2025 Mar 14, 20:39
Researchers from Osaka University in Japan have potentially found a cure for the genetic disease Myotonic dystrophy type 1 (DM1). DM1 is the most common form of muscular dystrophy, characterized by progressive muscular weakness. Currently, there is no treatment available for this disease, despite previous efforts. However, the innovative approach used by the team at Osaka University may change that. The researchers used drug repositioning screening to identify potential therapies for DM1
This method involves testing drugs that are already used for one purpose to see their effects on other diseases. In the case of DM1, the team found that erythromycin, an antibiotic widely used for other purposes, may be effective in improving splicing abnormalities, which are a key feature of DM1. After conducting successful preclinical trials in cell and mouse models, the researchers moved on to a clinical trial on 30 patients with DM1 to study the safety, tolerability, and efficacy of erythromycin. The patients were divided into three groups - a placebo group, a low-dose group (500 mg), and a high-dose group (800 mg). The safety data from the trial were encouraging, and although the efficacy testing showed few significant results, some patients did show major improvements in splicing markers after receiving erythromycin. The researchers noticed that the dosage of erythromycin may play a role in its efficacy, as some splicing markers improved significantly at one dose but not the other
Despite the small sample size, the researchers remain optimistic about the potential of erythromycin as a therapeutic option for DM1. They believe that further trials should be conducted to determine whether this widely used antibiotic can successfully treat DM1, or at least a subset of patients. In conclusion, the researchers from Osaka University have made significant progress in identifying a possible new treatment for DM1. Their findings suggest that erythromycin could be a promising therapeutic option, and further trials should be conducted to explore its efficacy in treating DM1.