Repurposing Decades-Old Drug Oxybutynin for Mitochondrial Disease Treatment

In an exciting development, scientists have found a potential new application for the well-known drug oxybutynin, traditionally used to treat bladder incontinence. Recent research indicates that this decades-old medication could play a vital role in tackling mitochondrial diseases, a group of severe genetic disorders that impair cellular energy production.

Published in the American Journal of Physiology-Cell Physiology, a team from Cornell University reported that oxybutynin can counteract mitochondrial dysfunction by boosting cellular glycolysis, thereby supporting healthy muscle growth. This discovery was made while testing a broad array of small molecules on muscle stem cells, which are crucial for muscle repair and regeneration.

Mitochondrial diseases, affecting roughly 1 in 5,000 individuals, often manifest early in life with symptoms like muscle weakness, neurological decline, heart problems, and sometimes shortened lifespan. Until now, there have been limited treatment options, and no cure exists.

The research team explained that, instead of repairing faulty mitochondria, oxybutynin rewires the cell’s energy pathways, enabling muscle cells to generate energy through glycolysis – a quick, glucose-based process. Their studies showed that oxybutynin binds to proteins involved in RNA processing, triggering a cascade of cellular changes that increase the transport of amino acids and glucose into cells.

Tests on mouse and human muscle stem cells revealed that oxybutynin treatment promoted cell proliferation and muscle fiber formation. The drug effectively helps these cells overcome the severe effects of Complex III mitochondrial dysfunction, a common form of mitochondrial disorder where ATP production halts.

The promising results are also being explored in clinical settings. Collaborations with Children's Hospital of Philadelphia involve testing oxybutynin on patient biopsy cells. While the drug has not yet been administered to human patients with mitochondrial diseases, its FDA approval for bladder issues suggests a more straightforward path toward future clinical trials.

This breakthrough offers hope for a new, accessible treatment avenue for patients with mitochondrial diseases—an area that previously lacked effective therapies. If ongoing studies confirm these benefits, oxybutynin could soon advance to trials, rapidly translating into potential treatment options.

Source: https://medicalxpress.com/news/2025-09-decades-drug-deployed-mitochondrial-diseases.html