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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Ok this is how I see what is happening right now with exon Skipping or what ever treatment that becomes our lifesaver.

I am from Lima, the capital of Peru, i´ve been into DMD for a little more than a year since my son Juan was diagnosed on november 2008, eversince I have been on the web searching and writing to everyone available, labs, scientists, Pat, Prosensa, GSK, Annemieke,...etc, etc.
Everyday I visit every single website (about 15) to search for new info., my son needs to skip exon 44 to get back in frame, I have been to CCH too (Brenda Wong...she and her team are amazing).

I have created a foundation in Peru to help out hundreds of families that know nothing about DMD and still they have been living with it for many years, trust me anyone in the states being able to be in touch with people like PPMD or MDA are very fortunate as you are informed and organized in many ways that I want to adopt for my country.

I tell you all this because you need to open your eyes and understand that even if FDA takes long to approve any treatment, the world is bigger and hopefully much simpler than the FDA so if we are having advances in Europe with any treatment, it is your duty to follow it as if it was done in the States.

WHERE EVER A TREATMENT TAKES OFF, I WILL BE THERE WITH ALL I CAN TAKE TO SUPPORT IT AND MAKE IT COME TRUE.

You need to calm down and wait for Emea to approve the exon skipping hopefully at the end of this year, the result will be the same, our sons will benefit from it in the USA or in Europe....as you can understand not much happens in Peru regarding research for DMD so as you I am waiting for EMEA or FDA and then travel to buy the treatment.

best energy from Lima!!!
JP.

cheryl cliff said:
Hi Jenn,

My son isn't far behind yours, his walking is almost over. I think that any medication for a life threatening condition can be administered under compassionate use in either Phase I, II or III Clinical trials. But the catch is BioPharms have to agree to allow compassionate use. If you can get Prosensa or AVI to agree to administer exon skipping to your sons then you don 't have to wait out the clinical trial phases.

The tough part is getting them to agree before their trials are completed because the FDA requires them to tell all, every result good and bad. If, during a clinical trial, they agree to let you or anyone else use their chemestry set, and something goes wrong, it could kill their entire trial process and end potential use for everyone. But, I really don't know how it works with the EMEA or other regulatory agencies. I think you have a better chance of obtaining compassionate use during a Phase II or certainly phase III, when risks are diminished. I understand your anger, being not far behind you. But I wouldn't worry so much right now about who is to blame (save for later?), but perhaps instead spend energy finding ways to obtain comapssionate use.

My son has to wait longer, needing to skip 44 & 45 which means utrophin will be here before they mix exon skipping compounds. I would congratulate any family that can escape the torture of DMD with or without compassionate use.



jenn said:
how long does a drug take to get from phase 3 to available? wont it just make sense to order pro051 when it hits the UK? we still have to order deflazacort outside of the US and US doctors have been prescribing it for years. i want to know who is responsible for delaying my son's treatment, he became a full time wheelchair user a few weeks ago... in october steve wilton said at a conference in London that he could treat ANY deletion in his lab RIGHT now!...WTF!

cheryl cliff said:
Thanks Sharon, I appreciate your helping us unravel the knots. And, hopefully this won't offend you but, frankly it doesn't matter to me if Prosensa or AVI had an FDA roadblock earlier or later in the game. Or if any biopharm decides to take their trials to a country outside the jurisdiction of the FDA because they are afraid of how the FDA "works". Especially in times like this where a new administration appoints new people and that takes more time.
The result is still the same, our children die while we wait. I wish it was different.



