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Drug Can Restore Dystrophin in Muscular Dystrophy Patients

Last Updated: August 26, 2009.

A drug that targets a molecular defect in Duchenne muscular dystrophy is safe and leads to functional dystrophin production in select patients with the disease, according to a study published online Aug. 26 in The Lancet Neurology.

WEDNESDAY, Aug. 26 (HealthDay News) -- A drug that targets a molecular defect in Duchenne muscular dystrophy is safe and leads to functional dystrophin production in select patients with the disease, according to a study published online Aug. 26 in The Lancet Neurology.

Maria Kinali, M.D., from University College London, and colleagues injected the extensor digitorum brevis muscle of seven patients with Duchenne muscular dystrophy with saline on one side and with one of two doses of AVI-4658 (a splice-switching oligonucleotide leading to skipping of exon 51 and restoration of functional dystrophin production) on the other side.

Three to four weeks after injection of the higher AVI-4658 dose, the researchers found increased dystrophin production. The increase was highest in areas adjacent to the needle track. Compared with healthy control muscle, the mean intensity of dystrophin staining ranged from 22 to 32 percent in treated muscles. The intensity of dystrophin staining in the dystrophin-positive fibers was up to 42 percent of that of healthy muscle.

"Intramuscular AVI-4658 was safe and induced the expression of dystrophin locally within treated muscles," Kinali and colleagues conclude. "This proof-of-concept study has led to an ongoing systemic clinical trial of AVI-4658 in patients with Duchenne muscular dystrophy."

AVI Biopharma manufactured and supplied AVI-4658 for the study and provided preclinical testing, packaging, labeling, and the investigator brochure for the drug. They also supported the toxicity studies and participated in the design of the protocol, the execution and monitoring of the study, and discussions with the regulatory authorities. One co-author is a consultant for AVI Biopharma, and one is an employee of the company.

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