Celecoxib shows potential benefit for Duchenne muscular dystrophy

Researchers have found that the FDA-approved anti-inflammatory drug Celecoxib (also known as Celebrex) had beneficial effects on the muscle function of mdx mice, a mouse model affected by muscular dystrophy. Celecoxib treatment of the mice also increased a protein called utrophin, which has been shown to improve dystrophic muscle and effectively compensate for the lack of dystrophin, a large protein that plays an important role in general muscle maintenance.

Duchenne muscular dystrophy (DMD) is the most common form of muscular dystrophy, affecting 1 in 3,500 male live births worldwide with genetic mutations that disrupt the formation of dystrophin. Children living with this fatal disease become wheelchair-bound by adolescence, with death typically occurring in their second or third decade due to respiratory and cardiac complications. Although many promising approaches are being developed to counteract the effects of DMD, no cure exists.

To conduct the newly published experiment, Dr Bernard Jasmin, Professor of Cellular and Molecular Medicine at the University of Ottawa, and colleagues treated a group of six-week-old mdx mice daily with either a low dose of Celecoxib or a control solution for four weeks. At the end of the treatment, the mice treated with Celecoxib showed a significant improvement in muscle strength, function, and integrity similar to that of healthy mice. The researchers also noticed an increase in utrophin protein levels in the mice treated with Celecoxib compared to the controls.

"Celecoxib treatment in this mdx mouse model increased utrophin expression levels in a variety of muscles, including the diaphragm, the heart, and the tibialis anterior in the lower leg — all while improving overall muscle function," Dr Jasmin explains. "This animal study points to the potential of Celecoxib to improve cardiac and respiratory function for DMD patients, as well as independent ambulation."

"While gene therapy and RNA interference-based drugs for DMD continue to be explored, the findings reported here have a tremendous clinical potential for addressing key syndromic features of the disease," said Thoru Pederson, PhD, Editor-in-Chief of The FASEB Journal.

The study has been published in The FASEB Journal.

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