Sharon Hesterlee said:
I've just posted a blog on my page (at the request of someone) who wanted more information about the FDA approval process....I've tried to highlight issues that are particularly relevant to DMD. I think the key thing to understand is that it's not always the FDA's fault when things don't move forward. If Prosensa and AVI have not submitted their INDs yet, the FDA can't "hold them up." Sometimes companies know in advance from FDA communications that the FDA isn't happy or is concerned about some aspect of the data, but the study really can't take place until the IND is submitted. Because the review process is completely confidential, it's almost impossible from the outside to know why things are taking longer than anticipated.
One other thing to consider Jenn is that, from what AVI & Prosensa say, the cost of such a treatment will be very high. I think they mentioned something like $250,000 per patient/per year. Of course, nothing can be certain until it's approved but it looks like only a limited number of people will be able to purchase it from the UK/EU unfortunately. That is why we either have it aproved here or we need to move to EU for a period of time until it's approved here.


jenn said:
how long does a drug take to get from phase 3 to available? wont it just make sense to order pro051 when it hits the UK? we still have to order deflazacort outside of the US and US doctors have been prescribing it for years. i want to know who is responsible for delaying my son's treatment, he became a full time wheelchair user a few weeks ago... in october steve wilton said at a conference in London that he could treat ANY deletion in his lab RIGHT now!...WTF!

cheryl cliff said:
Thanks Sharon, I appreciate your helping us unravel the knots. And, hopefully this won't offend you but, frankly it doesn't matter to me if Prosensa or AVI had an FDA roadblock earlier or later in the game. Or if any biopharm decides to take their trials to a country outside the jurisdiction of the FDA because they are afraid of how the FDA "works". Especially in times like this where a new administration appoints new people and that takes more time.
The result is still the same, our children die while we wait. I wish it was different.



Sharon Hesterlee said:
I've just posted a blog on my page (at the request of someone) who wanted more information about the FDA approval process....I've tried to highlight issues that are particularly relevant to DMD. I think the key thing to understand is that it's not always the FDA's fault when things don't move forward. If Prosensa and AVI have not submitted their INDs yet, the FDA can't "hold them up." Sometimes companies know in advance from FDA communications that the FDA isn't happy or is concerned about some aspect of the data, but the study really can't take place until the IND is submitted. Because the review process is completely confidential, it's almost impossible from the outside to know why things are taking longer than anticipated.
Not offended at all--I'm not on the "side" of the FDA or the companies...the only side here belongs to the kids. I just like to try to understand where, under all this tangled mess, the real roadblocks lie so that we can focus our efforts most effectively. It's also helpful to understand the incentives of all the players--what motivates the scientists? what motivates the companies, the FDA, the NIH? It's often not the obvious answer but you have to use the right carrots along with the stick.

cheryl cliff said:
Thanks Sharon, I appreciate your helping us unravel the knots. And, hopefully this won't offend you but, frankly it doesn't matter to me if Prosensa or AVI had an FDA roadblock earlier or later in the game. Or if any biopharm decides to take their trials to a country outside the jurisdiction of the FDA because they are afraid of how the FDA "works". Especially in times like this where a new administration appoints new people and that takes more time.
The result is still the same, our children die while we wait. I wish it was different.



Sharon Hesterlee said:
I've just posted a blog on my page (at the request of someone) who wanted more information about the FDA approval process....I've tried to highlight issues that are particularly relevant to DMD. I think the key thing to understand is that it's not always the FDA's fault when things don't move forward. If Prosensa and AVI have not submitted their INDs yet, the FDA can't "hold them up." Sometimes companies know in advance from FDA communications that the FDA isn't happy or is concerned about some aspect of the data, but the study really can't take place until the IND is submitted. Because the review process is completely confidential, it's almost impossible from the outside to know why things are taking longer than anticipated.
would it not be worth looking into Biomarin- least this would work for all the boys


Sharon Hesterlee said:
Not offended at all--I'm not on the "side" of the FDA or the companies...the only side here belongs to the kids. I just like to try to understand where, under all this tangled mess, the real roadblocks lie so that we can focus our efforts most effectively. It's also helpful to understand the incentives of all the players--what motivates the scientists? what motivates the companies, the FDA, the NIH? It's often not the obvious answer but you have to use the right carrots along with the stick.

cheryl cliff said:
Thanks Sharon, I appreciate your helping us unravel the knots. And, hopefully this won't offend you but, frankly it doesn't matter to me if Prosensa or AVI had an FDA roadblock earlier or later in the game. Or if any biopharm decides to take their trials to a country outside the jurisdiction of the FDA because they are afraid of how the FDA "works". Especially in times like this where a new administration appoints new people and that takes more time.
The result is still the same, our children die while we wait. I wish it was different.



