Science Translational Medicine publishes first in-human results from Dr. Jeffrey Bluestone’s regulatory T cell-based (Treg) immunotherapy for type 1 diabetes – December 1, 2015

Science Translational Medicine recently published results from the first in-human phase 1 trial of a regulatory T cell-based (Treg) immunotherapy for type 1 diabetes from Dr. Jeffrey Bluestone’s (UCSF, San Francisco, CA) team. Using a technique developed by Dr. Bluestone, Tregs were isolated from 14 participants with newly diagnosed type 1 diabetes and expanded ex-vivo to achieve a 1,500-fold increase in number before being infused back into the participants. As background, Tregs can promote immune tolerance by “re-educating” the immune system to mitigate beta cell destruction while preserving disease-fighting immune capabilities. The results of this dose-ranging trial found that the infusion was well tolerated, with 25% of the peak Treg level remaining in the circulation after one year. In addition, C-peptide levels did not decrease up to two years after the Treg infusion in the cohorts with the two lowest doses. The study authors thus concluded that the results support advancement of the treatment into phase 2 trials. These findings seem reassuring with regards to safety and tolerability and with Dr. Bluestone’s leadership in the type 1 cure field, we’ll be watching closely to see how development of this therapy progresses and the magnitude of its efficacy. As a reminder, Dr. Bluestone’s work was recently validated by an impressive $10 million donation from philanthropist Sean Parker to establish the Sean N. Parker Autoimmune Research laboratory, led by Dr. Bluestone, within the UCSF Diabetes Center. We hope he approaches or has already approached the Chan/Zuckerburg Initiative (“Advancing human potential and promoting equality”), who will now be putting even more funds toward health than before with the establishment of this major $45 billion enterprise.

-- by Helen Gao, Melissa An, and Kelly Close