TUCSON, Ariz., Aug. 15, 2011 /WWW.61OK.COM/ — additional than a decade of specific Muscular Dystrophy Association-funded research, made possible like a result of generous public support of the MDA Labor day Telethon and thousands of grass-roots specific events, has today culminated in MDA providing financial assistance to the start of the earliest phase 2 placebo-controlled, multiple dose efficacy, safety, tolerability and pharmacokinetics clinical trial of an exon-51 skipping drug, eteplirsen, like a potential therapy for Duchenne muscular dystrophy (DMD).
The earliest three of twelve DMD boys participating during the AVI BioPharma clinical trial at Nationwide Children’s Hospital in Columbus, Ohio today received the earliest of 24 weekly doses of eteplirsen or a placebo by intravenous infusion (i.v.). 4 additional participants had muscle biopsies vital to measuring the presence of the essential muscle protein dystrophin both prior to and after treatments. The seven boys traveling in to the study launch are from Calif., Ill., Fla., Wis., Va. and Mo.
“This is definitely an imperative day for households fighting muscular dystrophy,” said R. Rodney Howell, M.D., Chairman of the MDA Board of Directors. “AVI BioPharma already finished a 19-patient clinical trial during the United Kingdom confirming the potential of eteplirsen to be a safe and efficient disease-modifying drug for DMD (The Lancet, July 25, 2011). Now, a group led by Dr. Jerry Mendell is receiving funding from MDA to aid initiate this randomized, double-blind, placebo-controlled 12-patient trial needed to further test safety, efficacy and optimal dosing.”
“Twenty-five years ago, MDA-funded investigators identified the dystrophin gene that, when mutated (or defective) brings about Duchenne muscular dystrophy as well as the somewhat milder Becker muscular dystrophy (BMD),” explained Mendell, Curran-Peters Chair of Pediatric study at Nationwide Children’s Hospital, and Professor of Pediatrics and Neurology at Ohio point out University. “Today, we’re underway with a clinical trial of the drug that ultimately could create a shortened but functional dystrophin protein for DMD boys with specific out-of-frame gene deletions that may be corrected by skipping exon 51.”
“By administering eteplirsen by i.v. for 24 weeks,” Mendell added, “our goal would be to find the very best dosage to trick the system into skipping over genetic disruptions present in some cases of DMD, to produce dystrophin ranges common of Becker muscular dystrophy. In Becker many patients are able to stroll into late adulthood and to possess normal or close to normal life spans.”
The introduction of exon skipping to restore the open reading frame utilizing splice-switching oligomers targeting dystrophin exons is 1 of several eye-catching therapeutic strategies for Duchenne muscular dystrophy being pioneered by MDA-funded investigators.
“MDA continues to be funding exon skipping study for Duchenne muscular dystrophy for additional than a decade,” noted Valerie Cwik, M.D., MDA Executive Vice President – study and medical Director. ”We’re very pleased to now be collaborating with AVI BioPharma on this trial and are hopeful that eteplirsen will grow to be an efficient therapy for people residing with Duchenne muscular dystrophy.”