Sharon Hesterlee said:
I've just posted a blog on my page (at the request of someone) who wanted more information about the FDA approval process....I've tried to highlight issues that are particularly relevant to DMD. I think the key thing to understand is that it's not always the FDA's fault when things don't move forward. If Prosensa and AVI have not submitted their INDs yet, the FDA can't "hold them up." Sometimes companies know in advance from FDA communications that the FDA isn't happy or is concerned about some aspect of the data, but the study really can't take place until the IND is submitted. Because the review process is completely confidential, it's almost impossible from the outside to know why things are taking longer than anticipated.
Yes, Biomarin will help all our boys. Any info on Project catalyst of PPMD or TAT-Utrophin (which possibily will be more effective).
So, all of these issues and sub issues leave us with much to consider in the months ahead while the trials finalize in other countries. I never disagree with you Ofelia because you are extremely bright...but I do think the cost issue you rightfully brought up should be discussed a bit further. I really don't believe the proposed cost for treatments will actually turn out to be hundreds of thousands per tx, per patient. I refuse to allow that to get under my skin because without evidence or validation that proposal is nothing more than somebody's conceptual idea. Most likely a biopharm worst case scenario used to extract more funding.

My understanding, anyone please correct me if I am wrong, is that since Brittish medical care is socialized (residents don't have to pay out of pocket for services but they can if they wish to seek private treatments) and therefore "unavailable" to US citizens. If we move across the pond with the purpose of seeking treatments we must give up US citizenship and or create dual citizenship in order to accomplish the goal. Still, I haven't checked into details to confirm if new Brittish citizens are even able to receive socialized treatments (ie no cost).

Yet, I have discovered another way. You see I have a dear friend who maintains citizenship in UK. And, although this idea might seem far fetched I will do ANYTHING necessary to obtain treatments for my son just like you all will, and you all can (should?) consider this too. My friend offered to legally adopt my son so he could obtain treatments in the UK. I realize adopting out one's child puts parents at risk but I truely trust her intentions. And, upon considering risks one must consider the undeniable risk of death or permanent progression that comes from lack of timely treatment.

Yea, its a desperate move, but it beats death and what recourse has or will the FDA leave us?


Sharon Hesterlee said:
Not offended at all--I'm not on the "side" of the FDA or the companies...the only side here belongs to the kids. I just like to try to understand where, under all this tangled mess, the real roadblocks lie so that we can focus our efforts most effectively. It's also helpful to understand the incentives of all the players--what motivates the scientists? what motivates the companies, the FDA, the NIH? It's often not the obvious answer but you have to use the right carrots along with the stick.

cheryl cliff said:
Thanks Sharon, I appreciate your helping us unravel the knots. And, hopefully this won't offend you but, frankly it doesn't matter to me if Prosensa or AVI had an FDA roadblock earlier or later in the game. Or if any biopharm decides to take their trials to a country outside the jurisdiction of the FDA because they are afraid of how the FDA "works". Especially in times like this where a new administration appoints new people and that takes more time.
The result is still the same, our children die while we wait. I wish it was different.



Sharon Hesterlee said:
I've just posted a blog on my page (at the request of someone) who wanted more information about the FDA approval process....I've tried to highlight issues that are particularly relevant to DMD. I think the key thing to understand is that it's not always the FDA's fault when things don't move forward. If Prosensa and AVI have not submitted their INDs yet, the FDA can't "hold them up." Sometimes companies know in advance from FDA communications that the FDA isn't happy or is concerned about some aspect of the data, but the study really can't take place until the IND is submitted. Because the review process is completely confidential, it's almost impossible from the outside to know why things are taking longer than anticipated.
Totally agree! I think they just looked at the price of treatment for other genetic diseases (for example myozyme is $250,000/yr covered by most insurance plans (for kids, I think there were some issues with covering adults?)). There might be other ways to obtain funding to cover this kind of treatment in kids with fatal diseases too. Need to look into it.

