A new study is further assessing if a novel combination of drugs could help treat new onset type 1 diabetes

University of Florida researchers are ready to launch a new clinical study to examine if a combination of two U.S Food and Drug Administration (FDA) approved drugs, antithymocyte globulin (ATG) and granulocyte colony stimulating factor (GCSF), could benefit people with type 1 diabetes (T1D) to preserve their own residual beta cells when treatment is started within 100 days of diagnosis, known as recent onset T1D. The study will build upon a 2012 pilot clinical study where the researchers found that a number of people with T1D who started treatment with this combination between four months and two years of diagnosis benefitted from the combination immune therapy.

“The upcoming study is unique in that it represents the first combination of two FDA-approved agents to demonstrate preservation of beta cell function in people with established T1D. Based on these exciting results, we are moving forward with a larger confirmatory study,” said primary investigator Michael Haller, MD of University of Florida.

The new study differs from the pilot study in that the combination will be given to patients with the hope that intervening earlier might have a larger effect. The primary objective is to gauge safety of this combination therapy, but the trial will also look for maintenance or enhancement of residual beta cell function as evidenced by c-peptide production and insulin use.

According to JDRF’s Director of Discovery Research, Andrew Rakeman, “It is becoming increasingly clear that a combination approach will likely be necessary to stop progression or reverse a complex disease like T1D. This particular combination is attractive for a number of reasons – it uses two FDA-approved drugs, which means there’s years of clinical experience relating to their safety, that has helped to expedite their use in T1D. Plus, the positive results from the pilot trial give a strong suggestion that this particular combination could be safe and beneficial for T1D.”

If this study is successful it will set precedence for the effectiveness of immune targeted combination therapies in the recent-onset stage of the disease, where the immune system may be hardest to control. This, coupled with immune mechanistic insights gained from this trial, could potentially inform other follow-on studies, including using ATG/GCSF as a prevention therapy in people at risk for developing T1D and using the combination for a longer period of time.

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