2010, January

As I spend time on the community site and Facebook, I feel a little jittery. Expectations are high. Everyone is counting days and everyone seems to have a different calendar, a different opinion, and a different bit of information. Some have contacted a clinical center, asking how to get their son in a certain trial, wishing to be first in line. Others have gotten less than expert opinions on genetic testing and the potential ability to participate in an upcoming trial.

This week we have had a number of calls from parents, worried about what is happening with regard to antisense trials and always the ‘when’ question.

The words are familiar – clinical trials, exon skipping (51, 44), first quarter 2010, name dropping of potential sites, conversations with doctors or clinical coordinators followed by a cryptic report on the community site or Facebook. Fear, frustration, disappointment, anger, panic – all wrapped up into one. TIME seems to be slipping away while you are waiting… waiting. The Google alerts are rampant, but it seems there is silence on the exon skipping front, specifically the news you are waiting to hear. And when there is silence, we tend to fill in the blanks… ‘What if something is wrong’, ‘did the company make different decisions’, ‘problems with ???’. Worries increase, tears flow, and the world looks pretty dark. We never simply stop and think – they are working nonstop, need no distractions, and are on track. Once Duchenne enters our lives, we live waiting for the next shoe to drop.

As a community we really need to hold it together and try our best not to over think things.

Prosensa is moving forward. You are already aware of the GSK deal. This is amazing news, exactly what is needed and required for progress. Having GSK as a partner expands opportunities, may change the landscape a bit, but does not change the direction. They are still moving forward. Trials are in development. Discussions with FDA (EMEA) are ongoing. The commitment is solid.

Do not jump with every comment or every post. It is easy to say and very hard to do. Because you are not hearing from someone frequently does not mean the Prosensa/GSK folks are distracted. Be thankful. They are working nonstop to move these compounds forward. They are aware of this great need, very aware that our sons (and some daughters) have no time to wait. They ‘get’ this. This makes every step critical to ensure that the trials are successful and the therapies become approved and everyone who stands to benefit from the compound will have access.

I hear all of you saying – we are willing to take the risk. And I understand that statement, but down deep in your heart there is a follow-up comment – “as long as nothing happens to my son.”

I’m not saying to have patience. That word went out the window with the diagnosis of Duchenne. I’m saying, do not panic. Prosensa, AVI, BioMarin, PTC Therapeutics and many others are all committed to moving as fast as possible and silence should suggest Focus and Commitment.

Last evening Dr Giles Campion, Chief Medical Officer for Prosensa wrote:

Dear Pat

Just tell them that on the authority of the Chief Medical Officer for Prosensa and a member of the GSK Joint Project Team you have it that there is no question of delaying the trial until 2011, on the contrary, everyone on the team is working very hard to run the necessary clinical study as soon as possible.


Stay the Course. 2010 will be a good year for Duchenne.

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Comment by Ian Anthony Griffiths on January 16, 2010 at 4:22pm
Thank you very much Melissa. I don't doubt their working as hard as they can, but science does work on different timescales lol. I'm sure Wyatt will grow up to be everybit a fine unique young lad :)
Comment by Wyatt's Mommy, Melissa on January 16, 2010 at 12:44am
Thanks Pat! As always you are amazing. And I don't think I will ever be able to thank you enough for everything you have done for all these boys, but especially Wyatt. God Bless.
Comment by Wyatt's Mommy, Melissa on January 16, 2010 at 12:42am
Ian, you are so right. But you know what, even though they REALLY don't understand, I don't think they are working any less hard to find a good treatment. I'm still very new into this disease too, Wyatt is only 22 months old. He's still very energetic and isn't having medical issues right now. I pray every single day for every DMD boy or young man I've met. You're an inspiration to me, you're not giving up, I hope I can teach Wyatt to be that brave. God Bless You!!!

Comment by RAKTIM SINGH on January 15, 2010 at 12:18pm
We need to hold together and pray for the BEST. We need big drug companies here. That is the only way to leverage big money into this. And we need to keep our messages positive, loud and strong.
Comment by Ana Vaish on January 15, 2010 at 10:14am
Thank you Pat for your update on Prosensa. I also got to know that the trial is starting this year. Due to GSK taking over it might get delayed by a couple months, and might not start in the spring, But never did we hear of it getting postponed to 2011.

Take Care
Comment by Tulika on January 15, 2010 at 1:15am
When I get too excited to find a news link claiming cure to DMD via stem cells I get a calm advice to be careful with stem cells. When there is a delay in trails and I am blue there is support available to not loose hope.

As Pat rightly said as a comminity we need to hold it together. I am fighting, every single moment, as time ticks away.
Comment by jenn on January 14, 2010 at 8:43pm
austin stopped walking this month. we are absolutely paying attention to every bit of news,on facebook, or the ppmd community, because it's the best info available. we cant get anything from the companies themselves, whether its a possible site or what the inclusion criteria may be. surely they know something by now.we are in a hurry because we don't have time, it may even be too late for austin to get into a trial. he is waiting too, becoming more aware and more afraid. like ian i find it hard to believe that these folks "get it" because im pretty sure anybody who is not living with THIS disease cant possibly understand. we may be close , but close doesn't do it for me when my son is getting worse every day. i do appreciate you pat, all that you do for our boys, if anyone can get the answers it is you.
Comment by Ian Anthony Griffiths on January 14, 2010 at 8:28pm
I'm very into science, and know things take a raft of tests, trials, reformulations and costs. I appriciate that and have lived though twenty odd years of potential treatment hopes, I've learnt never to jump the gun on things like this. We do need to apply the brakes from time to time.

I know I haven't got long, I feel worse every month but they don't and won't 'get' it, do they live with it after hours, after work, when eating food and enjoying a quiet glass of wine, Do they stare at their own death? Where were they at 20-25, in med school training and partying the endless nights away. While many sons, brothers and uncles were and are struggling to eat, struggling to breathe being life supported by ventilators. When they live through that they'll get it. They certainly understand the urgency but unless they are in it (a family with DMD), they will never get it. If they get it, why are profits always mentioned in trial reports etc, I feel like we with DMD are cash cows.

Thats just my opinion.

Thanks Pat, another thoughtful post, we dont need to over rationalize or think. Take care.

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