Summit
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Permalink Reply by Keith & Jill VE on
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There are a couple other threads on the PPMD site asking the same question, but not a lot of updates.
The lack of coverage and support for Summit is strange to me, considering that Summit's drug seems to be the most promising since it will help ALL the boys with Duchenne and isn't as specialized and as 'cutting edge' as the exon-skipping drugs. More specifically, since Summit's drug signals the body to make utrophin (a chemical already present in the body), this should be easier to get regulatory approval versus a treatment that effectively modifies the processing of genetic codes in the body. To my knowledge, this innovative technique has never been done before. Please don't misunderstand - this is AWESOME, but will take more time to get the bureaucrats to approve.
And to be clear on my bias - my son's specific mutation will need two skips to complete the chain, so I'm not holding my breath on this being an effective treatment and/or being approved in my lifetime.
However, I make no claims to truly understand the details, since I'm no scientist. Feel free to correct me... :)
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Permalink Reply by Nadja on
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Hi Keith,
I understand what you mean.but summit is my really One big hope!
My son has a inframe deletion 3-48,there is no exon skipping working!
And the thing with the utrophin sounds so simple!
It must work.fast!
My son will be Four in may!the time is running so fast since we know the terrible diagnose!!!
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Permalink Reply by Jason Darienzo on
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Summit just started phase 2 and it will be completed in early 2016
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Thank you all.Yes i know this!but any results from Phase 1b?
When all is good at what Time is the start from Phase 2?
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Oops Phase 1b started in December
Phase 1b Top-line data Q2 2014
Phase 2 trial expected to start Q3 2014
Results of Phase 2 2016
latest presentation:
http://www.summitplc.com/userfiles/file/201403_Company%20Presentati...
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Nadja, I understand that phase 1b (which is a safety and dose escalating trial) is almost complete and results expected in the next 6 - 8 weeks. They have not had any safety issues to date (nor did they in the phase 1 trial in healthy patients). Then have the phase 2 designed, funded and ready to go by the Fall on 52 boys, with results in early 2016. As Keith points out above, as utrophin is not 'foreign' to the body, it's in effect being switched on. IF it works, as it did in animal tests, they (and most) will be hoping for accelerated approval at this point (but might be a false dawn!).
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Good point Steven on accelerated approval. That's why people feel so strongly about the Whitehouse.gov Petition
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Jason, quite right. If it's safe and it works, it and any other cure/treatment should be made available to all ASAP.
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I dont understand why we ever need the whithouse gov Petition.for Otter diseases such HIV it is already normal.
Why duchenne not?!it is quiet clear how necessary it is.that is a racing against the time.
Ok,phase2 should be Start this year and the results 2016.
I thought Phase 2 would start next year and then my son is five and i thought perhaps he can be a Part of the trial!so we Must wait and hope that summit would get the breakthrough and in 2016 all boys can benefit
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