Novel treatments

A therapy, technology for muscular dystrophy in the field of intracellular protein degradation inhibitors

Inactive Publication Date: 2014-05-28
MD PHARMA
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However, while its principle has been demonstrated in animal models, its success in clinical trials has yet to be proven

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[0144] Summary

[0145] Laminin-α2 chain-deficient congenital muscular dystrophy (also known as MDC1A) is a severe and disabling disease. Recent studies have shown that increased proteasome activity is a hallmark of this disease. The autophagy-lysosomal pathway is another major system involved in the degradation of proteins and organelles in muscle cells. However, whether the autophagy-lysosomal pathway is overactive in muscular dystrophies, including MDC1A, remains to be determined. Studies using a laminin-α2 chain-deficient dy3K / dy3K mouse model and MDC1A patient muscle cells showed that autophagy-related gene expression was upregulated in laminin-α2-chain-deficient muscles. Furthermore, we found that autophagy inhibition significantly improved the dystrophic dy3K / dy3K phenotype. Specifically, it was shown that systemic injection of 3-methyladenine (3-MA) ​​reduces muscle fibrosis, atrophy, apoptosis and increases muscle regeneration and weight. Importantly, longevity ...

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Abstract

The present invention provides an inhibitor of intracellular protein degradation for use in the treatment and prevention of muscular dystrophy in a mammal. In particular, the invention provides an autophagy inhibitor and / or an inhibitor of the ubiquitin-proteasome system (such as a proteasome inhibitor) for use in the treatment and prevention of muscular dystrophy (such as congenital muscular dystrophy [e.g. MDC1A) and Duchenne muscular dystrophy [DMD]). The invention further provides corresponding methods of treatment and prevention of muscular dystrophy.

Description

field of invention [0001] The present invention relates to medicine and method for treating and preventing muscular dystrophy. Specifically, the present invention provides inhibitors of intracellular protein degradation (such as autophagy inhibitors and / or proteasome inhibitors) for the treatment and prevention of muscular dystrophies, including but not limited to laminin-α2-deficient Congenital muscular dystrophy and Duchenne muscular dystrophy. technical background [0002] Muscular dystrophy (MD) refers to a group of inherited muscle diseases that weaken the muscles that move the body. MD is characterized by progressive skeletal muscle weakness, defects in muscle proteins, and death of muscle cells and tissues. Nine diseases are assigned to MD, which include Duchenne, Becker, limb-girdle, congenital, facioscapulohumeral, ankylosing, oculopharyngeal, distal, and Emery-Dreifuss, but there are more than a hundred in total This disease is similar to MD. [0003] Most type...

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Application Information

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Patent Type & Authority Applications(China)
IPC IPC(8): A61K31/00A61K31/52A61P21/00
CPCA61K38/44A61K45/06A61K31/56A61K31/52A61P21/00A61K2300/00
Inventor M.德比杰-贾尔特V.卡米格纳克
Owner MD PHARMA
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