Pharm'Up 
A new study published in the journal Trials explores the potential of metformin, a widely-used diabetes medication, as an effective and affordable treatment for Autosomal Dominant Polycystic Kidney Disease (ADPKD). ADPKD is a genetic disorder that causes cysts to grow on the kidneys, leading to progressive kidney function decline and, in many cases, end-stage renal disease (ESRD).
The IMPEDE-PKD Trial
The Implementation of Metformin theraPy to Ease Decline of Kidney Function in Polycystic Kidney Disease (IMPEDE-PKD) trial is a long-term, randomized, placebo-controlled Phase 3 clinical trial investigating the efficacy and safety of metformin in patients with ADPKD. The trial, sponsored by The University of Queensland, aims to determine if metformin can slow the rate of kidney function decline, reduce morbidity and mortality, and improve the quality of life for patients.
The trial has enrolled 1174 adults between 18 and 70 years old with an estimated glomerular filtration rate (eGFR) between 38 and 90 mL/min/1.73 m². Participants are randomly assigned to receive either extended-release metformin or a placebo, in addition to their standard care, over a two-year period.
Why Metformin?
While there is currently only one FDA-approved drug for ADPKD, tolvaptan (Jynarque), its use is limited by side effects, high cost, and restricted availability. Metformin, on the other hand, is an inexpensive and widely accessible drug used globally for type 2 diabetes. Preliminary research and preclinical studies have shown that metformin may have a positive impact on ADPKD by targeting the mechanisms that cause cyst growth.
For example, a 2021 study cited in the article demonstrated a slower annual decline in eGFR in patients treated with metformin compared to those on a placebo. If the results of the IMPEDE-PKD trial are positive, metformin could provide a much-needed, affordable, and accessible treatment option for a disease that currently has a significant impact on public health and healthcare spending.
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