In a study published in December 2015 in a peer review journal researchers from Japan and Poland found that a commonly used antibiotic might assist in the treatment of Myotonic Dystrophy. This is a sort of interesting discovery as there is no treatment identified to treat the disease. Treatment now consists of reducing symptoms. There are several other approaches that are in development maybe as soon as the end of this year. Here is a link to another promising therapy as well Cagumycin
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.
This drug has been used in the past on patients with myotonic dystrophy to help improve stomach issues. Erythromycin is also reported to stimulate gastrointestinal motor activity via effects on motilin receptors. In a previous study, the erythromycin ethylsuccinate ester was tested to improve gastric emptying in DM1 patients when administered at a dose of 50–200 mg/day for 4 weeks.36 While no signiﬁcant change in the gastric emptying rate was observed in the study, some gastrointestinal symptoms, including nausea, ability to eat, and diarrhea, were attenuated in DM1 patients. Although the dosage of erythromycin was relatively low and skeletal muscle symptoms were not evaluated, such improvements might have occurred through neutralization of toxic RNA.
Well the results looked very good. 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-splicing 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!!!
The research team in Japan is planning human studies. We will post information when this becomes available. Most likely the studies will be based in Osaka Japan. HEres an update on the research end. As of this update to the article in 2019 there are no human studies planned. Most of the resources of the community are going toward more complex ways to defeat the disease and there seems little interest in this simple, inexpensive approach. We are not sure why this is.
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
Summary Pages in Languages other than English.
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Spanish Español Posible Tratamiento para la Distrofia Miotónica
Russian русский Потенциальное лечение миотонической дистрофии
Portugese Português Potencial tratamento para Distrofia Miotônica
Korean 한국어 근긴장성 이영양증을 위한 잠재적 치료제
Japanese 日本語 筋緊張性ジストロフィーの治療剤
Italian italiano Potenziale trattamento per la Distrofia Miotonica
Hind हिंदी भामोटोननक डडस्ट्रोपी का सॊबावित उऩचाय
German Deutsche Mögliche Behandlung für Muskeldystrophie
French Francis Traitement potentiel pour la Dystrophie Myotonique
Arabic علاج محتمل لمرض الضمور العضلي العربية