Almost 4 ½ years ago I received a call from a small biotech company in the Netherlands named Prosensa. They were working on exon skipping therapies for Duchenne and wanted to see if CureDuchenne would be interested in contributing to the funding of their research. Our answer was yes, and during the next year as we conducted extensive due diligence on the science and the company, we began raising the $1.3 million that we committed to the company.
Yesterday Prosensa announced commencement of the phase I/II trial for exon skipping at three centers in Europe. This trial will hopefully show safety first, and of course, we are all hoping to see some efficacy as well. Because exon skipping is mutation specific, only a small percentage of Duchenne boys where a skip of exon 51 will restore reading frame will benefit from this particular compound, but other compounds for other deletions are on the drawing board.
Although this is fantastic news we also must remember that this is new technology and still unproved…that is what the trial is for. We really want to thank Dr. Gert-Jan van Ommen from Leiden University and Dr. Judith van Deutekom at Prosensa, and Gerard Platenburg and his team at Prosensa for their dedication to DMD. There are other chemistries in development to induce exon skipping and we will be able to see the benefits of each in the near future.
As we all look to our own sons with hope that a therapy is near, let’s not forget the brave boys that are participating in this trial, they are true pioneers. My prayers are with them for their safety and well being.
On a lighter note, what do Anne Heche (Men in Trees), James Tupper (Men in Trees), Kevin Wiesman (Alias), Willie Garson (Sex & the City), Joel Murray (Still Standing and Bill Murray’s brother), Kevin and Bean (KROQ) and Ralph Garmon (actor and Host of the Kevin & Bean Morning Show) have in common? They are all joining us this Saturday, May 10th at the Petersen Automotive Museum in Los Angeles for “Dealing for Duchenne.” Katrina Carlson will entertain us, Wolfgang Puck will feed us and if you are in the area, please join us. Feel free to pass this on to your friends in LA. A big thanks to Prosum Technology, the titile sponsor for the huge amount of work they’ve done to make this night a success.
-Debra
