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Scientific Articles

Liposomal Steroid Nano-Drug is Superior to Steroids as-is in MDX Mouse Model of Duchenne Muscular Dystrophy

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source: Nanomedicine : Nanotechnology, Biology, and Medicine

year: 2019

authors: Turjeman K, Yanay N, Elbaz M, Bavli Y, Gross M, Rabie M, Barenholz Y, Nevo Y


Glucocorticosteroids are the most efficacious anti-inflammatory agents and the gold standard treatment in Duchenne muscular dystrophy (DMD). However, their chronic use may lead to severe side effects. We evaluated the use of a novel injectable steroidal nano-drug in mdx mouse model of DMD by comparing the efficacy of nano-liposomes remotely loaded with the steroid prodrug, methylprednisolone hemisuccinate (MPS) with the same steroid as-is, in short (4-weeks) and long-term (58-weeks) treatments.

Liposomal-MPS was selectively targeted to the mouse diaphragm, the most dystrophic muscle at early stage of the disease. The bioactivity of the steroidal nano-drug was evidenced by a significant decreased serum TGF-β and reduced diaphragm macrophage infiltration after short-term treatment. In the long-term, the treatment with liposomal-MPS not only demonstrated improved muscle strength and mobility it also induced lower tibia and lumbar vertebrae osteoporosis indicating much lower bone-related adverse effects.

organization: The Hebrew University-Hadassah Medical School, Israel; Tel-Aviv University, Israel

DOI: 10.1016/j.nano.2018.11.012

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