Wind in My Sails

When I finished reading Pat Furlong's blog entitled Re-Visiting Katrina, I had to sit back and think for a little bit. How did I feel about last week’s news and events regarding Ataluren? Yes, I had to agree, I felt like I had gotten hit by a hurricane.

This past week was very stressful for my family.

First: we were meeting with the school district to discuss my son’s transportation issues. My husband has been dealing with the brunt of those issues. The bus driver and aid was requesting that my son crawl up the bus steps. My husband and I both lost it when my son came home one day from school saying that no one would help him onto the bus and they put a towel down across the steps and asked him to crawl into the bus. My son is 16 totally humiliated and crying by time I got home from work.

We did get that issue resolved after threatening to invoke an impartial hearing and my husband nearly getting arrested with a harassment charge. A county sheriff arrived at our home to speak with my husband, however no charges were pressed. The school district finally resolved the issue by contracting with another company to send a car/bus to transport our son to and from school, ensuring his safety and his dignity. The school district will not transport my son on a wheel chair bus because he doesn’t use a power wheelchair and the wheels on the manual chair can’t be locked in place. A scooter or a manual wheel chair with power assist wheels also can’t be locked in place on the bus.

Second: we received a call from the University of Utah telling us that the Ataluren drug trial was being suspended. My son was participating in that trial. He was one of the original 38 boys participating in the 2A and 2A extension trial. As trial participants my family hoped that Ataluren would buy five more minutes, more time being mobile, another birthday, and ultimately increase our son’s quality of life. Our son’s treatment has revolved around maintaining existing function for as long as possible and attempting to slow the progression. So the news that Ataluren did not meet its primary and secondary end points was more difficult to accept to my family then most in the Duchenne community. Gynzyme is a publically traded company and the SEC requirements called for PTC/Gynzyme to release the data and make a decision. The drug cannot be approved given the current data. Our reaction to this news was “What now?”

My husband and I have very different responses to things. He is very passionate in his response and tends to let you know what he is thinking right between the eyes. His first response is to seek retribution. I on the other hand, am more pragmatic about my approach by attempting to rationalize my response. As a result I have been accused of being the ice princess or the ice queen depending on the day. It's not that I don't care about my son’s transportation problem or the drug trial being stopped suddenly. I just view things as problems and obstacles to be overcome versus seeking retribution and/or invoking a law suit with the people who ultimately may be able to resolve the problem.

As a result of my service on the Board of Director’s for Parent Project Muscular Dystrophy, I have had the opportunity to get to know the people that make up the company known to the Duchenne community as PTC. This company has spent millions of dollars on Ataluren, and is not so big that they can just walk away and give up on this potential investment. PTC is still analyzing all the data obtained during the trial to figure out the next steps forward. I am very thankful PTC is willing to analyze all the data and not just say that Duchenne is too hard to demonstrate benefit and look at a different rare disease.

Yes, right now, the wind has been knocked from our sails; we are currently using a motor to navigate!

I prefer to wait for the following to occur:

· Wait for the data to be analyzed

· Hope that a new protocol will be developed with better primary and secondary end points.

· Remain optimistic that this drug trail will restart and ultimately be approved.

I believe seeking retribution should be used only when all other options have been explored and there is no other possible way forward. Let’s hope that optimism wins out and that retribution is the path of last resort.

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Comment by Christine Piacentino on March 12, 2010 at 6:14am
Thanks Donna - We appreciate the prayers.
Comment by Jacobs Mommom on March 11, 2010 at 9:50am
Hi Christine: I read your blog last night and I'm like your husband in the fact that I would have been at the school, plopping a towel on the floor and telling the principal to crawl on it and see how he felt! I used to be like you but the older I got, the more I saw that people with that I thought were pushy and had big mouths seemed to be getting what they wanted. Do I think it's right? Not really but when it comes to my grandchildren anything goes:) Jon was the first one I thought of when I heard the news. He's such a fighter and a huge hero in my book. Prayers are being sent to you all daily.
Comment by Christine Piacentino on March 11, 2010 at 5:27am
Thanks Cheryl. I hope that the wind comes back soon to fill the sails. I will remain forever hopeful!
Comment by cheryl cliff on March 10, 2010 at 10:43pm
Very sorry all this is happening to you and your family Christine :(. Reading about your son being forced to crawl up the bus steps really got me! I hate when those things happen to our guys. All our sons deserve better treatment than that. As far as being upset about the trial...who can blame you and the rest of the families? We are a safe harbor to each other- we DMD parents. Until the wind comes back again to fill the sails, rest assured anything said, typed or implied isn't a crime. It's not even difficult to understand.
best
cheryl
Comment by Christine Piacentino on March 10, 2010 at 7:14pm
Hi Susan, it has something to do with the law in New York State. We tried working around it very deligently to no avail.
Comment by Susan Rathfelder on March 10, 2010 at 7:11pm
I know this is not the major point of your post but my son was transported on a wheelchair equipped bus in both a manual and a manual/power assist chair. I don't understand what they mean by the wheels can't be locked in place. They used 4 tie downs and a seat/shoulder belt.

Susan

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