Posted on 02/23/2025 10:09:51 AM PST by ConservativeMind
A research team found that an osteoporosis drug might counter a rare genetic mutation underlying a type of heart disease. The results could have implications for treating other rare diseases.
Dilated cardiomyopathy is a group of disorders defined by weak cardiac muscles. DCM often causes life-threatening complications as the heart struggles to pump blood.
The body's natural response to a weak heart is to force it into overdrive to ensure blood delivery to every corner of the body.
"Maybe 30% or 40% of DCM is due to genetic mutation," Sadek said. "We don't have any mutation-specific therapies so far."
The heart's pump is powered by a "motor" made up of many constituent parts—proteins.
"A mutation might bend that protein out of shape and make it unable to work properly in that motor," Sadek said. "If you find a drug that pushes the mutant protein back into shape, you can correct the structure."
The team focused on K210del, the first DCM-associated mutation discovered, to investigate how its flawed shape prevents the heart's motor from running smoothly.
Sadek's team became the first to create a 3D model of K210del and compare it to its healthy counterpart to identify where it is misshapen and how it interacts with surrounding proteins to slow down the motor.
Next, they harnessed the power of supercomputers and artificial intelligence to screen 2,000 FDA-approved drugs to see if any of them were likely to bind to the misshapen protein and nudge it back to its correct position.
"The top five or six drugs were all used for osteoporosis," Sadek said.
After further testing in cell cultures and animal models, they refined the results.
"Only one of the drugs, risedronate, corrected the protein shape back to normal," Sadek said.
(Excerpt) Read more at medicalxpress.com ...
It helps restore bone mass in people, but it also has serious side effects, for some people.
Still, if your body needs the fix this appears to create, it might be worth exploring.
It seems rare that a substance can force proteins back into their perfect shape.
They sure are desperate to find other uses for these expensive drugs.
To be fair, it looks like the generic is as little as $40 a month.
Bkmk
When a new use for it is found, don’t they get to modify it a bit and then remarket it under a new patent to treat the new condition?
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