Researchers at the University of British Columbia have made a critical discovery that explains a debilitating side effect experienced by millions of people taking life-saving cholesterol medication. The breakthrough, which reveals precisely how statins cause muscle pain and weakness, opens the door to developing new, safer versions of the drug.
The Statin Side Effect Mystery Solved
Statins are a cornerstone of modern cardiovascular medicine, prescribed to lower cholesterol and dramatically reduce the risk of heart attacks and strokes. While most patients tolerate them well, a significant number report muscle-related side effects, ranging from mild fatigue to severe pain that can limit daily activities. Until now, the biological reason behind this common complaint remained unclear and debated within the scientific community.
A first-of-its-kind study from UBC’s Faculty of Medicine, in collaboration with the University of Wisconsin-Madison, has finally provided definitive evidence. Published in the journal Nature Communications, the research focused on atorvastatin, one of the most widely prescribed statins globally.
"Not only do they bind, they actually drastically alter the function of that protein in a way that can actually cause muscle damage," explained senior author Dr. Filip Van Petegem, a professor in UBC’s department of biochemistry and molecular biology. The team used advanced visualization techniques to see, at a molecular level, how the statin drug binds to a protein essential for proper muscle function and disrupts its activity.
A Path to a New Generation of Statins
This detailed 3D imaging is the key to the study's importance. For the first time, scientists can see exactly how statins interact with muscle tissue, distinct from how they work to lower cholesterol in the liver.
"We know exactly what parts of the statin are important for inhibiting cholesterol synthesis, and now that we have a very detailed 3D image of how the statins bind this critical muscle protein, we actually noticed that the way the statins interact is actually different between the two proteins," said Dr. Van Petegem. This crucial distinction provides a blueprint for designing future statins that retain their life-saving benefits for the heart while avoiding harmful interactions with muscles.
The discovery validates patient experiences and moves beyond previous unproven theories. "There have been many theories before on why statins affect muscles. None was really proven," Van Petegem noted. "I think now seeing it really bind, that’s definitely a first."
Why This Research Matters for Canada and Beyond
The implications of this finding are vast. More than 200 million people worldwide, including over 2.8 million in Canada, rely on statins to manage their cholesterol levels. For many individuals, especially those with a genetic predisposition to high cholesterol, diet and exercise alone are insufficient, making statin therapy essential for long-term health.
This research comes at a time when misinformation about statins circulates online, sometimes causing patients to question whether the drug's benefits outweigh its risks. By clearly identifying the cause of a major side effect, this work not only paves the way for improved medications but also provides concrete scientific clarity.
The ultimate goal, as outlined by the UBC team, is to use this molecular map to guide pharmaceutical development. The hope is to create next-generation statins that offer the same powerful protection against stroke and heart disease without the burden of muscle fatigue, ensuring more patients can safely continue this vital therapy for years to come.