Yes, adoption is one way. Dual citizenship also, but takes time. I wonder what happens if you have a job there? We should ask someone w/o dual citizenship who works in the UK. I am ready to start looking for a job in the EU if exon skipping gets close to approval there and Phase III Prosensa/GSK is still on hold here.

cheryl cliff said:
So, all of these issues and sub issues leave us with much to consider in the months ahead while the trials finalize in other countries. I never disagree with you Ofelia because you are extremely bright...but I do think the cost issue you rightfully brought up should be discussed a bit further. I really don't believe the proposed cost for treatments will actually turn out to be hundreds of thousands per tx, per patient. I refuse to allow that to get under my skin because without evidence or validation that proposal is nothing more than somebody's conceptual idea. Most likely a biopharm worst case scenario used to extract more funding.

My understanding, anyone please correct me if I am wrong, is that since Brittish medical care is socialized (residents don't have to pay out of pocket for services but they can if they wish to seek private treatments) and therefore "unavailable" to US citizens. If we move across the pond with the purpose of seeking treatments we must give up US citizenship and or create dual citizenship in order to accomplish the goal. Still, I haven't checked into details to confirm if new Brittish citizens are even able to receive socialized treatments (ie no cost).

Yet, I have discovered another way. You see I have a dear friend who maintains citizenship in UK. And, although this idea might seem far fetched I will do ANYTHING necessary to obtain treatments for my son just like you all will, and you all can (should?) consider this too. My friend offered to legally adopt my son so he could obtain treatments in the UK. I realize adopting out one's child puts parents at risk but I truely trust her intentions. And, upon considering risks one must consider the undeniable risk of death or permanent progression that comes from lack of timely treatment.

Yea, its a desperate move, but it beats death and what recourse has or will the FDA leave us?


Sharon Hesterlee said:
Not offended at all--I'm not on the "side" of the FDA or the companies...the only side here belongs to the kids. I just like to try to understand where, under all this tangled mess, the real roadblocks lie so that we can focus our efforts most effectively. It's also helpful to understand the incentives of all the players--what motivates the scientists? what motivates the companies, the FDA, the NIH? It's often not the obvious answer but you have to use the right carrots along with the stick.

cheryl cliff said:
Thanks Sharon, I appreciate your helping us unravel the knots. And, hopefully this won't offend you but, frankly it doesn't matter to me if Prosensa or AVI had an FDA roadblock earlier or later in the game. Or if any biopharm decides to take their trials to a country outside the jurisdiction of the FDA because they are afraid of how the FDA "works". Especially in times like this where a new administration appoints new people and that takes more time.
The result is still the same, our children die while we wait. I wish it was different.



Sharon Hesterlee said:
I've just posted a blog on my page (at the request of someone) who wanted more information about the FDA approval process....I've tried to highlight issues that are particularly relevant to DMD. I think the key thing to understand is that it's not always the FDA's fault when things don't move forward. If Prosensa and AVI have not submitted their INDs yet, the FDA can't "hold them up." Sometimes companies know in advance from FDA communications that the FDA isn't happy or is concerned about some aspect of the data, but the study really can't take place until the IND is submitted. Because the review process is completely confidential, it's almost impossible from the outside to know why things are taking longer than anticipated.
I have been following this thread, but it is still not clear to me when the Prosensa phase III (ambulatory) trial is scheduled. Is it known, or is it completely up in the air? Confused.
Rod

Ofelia Marin said:
Totally agree! I think they just looked at the price of treatment for other genetic diseases (for example myozyme is $250,000/yr covered by most insurance plans (for kids, I think there were some issues with covering adults?)). There might be other ways to obtain funding to cover this kind of treatment in kids with fatal diseases too. Need to look into it.

