For Doug Penner, now 14, being part of the original drisapersen trial was a hopeful time; the drug enabled him to go on four-mile walks, climb stairs and even jump. For a 9-year old boy with Duchenne this was remarkable. For Doug’s parents this was a time of enormous hope.
Created by Prosena, drisapersen was then licensed to GlaxoSmithKline, reacquired by Prosena, who then ultimately sold drisapersen to BioMarin. This forced many boys like Doug to be taken off trials, only to be added and taken off again, causing setbacks in overall muscle strength.
BioMarin sought out FDA approval, but sadly, the FDA did not approve the drug and even sadder were the dashed hopes of boys like Doug and their families. Even though the drug showed valuable results for boys like Doug, it was no longer going to be available in the U.S.
Through the new nonprofit, CD Access, the drug was acquired by CureDuchenne and is now available to Duchenne patients in Canada who participated in a clinical trial for drisapersen through a Health Canada Special Access Programme. For a small number of Duchenne patients, this will be a huge step forward.
Doug’s father, Cam Penner, reported seeing declines in his endurance and strength within a few months of being off the drug. Six months without the drug and Doug was unable to climb stairs. Through CD Access, we are excited to share that Doug has been redosed. It’s not likely Doug will be able to return to the hikes he took as a 9-year old but he may regain endurance and strength.
When battling a rare disease with no cure, any treatment that can arrest the progress of the disease is worth fighting for. Every boy with Duchenne is worth fighting for, even if we help them one-by-one. We can’t think of a better goal of an advocacy organization than to actually deliver therapies to patients.
At CureDuchenne, we believe in taking action to ensure that all boys with Duchenne have a chance to live life to the fullest. We never thought we’d be the owners of a medication but we will do whatever it takes to help boys with Duchenne, no matter where they live.
Patients who participated in previous clinical trials for drisapersen in Canada can now receive drisapersen at Children’s Hospital of Western Ontario, Children’s Hospital of Eastern Ontario, Children’s & Women’s Health Centre of BC and potentially one additional site in Canada thanks to our efforts through CD Access.
The drisapersen journey has been full of ups and downs, from positive hopeful results for many patients to frustration and despair when the FDA declined to approve the drug. Through this we’ve learned so much about getting a treatment approved, and as a community we’ve shared in the triumphs for those boys whose lives were improved by this drug. In our mission to find a cure, we realize that detours like now owning drisapersen are important steps along the way. And, we feel proud that CD Access is positioned to help additional patients when an opportunity arises. When it comes to improving the lives of boys with Duchenne, we’ll do whatever it takes.