Research Briefs: DMD

Brief items about myostatin inhibition, exon skipping and stem cell research in Duchenne muscular dystrophy

by Margaret Wahl on October 20, 2010 - 9:45am

Anti-myostatin drug trial shows good preliminary results

Acceleron Pharma announced Oct. 13, 2010, that its experimental drug ACE031, which interferes with the action of the muscle-growth-limiting protein myostatin, was generally well tolerated and increased average thigh muscle volume in some participants in a trial involving 60 healthy, postmenopausal women. Participants received the drug for four weeks and were then followed for 12 additional weeks. See Acceleron Presents Preliminary ACE-031 Results froma Phase 1 Multiple Ascending Dose Study in Healthy Volunteers.

ACE031 is now being studied in boys with Duchenne muscular dystrophy (DMD) (see Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy).

Prosensa-GSK exon-skipping drug passes safety review

The Dutch biotech company Prosensa announced Oct. 13, 2010, that it received 7.5 million pounds from the pharmaceutical company GlaxoSmithKline as a result of having achieved a successful safety review for PRO051/GSK2402968, an experimental compound that skips exon 51 of the dystrophin gene and is designed to treat patients with DMD who have mutations in or around exon 51. The safety data are from a phase 2a, open-label extension study of 12 patients with DMD who received weekly subcutaneous injections of the drug in Europe. See Prosensa Receives 7.5 Million-Pound Milestone Payment as Part of Its Program with GlaxoSmithKline in Duchenne Muscular Dystrophy.

In April, some potential safety concerns were expressed about this drug (see Progress in Exon Skipping for DMD). It's now being tested in DMD the United States (see First US Exon-Skipping Trial Opens).

Prosensa exon-skipping drug being tested

A trial recently opened in Europe to test PRO044, an experimental compound developed by Prosensa to skip exon 44 of the dystrophin gene, in some 12 boys with DMD who have certain mutations in or around exon 44. See Phase I/II Study of PRO044 in Duchenne Muscular Dystrophy.

On Oct. 18, 2010, GlaxoSmithKline announced its continued intentions to develop drugs to treat rare diseases. DMD is among the targeted disorders. See GSK Outlines Approach to Delivering Advances in the Treatment of Rare Diseases.

Connections between inflammation and stem cell activities
are probed in DMD mice

A new study, conducted in mice with a disease resembling DMD, has identified details of how biochemical signals associated with inflammation cause muscle stem cells to mature into muscle cells, and how blocking those signals causes them to proliferate and stay stemlike. Control over these processes presumably would be helpful for treatment development.

See TNF/p38-alpha/polycomb signaling to pax7 locus in satellite cells links inflammation to the epigenetic control of muscle regeneration.

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