New Type 2 Diabetes Treatment Shows Promise in First Human Study

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New Type 2 Diabetes Treatment Shows Promise in First Human Study

Mitchel L. Zoler, Ph.D., for Medscape

November 18, 2021

Key Takeaways

  • Patients with type 2 diabetes who received daily treatment with sodium phenylbutyrate for 2 weeks had significant improvements in peripheral insulin sensitivity and glucose oxidation.
  • The treatment appeared safe, with no reported adverse events.

Why This Matters

  • The results show, for the first time in humans, that treatment with an agent that cuts levels of branched-chain amino acids by boosting their breakdown produces substantial beneficial effects on glucose metabolism in patients with type 2 diabetes.
  • The findings are proof-of-concept that treatments that target branched-chain amino acids may be a new management strategy for patients with type 2 diabetes.

Study Design

  • A single-center, randomized, double-blind, 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.
  • On top of their background glucose-lowering medications, participants received 4.8 g/m2/day sodium phenylbutyrate or placebo divided into three doses per day for 2 weeks, followed by a 6-8 week washout period, and then 2 weeks on the alternative treatment.

Key Results

  • The primary outcome was change from baseline in peripheral insulin sensitivity, measured by the insulin-stimulated glucose disposal rate.
  • Peripheral insulin sensitivity improved after 2 weeks on sodium phenylbutyrate by a significant 27% compared with placebo (P = .0155).
  • Sodium phenylbutyrate also significantly improved several secondary measures compared with placebo including carbohydrate-driven muscle mitochondrial oxidative capacity, whole-body insulin-stimulated carbohydrate oxidation, and reduced plasma levels of branched-chain fatty acids.

Limitations

  • The 16 evaluable patients were skewed by sex, with three women and 13 men participating.
  • The study focused on treatment effects in skeletal muscle and liver and did not assess adipose tissue.
  • Background therapy was not uniform across all 16 participants.

Disclosures

  • The study received no commercial funding.
  • The authors had no disclosures.

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