Muscular Dystrophy UK has recently allocated funding to support a pioneering research initiative led by Professor Rita Horvath, a distinguished neurogeneticist at the University of Cambridge. The project, which forms part of a broader £1.7 million investment into 12 new research ventures across England and Scotland, aims to explore the potential of dietary supplements as a novel treatment for mitochondrial myopathies—a group of disorders characterized by muscle weakness and wasting.
Professor Horvath’s research will specifically assess whether supplementing the diets of individuals with mitochondrial aminoacyl-tRNA synthetase (mt-aaRS) deficiencies with higher doses of certain amino acids could lead to significant symptom improvements. If the study proves successful, it could revolutionize the clinical management of these patients both in the UK and globally.
“We will perform further studies to optimize the dose and administration, but amino acids are cheap and considered food supplements. They can therefore easily be given to patients, even on the NHS,” said Professor Horvath. “In the long term, our results could change clinical practice for some mitochondrial diseases and will also benefit translational research and clinical trials in other mitochondrial and neuromuscular disorders.”
The research, which is set to span 36 months and costs £224,832, reflects Muscular Dystrophy UK’s commitment to funding high-quality studies that not only advance scientific knowledge but also aim to improve the quality of life for those affected by muscle-wasting conditions. Dr. Kate Adcock, Director of Research and Innovation at Muscular Dystrophy UK, expressed the charity’s enthusiasm for the project, noting that it aligns with their mission to back research with the potential to make a real-world impact on patient care.
This study stands as a significant stride towards innovative treatments that could potentially transform the landscape of care for mitochondrial myopathies and related neuromuscular disorders.