The last few weeks have brought a bevy of exciting stories from press releases and presentations. AVI BioPharma has been featured prominently due to results from two separate studies.
Study 1: Safety Data from Ongoing Systemic Trial of AVI-4658 “The open label dose-finding clinical trial is evaluating the systemic delivery of AVI-4658 once per week for 12-weeks by intravenous infusion. Although the study is primarily a safety trial, it includes measures of drug efficacy and pharmacokinetics…”
– Dose escalating safety study in DMD patients
– 12 week dosing periods showed no adverse side effects
– Data for clinical results will be collected 26 weeks from the first dose
– Final cohort to be dosed at 20 mg/kg
Study 2: Drug Prevents Muscle Wasting and Maintains Muscle Function in Severely Affected, Dystrophin Deficient Mice
– Study used mouse-specific PPMO
– Results hailed as “spectacular” (see video here)
The summary of these two studies is that the first generation exon skipping medicine from AVI BioPharma is well-tolerated in boys with DMD up to doses that may be required for prolonged chronic treatment. We are eagerly anticipating the efficacy data that is being collected in those patients. Further, the next generation treatment is working extremely well in what is considered the most challenging and most indicative mouse model of DMD. The double knockout mice exhibit systemic dystrophy and most closely mimic the human disease. Video results are posted on the AVI website, and they are exciting to say the least.
The final takeaway from all of this exciting news is that there is still a lot of work to be done. Funding is required to develop these medicines and move them through the regulatory process from IND to Phase I-III and eventual approval. Charley’s Fund is providing the vital funding that allows companies like AVI BioPharma and investigators at labs like that of Dr. Kay Davies and Dr. Steve Wilton. It is almost unbelievable how close these folks and others are to finding an effective therapy and even a cure for DMD. But they need our help to complete their work. Collectively we can make a difference for this generation of boys. Boys like Darius and Charley and all of the others. Please donate to Charley’s Fund or Darius Goes West. Purchase the Live Inspired app for the iPhone. Tell your friends and family about the exciting news and about how they can help as well.