratories of Drs. Jeffrey Bluestone and Qizhi Tang, to collaborate on the development of a therapy for the treatment of type 1 diabetes.

In this collaboration, NeoStem will sponsor and manufacture a Treg product consisting of polyclonally expanded Tregs for the planned Phase 2 trial to treat patients newly diagnosed with type 1 diabetes. The collaboration also includes research efforts to develop the next generation of Treg products for therapeutic use. Dr. Bluestone, the Study Director, and Dr. Kevan Herold, the Study Principal Investigator (Yale University), completed the Phase 1 study of autologous Tregs in type 1 diabetes and reported the Phase 1 results at the American Diabetes Association Scientific Sessions in Chicago in June 2014.

The Trutina Study: Phase 2 in Adolescents
Design Double blind, placebo controlled, randomized (1:1:1) for adolescent patients with recent onset T1D ages 12 to 18
Primary endpoint Preservation of C-peptide at 52 weeks in comparison to placebo
Powering 80% power to detect improvement in mixed meal tolerance test stimulated c-peptide in comparison to placebo, adjusted for baseline
Study Size United States, ~11 sites, planned enrollment of 111 patients
Treatment 2 dose groups of NBS03D (single dose autologous ex-vivo expanded polyclonal T Regulatory cell therapy): 10 and 20 million cells/kg
Control Matching infusion

 

  1. Hamman RF, et al. Diabetes Care. 2014; Sosenko JM, et al. Diabetes Care. 2008; Palmer JP. Diabetes/metabolism research and reviews. 2009
  2. The DIAMOND Project Group. Diabetic Medicine. 2006;23:857-866.
NeoStem - Cell Therapy Development - Clinical