300 ‘antisense’ compounds developed for possible use in DMD

An MDA-funded team in Australia develops some 300 “antisense” compounds that can coax muscle cells to skip over errors in the dystrophin gene and produce functional dystrophin protein molecules. Dystrophin is needed but missing in DMD. One such compound is already being tested in boys with the disease.

Researchers release molecular ‘brake’ on protein that could help treat DMD

MDA-supported researchers identify a molecule called ERF that keeps a potentially therapeutic protein, utrophin, confined to one small area of muscle fibers. Reducing ERF levels appears to release this “brake” on utrophin production, allowing it to be produced all over the fibers and opening up a possible new therapeutic pathway for DMD.

Toxic neighboring cells identified in ALS-affected nervous system

MDA-supported researchers find that nervous system cells called glia secrete an unknown toxic compound that kills neighboring motor neurons, the muscle-controlling nerve cells affected in amyotrophic lateral sclerosis. They say transplanting stem cells that become good glia into people with ALS might be beneficial.

Blocking inflammation pathway helps in DMD

MDA-backed researchers confirm that blocking inflammation has significant benefits in Duchenne muscular dystrophy. When they treated DMD-affected mice with an engineered molecule that blocks a specific part of the inflammatory pathway, the animals had more regeneration of muscle tissue and more effective breathing muscles than untreated mice did. The researchers believe these findings may help unravel some of the underlying mechanisms involved in DMD and improve understanding and use of anti-inflammatory drugs, such as prednisone.

Researchers identify new type of muscle stem cell

MDA-supported researchers in Italy announce they’ve identified a new type of muscle stem cell that they believe is highly promising for treatment of muscular dystrophies. These new stem cells, called “pericyte-derived,” are located around small blood vessels in muscle tissue. When injected into mice with Duchenne muscular dystrophy, they matured into muscle fibers and improved the animals’ ability to grip a rod and stay on a treadmill.

Two anti-scarring drugs show promise in mice with DMD

An MDA research grantee is among the scientists who announced that two drugs, losartan and pirfenidone, have shown promise in reducing scar formation (fibrosis) in mice affected by Duchenne muscular dystrophy. Scar formation resulting from excess deposits of connective tissue is a major factor in muscle damage in DMD and other muscle diseases.

Largest ever ALS drug search begins

MDA and the ALS Therapy Development Institute in Cambridge, Mass., launch the largest drug discovery project in amyotrophic lateral sclerosis in history. The three-year, $36 million endeavor will attempt to identify biochemical targets and find drugs that work on them.