Ataluren data!
This is for the naysayers! Jill you were right! :)
http://ptct.client.shareholder.com/releasedetail.cfm?ReleaseID=518941
Replies to This Discussion
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Permalink Reply by Ofelia Marin on
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"These results suggest for the first time that a therapy which addresses the underlying cause of the disease can slow the loss of walking ability, the primary clinical symptom of dystrophinopathy," said Richard Finkel, M.D., Director of the Neuromuscular Program, Children's Hospital of Philadelphia. "The variability of symptom onset and disease progression in DBMD makes it challenging to assess the clinical benefit of investigational treatments. However, the ataluren data provide encouraging evidence of clinical benefit in patients whose prognosis is poor even with currently available palliative treatments."
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Permalink Reply by Felix Güthe on
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no news there!
Is there more treatment planned ?
Will it be released for use as orphan drug?
Maybe with more data the situation becomes clearer on if this is a useful drug or not?
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Good news!, and a confirmation that the decision to abruptly terminate all the trials may have been too rash after all. What I understand from the announcement by PTC is that: (a) "due to technical problems with the assay", it was not possible to determine a numerical correlation between the remarkably lower pace of deterioration in the ability to walk in the low-dose cohort (as compared with the placebo group) with the amount of distrophin present in the muscle, but (b) unles we believe in magic, the only reasonable explanation for the marked improvement in the walking ability of the low dose group is the presence of hitherto absent dystrophin; and (c) the shortfall between the measured results after 48 weeks of testing and the pre-determined primary endpoint has been narrowed down to a meager 30 centimeters!!! (less than 12 inches), which is the diffrerence between the now reported 29.7 meters achieved by the low dose group and the 30 meters required before the trials began.
This should send a very powerful message to restart the trials immediately, and to advance as fast as we can to have Ataluren approved as a drug for nonsense mutation DMD.
Please PPMD, raise your voice!!! Thank you Ofelia!!
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Permalink Reply by Blazena Kaderabkova on
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Thats great information!
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Permalink Reply by Dave Kramer on
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It's understandable, with the heartbreak associated to the discontinuation of the Ataluren trial, that there'd be a great deal of excitement with this news. I'm excited, it reignites some of the hope for an option to buy us more time, but I have to temper this with the immunological discoveries as of late that point to reactivity that may seriously hinder therapies which directly address dystrophin production as opposed to homologues or other related regulators (i.e. Utrophin or follistatin, etc).
http://www.medpagetoday.com/Neurology/GeneralNeurology/22599
Are any of you aware of further published layman's breakdown/interpretation of the "new" PTC results as of yet?
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Permalink Reply by Michael Piacentino on
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So I guess this is code for "Oops, we make boo-boo, drug good, get going again": Hey we already knew that in 2/2010.
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I also don't see much behind that note.
This would be the time to get going with rolling out the medication for a larger.number of patients.
So far it is still not clear what any additional test had shown although that result was promised fro July.
It looks we have not enough to get it through. Either:
It does not work.
Trial was badly conducted.
Trial was stopped too early.
I am loosing my hope for this one.
What do you think?
Michael Piacentino said:So I guess this is code for "Oops, we make boo-boo, drug good, get going again": Hey we already knew that in 2/2010.
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Permalink Reply by Tracey Hartz on
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I don't want to get my hopes up too terribly much, but I don't understand why this release would cause anyone to lose hope. It sounds like good news to me, that they are going to work with the FDA to get this drug to the patients. Which I know can be a frustrating process, but if Sharon Hesterlee seems optimistic about it: "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)." then I am going to try to be optimistic too.
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Permalink Reply by Cheri Gunvalson on
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Micheal,
Do you have any idea or guess when or if our sons who hwre in the trial will get back on the drug? Weh ave not heard anything from our trial site since the FDA told PTC they needed to do a single tratment IND rather than invidual INDs.
Cheri
Michael Piacentino said:So I guess this is code for "Oops, we make boo-boo, drug good, get going again": Hey we already knew that in 2/2010.
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OMG the reply has HTML all over it.
OK... Yes I am extreemly optomistic that Ataluren (aka PTC-124) will be available again for our kids. I know from the trial that our son did very well, though he's no all star, he was in the 2b extension study. So, there is this push for compassionate use via the FDA, and possibility for going to market next year for Cystic Fibroisis where it does work well. My son is 17 now so there is more "need/anger/hope" from me. The 'smart girls and boys' who constructed the trial should have known you don't make muscle, you (that would be impossible) you just slow the degredation. So yeah, really hopeful, angry, about the way things were done, but very, very hopeful.
Michael
Cheri Gunvalson said:Micheal,
Do you have any idea or guess when or if our sons who hwre in the trial will get back on the drug? Weh ave not heard anything from our trial site since the FDA told PTC they needed to do a single tratment IND rather than invidual INDs.
Cheri
Michael Piacentino said:So I guess this is code for "Oops, we make boo-boo, drug good, get going again": Hey we already knew that in 2/2010.
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Hi Mike,
I am not sure what you mean by HTML but what is your best guess for a time fram as to when our sons will get back on the drug as like you I believe it stabilized Jacob and he is losing ground off the drug.
Thanks, cheri
Michael Piacentino said:OMG the reply has HTML all over it.OK... Yes I am extreemly optomistic that Ataluren (aka PTC-124) will be available again for our kids. I know from the trial that our son did very well, though he's no all star, he was in the 2b extension study. So, there is this push for compassionate use via the FDA, and possibility for going to market next year for Cystic Fibroisis where it does work well. My son is 17 now so there is more "need/anger/hope" from me. The 'smart girls and boys' who constructed the trial should have known you don't make muscle, you (that would be impossible) you just slow the degredation. So yeah, really hopeful, angry, about the way things were done, but very, very hopeful.
Michael
Cheri Gunvalson said:Micheal,
Do you have any idea or guess when or if our sons who hwre in the trial will get back on the drug? Weh ave not heard anything from our trial site since the FDA told PTC they needed to do a single tratment IND rather than invidual INDs.
Cheri
Michael Piacentino said:So I guess this is code for "Oops, we make boo-boo, drug good, get going again": Hey we already knew that in 2/2010.
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Nope - I assume that's in the FDA's hands and the sites that are responding with IRB's - hopefully by Thanksgiving we should hear something.
Michael
Cheri Gunvalson said:Hi Mike,
I am not sure what you mean by HTML but what is your best guess for a time fram as to when our sons will get back on the drug as like you I believe it stabilized Jacob and he is losing ground off the drug.
Thanks, cheri
Michael Piacentino said:OMG the reply has HTML all over it.OK... Yes I am extreemly optomistic that Ataluren (aka PTC-124) will be available again for our kids. I know from the trial that our son did very well, though he's no all star, he was in the 2b extension study. So, there is this push for compassionate use via the FDA, and possibility for going to market next year for Cystic Fibroisis where it does work well. My son is 17 now so there is more "need/anger/hope" from me. The 'smart girls and boys' who constructed the trial should have known you don't make muscle, you (that would be impossible) you just slow the degredation. So yeah, really hopeful, angry, about the way things were done, but very, very hopeful.
Michael
Cheri Gunvalson said:Micheal,
Do you have any idea or guess when or if our sons who hwre in the trial will get back on the drug? Weh ave not heard anything from our trial site since the FDA told PTC they needed to do a single tratment IND rather than invidual INDs.
Cheri
Michael Piacentino said:So I guess this is code for "Oops, we make boo-boo, drug good, get going again": Hey we already knew that in 2/2010.
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