Caladrius Biosciences recently announced a collaboration with Sanford Research to develop the autologous regulatory T cell (Treg) therapy CLBS03 for type 1 diabetes. Caladrius (formerly known as NeoStem) has been collaborating with several renowned academic investigators, including the highly regarded Dr. Jeffrey Bluestone and Dr. Steven Gitelman (UCSF, San Francisco, CA) and Dr. Kevan Herold (Yale University, New Haven, CT) on this therapy. Dr. Gitelman presented phase 1 results at ADA 2014 showing a strong safety and tolerability profile (the study was not powered to evaluate efficacy). The initial focus of the Sanford partnership will be the phase 2 Sanford Project Trutina Study, which will evaluate the safety and efficacy of two doses of CLBS03 (10 and 20 million cells/kg) vs. placebo in 111 (! a big n) adolescents with recent onset type 1 diabetes. The primary endpoint will be preservation of C-peptide; enrollment is expected to begin as early as 1Q16. Based on an update from Caladrius Chief Medical Officer Dr. Douglas Losordo at GTC Bio in April, the study will involve some TrialNet investigators and include A1c and insulin usage as secondary endpoints. Dr. Losordo tentatively forecast that the therapy should lead to reduced insulin requirements and stated that achieving insulin independence even in some patients would be an “absolute grand slam.” The definition of success is an important question facing the type 1 diabetes biological “cure” field. Anything short of a complete cure could fall short of expectations given the amount of hype in this area in the past, but several speakers at recent conferences have stressed that even a therapy that does not achieve full insulin independence in all patients could still offer very meaningful benefits. Perception will be interesting on this front.
-- by Emily Regier and Kelly Close