Are there Additional benefits of Eteplirsen?
Posted by: Christine McSherry on May 17, 2013
Since the beginning of August 2012, my mornings have started the same, I literally live on my mac and my iphone…waiting. I wait for the good news that Sarepta will seek accelerated approval, for the hope that someday soon I will be able to give Jett eteplirsen, and many boys will benefit. Today Jett is home from school, too tired to make a full week. This means my workday is limited, but that is ok. I use this time to watch the videos, read the testimonials and witness the excitement around boys with Duchenne on eteplirsen who are either getting better or stabilizing. I often wonder how Jett would be if he were included in the trial when he was still ambulatory. I think about the changes our family has undergone because of Jett’s progression and how these families, the ones participating in the trial – how they are and will be delaying these changes in their lives. How encompassing Duchenne is on our financial and professional lives – how both of these issues, changes – can further affect quality of life. Our changes came like a train, a fast train that you could not stop.
When Jett was near the end of his ambulatory time, he was falling a lot. He wanted to walk, even when we insisted he use his scooter – he wanted to walk, and then he would fall. One night, he was standing in the shower, I was not more than an arms length away and he fell, hard. He fell so hard that he began to cry from the pain and out of frustration. That night, he told me he didn’t want to walk again, it just wasn’t worth it, there were too many painful falls. I don’t know who cried harder that night, Jett or myself.
The big power chair (3/4 covered by insurance) showed up within a couple weeks of that fall, then the changes came. We started by tearing apart the former garage and placing a ramp into the new entrance to accept the power chair. Then the back of the house, removing the stairs and changing the deck, tearing up the concrete around the pool area and ramping it for access (25K), purchasing a lift for the swimming pool (around 4K). Changing the grade of the side yard to put a ramp into the basement so he could hang out with the kids – in the “kids hangout” area (10K). Taking more space in the garage to add on a handicap bathroom (20K). Bringing in lifts, shower chair and a bed that was similar to a hospital bed (8K). There is no assistance to help pay for any of these modifications, families, like mine, are left to figure this out on their own.
Along with that power chair, we also needed the handicap van to get him and his new heavy chair around. No longer could we just throw the scooter into my SUV for trips – the power chair was over 300 lbs. Funny thing about handicap vehicles, in Massachusetts there is no assistance to purchase them and they cost about 60K new. They are converted vans – meaning that they were not originally built to handle all of the conversion equipment (the ramp, the electronics to open the ramp, the weight of the equipment and chair, etc.), they end up requiring a tremendous amount of abnormal costly maintenance not covered under warranty. (Now we have Clifford, a MV1 made by VPG - made as a handicap vehicle, much more reliable and Jett can ride up front).
And it doesn’t end there…want to take Jett on vacation? The steps and costs involved are enormous. A vacation might involve boarding an airplane if we choose not to drive. This means we need to transfer Jett from his power chair to an aisle chair to a seat on the aircraft. The power chair then needs to be loaded into the cargo portion of the plane. On several some of the early trips taken with the power chair, the crew did not store it properly and we would have to rent a manual chair while trying to locate someone to fix his power chair at the location, not easy. Transportation is an issue if we don’t bring our own, the cost of renting a handicap accessible van is about $200 per day, and because we have 5 children, we would still have to rent an additional car. Typical handicap beach chairs are not only uncomfortable, many resorts/beaches will not allow you to use them for the entire time, they ask you to transfer the handicap person to a normal stationary chair, then use it again to transport the person off the beach. We purchased a hippocampe (5K) to so Jett would be comfortable, be able to be pushed along the shore and so he could get in the water if he wanted.
Although the state of Massachusetts does provide some assistance with caring for Jett at home, it is not nearly enough. Someone, whether a parent or one of his siblings, need to be with him 24/7. He needs his head scratched, a glass of water, someone to get his computer, prepare his meals and help feed him, help to turn him every couple of hours at night, brush his teeth…Jett is completely dependent on others for everything. Priceless.
I am not trying to add up all the costs we have endured over the last several years for other to see – I wouldn’t change a single thing. Our family made sacrifices – as all families do with a family member who is ill, and we all do this without looking back - I am merely trying to point out the additional benefits of eteplirsen in the lives of these families that seem to be overlooked. They may have to face the same challenges that I have some day – but at least it’s not today, and given the positive trend that is emerging, it appears that these changes could be delayed for a significant amount of time.
For all of the reasons that we are witnessing through the data presented by Sarepta, for the reasons we haven’t identified yet but will through confirmatory trials and for the reasons listed above - quality of life, eteplirsen needs accelerated approval and it needs it NOW. Lives can be dramatically changed, not only physically for these boys as we are seeing through gains and stability, but it runs deeper than that; quality of life should not be overlooked, the financial burden presented to families can be staggering and the personal/professional toll this disease takes on parents and siblings can not be measured.
These are the additional messages that we need to get to the FDA. From my past meetings with Drs. Woodcock, Temple and Katz, who verbalized their compassion for the many changes families endure when caring for a child with Duchenne, I believe this additional color will further add to their confidence when considering eteplirsen for accelerated approval. I believe that the FDA will take all of these facts into consideration, that eteplirsen is also reasonably likely to predict a benefit, a better quality of life for patients and families affected by Duchenne.
I also believe that one of these mornings; soon, I will open my computer to good news – hope for Jett and many other boys. While we don’t know if Jett would ever improve on eteplirsen, I can only predict that his quality of life and ours, how he is living right now – just might be stabilized longer, and to me – that is the additional benefit of eteplirsen.
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