Yes, adoption is one way. Dual citizenship also, but takes time. I wonder what happens if you have a job there? We should ask someone w/o dual citizenship who works in the UK. I am ready to start looking for a job in the EU if exon skipping gets close to approval there and Phase III Prosensa/GSK is still on hold here.

cheryl cliff said:
So, all of these issues and sub issues leave us with much to consider in the months ahead while the trials finalize in other countries. I never disagree with you Ofelia because you are extremely bright...but I do think the cost issue you rightfully brought up should be discussed a bit further. I really don't believe the proposed cost for treatments will actually turn out to be hundreds of thousands per tx, per patient. I refuse to allow that to get under my skin because without evidence or validation that proposal is nothing more than somebody's conceptual idea. Most likely a biopharm worst case scenario used to extract more funding.

My understanding, anyone please correct me if I am wrong, is that since Brittish medical care is socialized (residents don't have to pay out of pocket for services but they can if they wish to seek private treatments) and therefore "unavailable" to US citizens. If we move across the pond with the purpose of seeking treatments we must give up US citizenship and or create dual citizenship in order to accomplish the goal. Still, I haven't checked into details to confirm if new Brittish citizens are even able to receive socialized treatments (ie no cost).

Yet, I have discovered another way. You see I have a dear friend who maintains citizenship in UK. And, although this idea might seem far fetched I will do ANYTHING necessary to obtain treatments for my son just like you all will, and you all can (should?) consider this too. My friend offered to legally adopt my son so he could obtain treatments in the UK. I realize adopting out one's child puts parents at risk but I truely trust her intentions. And, upon considering risks one must consider the undeniable risk of death or permanent progression that comes from lack of timely treatment.

Yea, its a desperate move, but it beats death and what recourse has or will the FDA leave us?


Sharon Hesterlee said:
Not offended at all--I'm not on the "side" of the FDA or the companies...the only side here belongs to the kids. I just like to try to understand where, under all this tangled mess, the real roadblocks lie so that we can focus our efforts most effectively. It's also helpful to understand the incentives of all the players--what motivates the scientists? what motivates the companies, the FDA, the NIH? It's often not the obvious answer but you have to use the right carrots along with the stick.

cheryl cliff said:
Thanks Sharon, I appreciate your helping us unravel the knots. And, hopefully this won't offend you but, frankly it doesn't matter to me if Prosensa or AVI had an FDA roadblock earlier or later in the game. Or if any biopharm decides to take their trials to a country outside the jurisdiction of the FDA because they are afraid of how the FDA "works". Especially in times like this where a new administration appoints new people and that takes more time.
The result is still the same, our children die while we wait. I wish it was different.



Sharon Hesterlee said:
I've just posted a blog on my page (at the request of someone) who wanted more information about the FDA approval process....I've tried to highlight issues that are particularly relevant to DMD. I think the key thing to understand is that it's not always the FDA's fault when things don't move forward. If Prosensa and AVI have not submitted their INDs yet, the FDA can't "hold them up." Sometimes companies know in advance from FDA communications that the FDA isn't happy or is concerned about some aspect of the data, but the study really can't take place until the IND is submitted. Because the review process is completely confidential, it's almost impossible from the outside to know why things are taking longer than anticipated.
We have no idea either!

Roderick Scott said:
I have been following this thread, but it is still not clear to me when the Prosensa phase III (ambulatory) trial is scheduled. Is it known, or is it completely up in the air? Confused.
Rod

Ofelia Marin said:
Totally agree! I think they just looked at the price of treatment for other genetic diseases (for example myozyme is $250,000/yr covered by most insurance plans (for kids, I think there were some issues with covering adults?)). There might be other ways to obtain funding to cover this kind of treatment in kids with fatal diseases too. Need to look into it.

Yes, adoption is one way. Dual citizenship also, but takes time. I wonder what happens if you have a job there? We should ask someone w/o dual citizenship who works in the UK. I am ready to start looking for a job in the EU if exon skipping gets close to approval there and Phase III Prosensa/GSK is still on hold here.

cheryl cliff said:
So, all of these issues and sub issues leave us with much to consider in the months ahead while the trials finalize in other countries. I never disagree with you Ofelia because you are extremely bright...but I do think the cost issue you rightfully brought up should be discussed a bit further. I really don't believe the proposed cost for treatments will actually turn out to be hundreds of thousands per tx, per patient. I refuse to allow that to get under my skin because without evidence or validation that proposal is nothing more than somebody's conceptual idea. Most likely a biopharm worst case scenario used to extract more funding.

