Promising New Treatment for Myotonic Dystrophy Type 1: Potential of Heart Medications Explored

2024-01-04 01:48:00
“We believe that the calcium channel is a new therapeutic target and, if we can target it correctly, pharmacologically, it will improve muscle function and health,” said one of the experts.

(Dennis Thompson- HealthDay News) — A common class of drugs to treat heart problems may be able to improve one of the most common forms of muscular dystrophy, a new study in mice suggests.

Myotonic dystrophy type 1 (DM1) is caused by an abnormal RNA that affects the function of calcium channel receptors, which help convert nerve cell impulses into chemical signals that guide muscle movement, the researchers explained. .

Given the above, a type of heart medication called a calcium channel blocker might be able to eliminate the “noise” caused by this abnormal RNA, they reasoned.

These drugs successfully alleviated the symptoms of T1DM in laboratory mice bred to have the genetic problem that causes that form of muscular dystrophy, according to the results, which were published in the Jan. 2 issue of the Journal of Clinical Investigation.

“We believe that the calcium channel is a new therapeutic target and, if we can target it correctly, pharmacologically, it will improve muscle function and health,” the researchers wrote.

People with T1D suffer from muscle weakness and prolonged muscle tension, making it difficult for muscles to relax following use, they said in supporting notes.

Muscle problems caused by the disease affect the eyes, heart and brain, eventually leading to problems walking, swallowing and breathing.

More than two decades ago, co-investigator Dr. Charles Thornton, a neurologist at the University of Rochester Medical Center, discovered how a genetic defect causes T1D. A genetic “stutter” results in thousands of code repetitions in a segment of chromosome 19, according to Thornton’s previous research.

In turn, this defect causes an accumulation of abnormal RNA that interferes with healthy muscle function. To investigate this cycle and better understand T1D, researchers bred laboratory mice that had four of the genetic defects found in T1D, specifically, in genes associated with calcium and chloride channels.

“Myotonic dystrophy is a really complicated disorder that can be seen almost as an aggregate of many diseases,” co-investigator John Lueck, an associate professor at the University of Rochester Medical Center, said in a University of Rochester news release.

They then tried treating these mice with calcium channel blockers that are typically used to treat high blood pressure, heart rhythm problems, and migraines.

Verapamil, a calcium channel blocker used to treat blood pressure and chest pain, caused the mice to quickly regain muscle function and appear as healthy as normal laboratory mice, the researchers reported.

“Our research also suggests that muscle impairment in T1D is potentially mitigated by common clinically available calcium channel blockers, and that calcium channel modulation is a potential therapeutic strategy,” Lueck said.

However, the researchers noted that verapamil should not be considered for use in humans with T1D, as it may have harmful side effects. Additionally, research in mice doesn’t always work in humans.

“Our goal now is to find the right and safe calcium channel blocker that does the job, and we believe it exists,” the researchers concluded.

More information

The Cleveland Clinic has more information on calcium channel blockers.

SOURCE: University of Rochester Medical Center, news release, January 2, 2023

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