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.
Regards,
Giles
Stay the Course. 2010 will be a good year for Duchenne.
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.
Regards,
Giles
Stay the Course. 2010 will be a good year for Duchenne.
1 comment:
Thanks for this post, Pat. I had to step away from all the parent sites due to the reasons you mention. DMD is hard enough on us as it is. But once we get engulfed by Google alerts and/or what other parents say/think, DMD life becomes harder, aggravating, and 100x’s more stressful. October 2008 was when I set myself free from all that added stress. Seeing, listening, and speaking with all the doctors and researchers at the PPMD Conference 2008 in Philly made me realize that the people who can help our children are doing the very best they can…and they don’t need me getting in their way.
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