My understanding, anyone please correct me if I am wrong, is that since Brittish medical care is socialized (residents don't have to pay out of pocket for services but they can if they wish to seek private treatments) and therefore "unavailable" to US citizens. If we move across the pond with the purpose of seeking treatments we must give up US citizenship and or create dual citizenship in order to accomplish the goal. Still, I haven't checked into details to confirm if new Brittish citizens are even able to receive socialized treatments (ie no cost).

Yet, I have discovered another way. You see I have a dear friend who maintains citizenship in UK. And, although this idea might seem far fetched I will do ANYTHING necessary to obtain treatments for my son just like you all will, and you all can (should?) consider this too. My friend offered to legally adopt my son so he could obtain treatments in the UK. I realize adopting out one's child puts parents at risk but I truely trust her intentions. And, upon considering risks one must consider the undeniable risk of death or permanent progression that comes from lack of timely treatment.

Yea, its a desperate move, but it beats death and what recourse has or will the FDA leave us?


Sharon Hesterlee said:
Not offended at all--I'm not on the "side" of the FDA or the companies...the only side here belongs to the kids. I just like to try to understand where, under all this tangled mess, the real roadblocks lie so that we can focus our efforts most effectively. It's also helpful to understand the incentives of all the players--what motivates the scientists? what motivates the companies, the FDA, the NIH? It's often not the obvious answer but you have to use the right carrots along with the stick.

cheryl cliff said:
Thanks Sharon, I appreciate your helping us unravel the knots. And, hopefully this won't offend you but, frankly it doesn't matter to me if Prosensa or AVI had an FDA roadblock earlier or later in the game. Or if any biopharm decides to take their trials to a country outside the jurisdiction of the FDA because they are afraid of how the FDA "works". Especially in times like this where a new administration appoints new people and that takes more time.
The result is still the same, our children die while we wait. I wish it was different.



Sharon Hesterlee said:
I've just posted a blog on my page (at the request of someone) who wanted more information about the FDA approval process....I've tried to highlight issues that are particularly relevant to DMD. I think the key thing to understand is that it's not always the FDA's fault when things don't move forward. If Prosensa and AVI have not submitted their INDs yet, the FDA can't "hold them up." Sometimes companies know in advance from FDA communications that the FDA isn't happy or is concerned about some aspect of the data, but the study really can't take place until the IND is submitted. Because the review process is completely confidential, it's almost impossible from the outside to know why things are taking longer than anticipated.
Rod--it's not known. I'm sure the company's own internal time line is probably tentative, but it will come after a short non-ambulatory study designed to measure how the oligos are distributed and broken down within the body. Pat is vetting some information with Prosensa now and will put it in her next blog.

Roderick Scott said:
I have been following this thread, but it is still not clear to me when the Prosensa phase III (ambulatory) trial is scheduled. Is it known, or is it completely up in the air? Confused.
Rod

Ofelia Marin said:
Totally agree! I think they just looked at the price of treatment for other genetic diseases (for example myozyme is $250,000/yr covered by most insurance plans (for kids, I think there were some issues with covering adults?)). There might be other ways to obtain funding to cover this kind of treatment in kids with fatal diseases too. Need to look into it.

Yes, adoption is one way. Dual citizenship also, but takes time. I wonder what happens if you have a job there? We should ask someone w/o dual citizenship who works in the UK. I am ready to start looking for a job in the EU if exon skipping gets close to approval there and Phase III Prosensa/GSK is still on hold here.

cheryl cliff said:
So, all of these issues and sub issues leave us with much to consider in the months ahead while the trials finalize in other countries. I never disagree with you Ofelia because you are extremely bright...but I do think the cost issue you rightfully brought up should be discussed a bit further. I really don't believe the proposed cost for treatments will actually turn out to be hundreds of thousands per tx, per patient. I refuse to allow that to get under my skin because without evidence or validation that proposal is nothing more than somebody's conceptual idea. Most likely a biopharm worst case scenario used to extract more funding.

