Customize Consent Preferences

We use cookies to help you navigate efficiently and perform certain functions. You will find detailed information about all cookies under each consent category below.

The cookies that are categorized as "Necessary" are stored on your browser as they are essential for enabling the basic functionalities of the site. ... 

Always Active

Necessary cookies are required to enable the basic features of this site, such as providing secure log-in or adjusting your consent preferences. These cookies do not store any personally identifiable data.

No cookies to display.

Functional cookies help perform certain functionalities like sharing the content of the website on social media platforms, collecting feedback, and other third-party features.

No cookies to display.

Analytical cookies are used to understand how visitors interact with the website. These cookies help provide information on metrics such as the number of visitors, bounce rate, traffic source, etc.

No cookies to display.

Performance cookies are used to understand and analyze the key performance indexes of the website which helps in delivering a better user experience for the visitors.

No cookies to display.

Advertisement cookies are used to provide visitors with customized advertisements based on the pages you visited previously and to analyze the effectiveness of the ad campaigns.

No cookies to display.

Health

Erythromycin shows promise in treatment of myotonic dystrophy type 1, study finds

A recent study published in eClinicalMedicine has shed light on the potential of erythromycin in the treatment of myotonic dystrophy type 1 (DM1). DM1 is a debilitating condition caused by a CTG repeat expansion in the DMPK gene, leading to toxic RNA expression and dysregulated splicing. In a preclinical study, it was found that erythromycin could mitigate the toxicity of abnormal RNA and improve splicing and motor function in DM1 model mice.

To translate these promising preclinical findings into practical applications for DM1 patients, a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial was conducted at three centres in Japan. The trial, which took place between Nov 29, 2019, and Jan 20, 2022, enrolled 30 adult patients with DM1 who were randomly assigned to receive either placebo or erythromycin at two different daily doses (500 mg or 800 mg) for 24 weeks.

The primary outcome of the trial was to evaluate the safety and tolerability of erythromycin, while secondary measures included splicing biomarkers, 6-minute walk test results, muscle strength, and serum creatinine kinase (CK) values. The findings revealed that treatment-related gastrointestinal symptoms were more common in the erythromycin group, but all adverse events were mild to moderate and resolved spontaneously. No serious safety concerns were identified.

Furthermore, the study observed a decrease in CK levels from baseline to week 24 in the overall erythromycin group compared to the placebo group. Although this difference was not statistically significant (p = 0.070), there were statistically significant improvements in splicing biomarkers MBNL1 (p = 0.048) and CACNA1S (p = 0.042) in the erythromycin treated groups compared to placebo.

The researchers concluded that erythromycin demonstrated favorable safety and tolerability profiles in patients with DM1. They emphasized the need for a well-powered phase 3 trial to further evaluate its efficacy, building on the preliminary findings from this study.

This study was funded by the Japan Agency for Medical Research and Development and holds promise for the future treatment of DM1, offering hope to individuals affected by this challenging condition.

LEAVE A RESPONSE

Your email address will not be published. Required fields are marked *