Author: Aldiyarbek Nurlan
Editor: Merentsova Anastasia
The study showed that the use of extracellular vesicles as bioactive mediators ("transporting" molecules) for HDAC inhibitors (drugs that promote cell cycle arrest, differentiation and / or apoptosis) has a rather effective therapeutic effect in patients with DMD.
Scientists discovered the promise of this treatment about 20 years ago, and a lot of preclinical work was carried out, which opened the way for this clinical study of boys with DMD. However, the therapeutic potential of HDAC inhibitors (HDACi) is still limited by its systemic side effects.
This study involved the treatment of boys with DMD with HDACi, but at suboptimal doses due to the risk of side effects.
Scientists hope that extracellular vesicles can become an extracellular, non-immunogenic "vehicle" for the local delivery of bioactive particles, namely HDACi, to affected muscles, thereby overcoming unwanted secondary effects caused by chronic high doses.
“We believe this new approach to the use of pharmacologically adjusted extracellular vesicles can be used to safely deliver drugs such as HDACi directly to muscles affected by DMD, which will have a beneficial effect on them,” they say.
Source: https://www.sciencedaily.com/releases/2020/09/200915090134.htm
