New Anti-Inflammatory Drug Helps Mice With MD

When MDA grantee Giulio Cossu at the Stem Cell Research Institute of the San Raffaele Scientific Institute of Milan (Italy) and colleagues tested the experimental drug HCT 1026 in mouse models of Duchenne muscular dystrophy (DMD) and type 2D limb-girdle MD (LGMD2D), they found it was more effective than a corticosteroid drug in the prednisone family, and that it didn’t have the side effects of corticosteroids.

HCT 1026 is derived from flurbiprofen (Ansaid), an anti-inflammatory drug approved by the U.S. Food and Drug Administration for the treatment of arthritis. Besides easing the pain of inflammation, HCT 1026 also releases nitric oxide, which has been shown to have beneficial effects on muscle repair and regeneration.

The researchers, who published their findings in the Jan. 2 issue of Proceedings of the National Academy of Sciences, tested the compound in mice with diseases resembling DMD and LGMD2D.

In both instances, HCT 1026 maintained muscle structure and function, and reduced inflammation. The mice gained strength and performed better on tests of movement. HCT 1026 was “significantly more potent” than the corticosteroid prednisolone, with no detectable side effects. (In humans, side effects of corticosteroids, such as weight gain, can be problematic.)

The investigators say that, when they combined the drug treatment with injections of mesoangioblast stem cells into an artery, they saw better integration of the stem cells into muscle, more improvement of muscle tissue structure and better treadmill performance than they saw with stem cells alone.

Mesoangioblasts were identified by Cossu’s team and have shown promise in treating dystrophin-deficient dogs.