My understanding, anyone please correct me if I am wrong, is that since Brittish medical care is socialized (residents don't have to pay out of pocket for services but they can if they wish to seek private treatments) and therefore "unavailable" to US citizens. If we move across the pond with the purpose of seeking treatments we must give up US citizenship and or create dual citizenship in order to accomplish the goal. Still, I haven't checked into details to confirm if new Brittish citizens are even able to receive socialized treatments (ie no cost).

Yet, I have discovered another way. You see I have a dear friend who maintains citizenship in UK. And, although this idea might seem far fetched I will do ANYTHING necessary to obtain treatments for my son just like you all will, and you all can (should?) consider this too. My friend offered to legally adopt my son so he could obtain treatments in the UK. I realize adopting out one's child puts parents at risk but I truely trust her intentions. And, upon considering risks one must consider the undeniable risk of death or permanent progression that comes from lack of timely treatment.

Yea, its a desperate move, but it beats death and what recourse has or will the FDA leave us?


Sharon Hesterlee said:
Not offended at all--I'm not on the "side" of the FDA or the companies...the only side here belongs to the kids. I just like to try to understand where, under all this tangled mess, the real roadblocks lie so that we can focus our efforts most effectively. It's also helpful to understand the incentives of all the players--what motivates the scientists? what motivates the companies, the FDA, the NIH? It's often not the obvious answer but you have to use the right carrots along with the stick.

cheryl cliff said:
Thanks Sharon, I appreciate your helping us unravel the knots. And, hopefully this won't offend you but, frankly it doesn't matter to me if Prosensa or AVI had an FDA roadblock earlier or later in the game. Or if any biopharm decides to take their trials to a country outside the jurisdiction of the FDA because they are afraid of how the FDA "works". Especially in times like this where a new administration appoints new people and that takes more time.
The result is still the same, our children die while we wait. I wish it was different.



Sharon Hesterlee said:
I've just posted a blog on my page (at the request of someone) who wanted more information about the FDA approval process....I've tried to highlight issues that are particularly relevant to DMD. I think the key thing to understand is that it's not always the FDA's fault when things don't move forward. If Prosensa and AVI have not submitted their INDs yet, the FDA can't "hold them up." Sometimes companies know in advance from FDA communications that the FDA isn't happy or is concerned about some aspect of the data, but the study really can't take place until the IND is submitted. Because the review process is completely confidential, it's almost impossible from the outside to know why things are taking longer than anticipated.
Sharon,

Will this short non-ambulatory study provide additional information/data that is not obtained in the systemic extention trial going on at the moment? I do not get it? Why would this provide more info than the trial going on now in ambulatory boys for a Phase III ambulatory trial?

This is what they have in their web page:

An extension study is ongoing in all 12 patients in order to collect longer term safety data before enrolling patients for a large international multi-center pivotal study in 2010. Prosensa is also preparing for a safety and PK study in non-ambulatory DMD boys. Prosensa has partnered with GSK for the further clinical development of PRO051/GSK2402968.

They do not say that they need to wait until completion of non-ambulatory trial, but will wait until completion of the extension ambulatory systemic trial.
All I can tell you is that the FDA is a different agency from the EMEA-they may be asking for slightly different measurements or maybe not. There is no straightforward mechanism for the FDA to accept data from a study conducted under the auspices of the EMEA. It is crazy...but there is not a lot of harmonization and there's no doubt that the lack of coordination slows progress and dramatically increases expenses. At the same time, keep in mind that thalidomide was approved in Europe but rejected in the U.S. saving thousands of U.S. families the devestation of having a baby born with deformities. Of course that was many years ago and both agencies are probably using higher safety standards now. It is a problem.

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