Medical Technology

New Type 2 Diabetes Treatment Shows Promise in First Human Study

The most important takeaways

  • Patients suffering from type 2 diabetes who received daily treatment with sodium phenylbutyrate for 2 weeks had significant improvements in peripheral insulin tolerance and glucose oxidation.

  • The treatment was safe and there were no adverse reactions.

What is it important?

  • The results showed that patients suffering from type 2 diabetes treated with an agent that reduces branched-chain amino acid levels through increasing their breakdown have significant positive effects on glucose metabolism.

  • The findings provide proof of concept that treatments that target branched-chain amino acids could be a new management strategy for patients suffering from type 2 diabetes.

Study Design

  • A single-center, double-blind, randomized, single-center, placebo-controlled, crossover study with 18 enrolled patients and 16 patients with evaluable results who were included in the analysis.

  • Patients were an average of 66 years old and had been diagnosed with type 2 diabetes for an average of 1.5 years.

  • In addition to their background diabetes-lowering medication, participants received 4.8 grams 2/day sodium phenylbutyrate or placebo , divided into three doses daily for 2 weeks, followed by a 6-8 week washout, and then 2 weeks on the alternative treatment.

Key Results

  • The primary outcome was a shift in the sensitivity of peripheral insulin to baseline, as measured by insulin-stimulated sugar disposal rate.

  • The peripheral insulin sensitivity increased after 2 weeks of sodium phenylbutyrate, which was a significant 27% compared with placebo ( P = .0155).

  • Other secondary measures that were significantly improved by sodium phenylbutyrate over placebo included carbohydrate-driven mitochondrial oxidative capability and whole-body insulin stimulation of carbohydrate oxidation, and lower levels in plasma of branched-chain fat acids.


  • The 16 patients who were evaluated were skewed by sexual activity. Three women and 13 men were involved.

  • The study was restricted to the effects of treatment on the liver and skeletal muscle but did not include a review of the adipose tissues.

  • The background therapy was not the same for all 16 participants.


This is a brief summary of a research study on preprints composed by a group of researchers at the Department of Nutrition and Movement Sciences at Maastricht University, The Netherlands, and their collaborators on Preprints in collaboration with The Lancet, provided to you by Medscape. This study has not yet been peer-reviewed. The complete study’s content is available onSSRN First Look.

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