In a study published in December 2015 in a peer review journal researchers from Japan found that a commonly used antibiotic might assist in the treatment of Myotonic Dystrophy. This is a sort of stunning discovery as there is no treatment identified to treat the disease. Treatment now consists of reducing symptoms.
The researchers first began by screening antibiotics. In a screen of 20 antibiotics 2-3 were found to have some potential with the disease.When screening the drugs they first used mice cells and lab equipment to find the most promising compounds (drugs). . Erythromyicin was found to have the highest attraction to the RNA CUG expansion (The opposite of CTG repeats in the DNA) Erythromycin was the drug that the researchers chose to study. Click here for the screening graph Muscleblind and Various antibiotics and compounds
The researchers were looking at drug re-positioning or “Off label Use” of the some drugs. That is that were looking for already approved safe drugs that might, as a side effect, help to treat myotonic dystrophy. Erythromycin seemed to fit the bill.
Well the results were Stunning. The drug Erythromycin reduced the Foci Formation in the cells of mice. The foci are thought to be where the RNA’s that are defective are binding with needed proteins causes the cells to “Clog” and reduce there critical functions. So the bad foci’s are reduced. The drug also reduces mi-ssplicing events or errors in the cells producing the right components.
Importantly the oral administration of This drug (the easiest way to take it) in Mice that had myotonic dystrophy showed the reversal of this mis-splicing events. It also showed improvement of the myotonia that the mice had with no toxic effects! “Long term oral administration of erythomycin at the dose used in humans also improved improved the splicing abnormality in DM1 model mice”
Now the limitations. This drug erythromycin affects the heart. That’s bad. Myotonic Dystrophy patients, many of them have heart problems. Some people with this disease just die of sudden cardiac failure. So don’t do anything without discussing this with your doctor and cardiologist!!!
As their last statement the researchers said ” Interpretation: Oral erythromycin treatment, which has been widely used in humans with excellent tolerability, may be a promising therapy for DM1 (myotonic dystrophy type 1)
Here is the study ==> Promising treatment for Myotonic Dystrophy
Here is a supplement on treatment protocol ==> Dosing versus results