Hi--Just wanted to introduce myself.  I joined PPMD in January as the Research and Advocacy Director after 11 years at MDA running their translational research and drug development programs.  At PPMD I'll be further developing work with drug companies, reviewing and growing the main research program and participating in advocacy intiatives.  I will also try to jump in as much as possible to answer questions about research and track down information about new treatments/therapies that anyone hears about.  One of my first projects along this line is to get to the bottom of this stem cell report from Costa Rica.  If anyone posts something that needs a response from me and you don't hear from me, you can always send me a nudge at sharon@parentprojectmd.org.  I'm really excited about the chance to focus exclusively on Duchenne and am looking forward to hearing from all of you.

 

Best Wishes,

Sharon Hesterlee

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My biggest problem with the FDA is that they don't have a standard model for risk assumption.

Here's an example: Washington state is considering banning the use of BPAs because there is a large body of evidence suggesting all kinds of harm in humans and animals. BPAs are in tons of stuff, including the lining of canned goods that people, including children, consume every day.

So the FDA is perfectly willing to let the public assume risk on behalf of the plastics industry. Right now, we're all assuming risk of heart disfunction, endocrine disruption, reproductive disorder (including the mutation of egg and sperm cells that may very well contribute to diseases like Duchenne) because the FDA won't ban the use of BPA.

But we're not allowed to assume risk on our own behalf in an attempt to save our lives.

To me, it seems like their desire to protect us from harm only extends until it runs right into a well-funded lobby.

That's obviously different divisions of the FDA, but they do have overall leadership that should be setting consistency here.
I think when we lean on Congress there should be a list of names of children in the USA who have died in the past 5 years from Duchenne. There should also be a list of names of families currently seeing their children at the end of thier lives this year. Families on each list should provide permission to supply their address and phone, maybe a pic of their child so we can tell Congress/FDA it will be THEIR responsibility to contact these families and explain what went wrong, what will be corrected, exactically WHEN the corrections will take place.

Maybe this way the urgency of our situation will sink in!

Paul Cliff said:
Sharon,

I think your list is well focused, and in most instances, these are issues common to all genetic diseases. As I see it, one of the problems is that FDA personnel feel accountable for instances in which harmful drugs and devices are approved when they shouldn't have been or aren't removed from the market fast enough. Unfortunately, they don't feel accountable for instances in which they are too slow to approve life saving medications. With apologies to Ofelia, my father and all the other professional statisticians I have known, there are lies, damned lies and statistics, but I expect it would be easy to demonstrate that more boys have died from Duchenne, to say nothing of the other diseases out there for which there is no treatment, in the last ten years then died from Vioxx, phen-fen (sp?) and all the other inappropriate life style drug approvals combined.

It would seem too late this year, but perhaps next year we could build on the success of the MD Care Act which made the NIH accountable for translating basic research into usable drugs and standards of care for neuromuscular diseases. Lean on Congress to hold hearings to have the head of the FDA come down and identify which conditions have orphan drugs in the approval process, where they are in the approval process and how many people died of that condition since the last hearing. That would get old real fast for our friends at the FDA, and I'm sure that they would be a little less cautious and a little more generous with their funding and time.
Cheryl,

The only way to put some pressure on FDA or any agencie is by making them feel the urgency doing what you are saying or with media pressure.
JP.

cheryl cliff said:
I think when we lean on Congress there should be a list of names of children in the USA who have died in the past 5 years from Duchenne. There should also be a list of names of families currently seeing their children at the end of thier lives this year. Families on each list should provide permission to supply their address and phone, maybe a pic of their child so we can tell Congress/FDA it will be THEIR responsibility to contact these families and explain what went wrong, what will be corrected, exactically WHEN the corrections will take place.

Maybe this way the urgency of our situation will sink in!

Paul Cliff said:
Sharon,

I think your list is well focused, and in most instances, these are issues common to all genetic diseases. As I see it, one of the problems is that FDA personnel feel accountable for instances in which harmful drugs and devices are approved when they shouldn't have been or aren't removed from the market fast enough. Unfortunately, they don't feel accountable for instances in which they are too slow to approve life saving medications. With apologies to Ofelia, my father and all the other professional statisticians I have known, there are lies, damned lies and statistics, but I expect it would be easy to demonstrate that more boys have died from Duchenne, to say nothing of the other diseases out there for which there is no treatment, in the last ten years then died from Vioxx, phen-fen (sp?) and all the other inappropriate life style drug approvals combined.

It would seem too late this year, but perhaps next year we could build on the success of the MD Care Act which made the NIH accountable for translating basic research into usable drugs and standards of care for neuromuscular diseases. Lean on Congress to hold hearings to have the head of the FDA come down and identify which conditions have orphan drugs in the approval process, where they are in the approval process and how many people died of that condition since the last hearing. That would get old real fast for our friends at the FDA, and I'm sure that they would be a little less cautious and a little more generous with their funding and time.
Sharon,

I think that strategy sounds good. I will see you in DC on the 20th. I also think getting Congress to send the letter should be followed by networking among other orphan disease advocacy groups to hit those Representatives and Senators with the same simple message: People are dying while these bureaucrats have committee meetings. Unity of message, commitment of members and persistence can move mountains up there. It's not like we're wrong.
Will you be visting Acceleron Pharma.
Hi Jason--I'm setting up a call now for PPMD. When I was at MDA we were in active discussions with Acceleron and I've been to their headquarters twice already for site visits. Sharon
Thanks, Do you have any more info on there progress other than what's on there website.
Did they start Phase 2 clinical trial's on ACE-031 yet?

Sharon Hesterlee said:
Hi Jason--I'm setting up a call now for PPMD. When I was at MDA we were in active discussions with Acceleron and I've been to their headquarters twice already for site visits. Sharon
I am a bit confused. Could you please explain to me exactly how the 6 min walk test is conducted and in what phase's if any.
Hi Bob:

The six minute timed walk can be used in any phase of clinical testing, but will probably be required in phase III. It's a way to measure strength and endurance by looking at how far the guys can walk in six minutes.



bob koch said:
I am a bit confused. Could you please explain to me exactly how the 6 min walk test is conducted and in what phase's if any.
Could you be more specific as to how many walks/when; per patient occur and what physical therapy if any is conducted following treatment prior to last walk test?
Hi Sharon!

Many thanks for making yourself so available. Out of curiosity, is there an interpretation we can take back from the PTC/Ataluren news that's been making the rounds from the World Muscle Society Congress meeting?

With the positive suggested/observed outcomes, and the company assertions, this leads me to believe that continuation of trials may be in the near future - am I right? Slowing progression is certainly an exciting option to hope for short-term.
Hi Dave:

I think the main strategy is to try to convince the FDA that the "re-analysis" of the data is compelling enough to get the low dose drug approved now, with an additional study conducted after marketing approval. The problem is that this is a deviation from the original endpoints that were part of the development plan, but there is precedence for drugs being approved on different endpoints after re-analyzing the data. That's why it's been so quiet over at PTC for so long--they've been frantically compiling evidence for the new drug application based on the low dose data (about 14,000 pages worth).




Dave Kramer said:
Hi Sharon!

Many thanks for making yourself so available. Out of curiosity, is there an interpretation we can take back from the PTC/Ataluren news that's been making the rounds from the World Muscle Society Congress meeting?

With the positive suggested/observed outcomes, and the company assertions, this leads me to believe that continuation of trials may be in the near future - am I right? Slowing progression is certainly an exciting option to hope for short-term.

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