Novel therapeutic approaches for inclusion body myositis.
OBJECTIVE
This review will highlight recent advances in developing strategies to accelerate muscle regeneration and to slow muscle degeneration in myositis, focusing primarily on inclusion body myositis (IBM).


RESULTS
Therapies for accelerating muscle regeneration, primarily through inhibition of myostatin, have shown promise in the laboratory and are now entering clinical trials. Recent studies have implicated autophagy, a key cellular process involved in clearance of ubiquitinated aggregates, in the pathogenesis of familial and sporadic inclusion body myositis (sIBM). IBM has joined a growing list of diseases known as TDP-43 proteinopathies, in which this protein becomes mislocalized to the cytoplasm; however, it is unclear whether these protein aggregates or others are pathogenic in this disease.


CONCLUSIONS
New discoveries of biomarkers in sIBM and new insights into the pathogenesis of familial IBM are opening novel therapeutic pathways for these disorders. In particular, drugs that stimulate autophagy, already in development for cancer and neurodegenerative diseases, are candidates for clinical trials. These disease-specific therapies combined with novel therapies to accelerate muscle regeneration hold promise for future therapy for this devastating disease.
Lloyd
Thomas E
TE
Department of Neurology, Johns Hopkins School of Medicine, 600 N. Wolfe St./Meyer 5-119, Baltimore, MD 21287, USA. tlloyd4@jhmi.edu
D015195:Drug Design; D006801:Humans; D018485:Muscle Fibers, Skeletal / Q000187:drug effects / Q000473:pathology*; D018979:Myositis, Inclusion Body / Q000175:diagnosis / Q000188:drug therapy / Q000628:therapy*; D012038:Regeneration / Q000187:drug effects / Q000502:physiology
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