I just read in the on-line Quest Magazine for MDA, that Dr. Mendell's AVIPharma Exon Skipping trials are beginning in March of 2010. I signed up for Nicolas to be a part of the trials back in April and have every now and then emailed the coordinator to get updates. He did confirm that it would be taking place at that time, and right now they are getting their protocols together and should have more information in a couple weeks.

Here's my dilemma No. 1. We see Dr. Wong, and she too will be starting clinical trials for the Netherlands exon skipping drug trial (not the same as Dr. Mendell), but I have no timeframe from her of when her trials will begin. I was told last year that they would start summer of this year.... It's almost Christmas. I want something now and don't want to wait any longer.... first come first serve I guess.

My other dilemma is that my husband and I both work in Illinois and the trial is on-site in Columbus Ohio for 12 weeks, and then about 5 follow up visits, every couple weeks after that. This will of course cost me to lose my job, and in this economy, we absolutely cannot afford that. Of course my heart and thoughts are with my son and no matter what, if he is accepted into the trial, we will be there, job or no job. This is definitely more important, but I can't help but think about the future of not having a job... most likely having a home foreclose on us (we are definitely not wealthy), and living with relatives (really not close with alot of them), etc. etc. etc. Also, not having enough $$ to afford to buy Nicolas his deflazacort, and supplements, and not to mention still paying for this years visit to see Dr. Wong.
I guess I just wanted to throw my worries out there. Hopefully it's all for the best. Is anyone else up for the trials or know about Dr. Wong's???

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Who thinks exon skipping will NOT an effective treatment?

jenn said:
the response was not really clear, if you message me your email i will forward it to you if you'd like

Ofelia Marin said:
Does he give an explanation of why he thinks exon skipping will not become an effective treatment (if approved)? What does he think would be a better alternative?

jenn said:
i know dr brian at mass general in boston seemed to not like exon skipping at all when i asked him if that hospital may be a site for avi or prosensa... so confusing for sure!

Laurie Paschal said:
I'm confused, too. We were there in August and she didn't say a word to us.

jenn said:
im confused, we are also dr wongs patients, and also set to hopefully take part in the trial at nationwide childrens...but dr wong never told us about her possible trial, in fact i believe she mentioned we would have choices, prosensa or avi, in the next year but no further info. if anyone has info on where prosensa will be doing their trials id love to hear it.
This just in from Dr. Wong.... again too hard to wait. First come first serve.... Looks like if Nicolas is able to be a part of AVI, we wil be there...

"Per Prosensa, the PRO051 trials will be conducted by GSK - timeline - JULY, AUGUST OR September 2010"
I do believe exon skipping is an effective treatment as it stands.... for something to be able to transform duchenne into becker's is not a cure but a definitive treatment, and we'll be fine with that for now. Of course, and controversial, the one CURE i'm looking forward to is stem cells. I'm not on the controversial side (although a couple of my siblings debate this)... my thought is - have a sick child like we have, and then make your decision... I'm fine with stem cells, as most of us are, because it can actually be a cure for our boys... what wouldn't we do for them? I would do ANYTHING!! I'm willing to lose my job, move into family member's house, just so we can be a part of a TRIAL!! What wouldn't I do for a CURE!!

irishgirl said:
Who thinks exon skipping will NOT an effective treatment?

jenn said:
the response was not really clear, if you message me your email i will forward it to you if you'd like

Ofelia Marin said:
Does he give an explanation of why he thinks exon skipping will not become an effective treatment (if approved)? What does he think would be a better alternative?

jenn said:
i know dr brian at mass general in boston seemed to not like exon skipping at all when i asked him if that hospital may be a site for avi or prosensa... so confusing for sure!

Laurie Paschal said:
I'm confused, too. We were there in August and she didn't say a word to us.

jenn said:
im confused, we are also dr wongs patients, and also set to hopefully take part in the trial at nationwide childrens...but dr wong never told us about her possible trial, in fact i believe she mentioned we would have choices, prosensa or avi, in the next year but no further info. if anyone has info on where prosensa will be doing their trials id love to hear it.
So "early" 2010, as Prosensa stated couple of months ago, is now July, August or September 2010... Looks like the fact that GSK is planning the trials will postpone the start (?).

Wouldn't it be great if we could do the same with our boy's DMD progression, stop it for 6 months or so? For them 6 months delay might not seem like a big deal but for us it is.



Michelle Gonzales said:
This just in from Dr. Wong.... again too hard to wait. First come first serve.... Looks like if Nicolas is able to be a part of AVI, we wil be there...

"Per Prosensa, the PRO051 trials will be conducted by GSK - timeline - JULY, AUGUST OR September 2010"
I totally agree with you Ofelia!! I love Dr. Wong and her team, don't get me wrong, but I was told last year that trials could begin this summer. I found out earlier this year it was being put off another year. Now, only to find out it will maybe, probably start the last quarter of next year. I don't have time. My son has NO TIME! None of our boys do. I would love to be able to stop Nicolas' progression for a while until the trials that can actually help are here... but i've been waiting since Nicolas was 4 and he's now 7 - AND NO TRIALS OR BENEFITS OR CURES HAVE OCCURRED!! I'm tired of waiting for our scientific team to magically appear and give our sons what they need and deserve... a cure!! I'm sure there are many older DMD guys out there and parents of young men who have waited a life time. I don't understand how there has been basically NOTHING except for steroids in the past umpteen years!! I'm fed up!! Where is the stem cell research that Obama okayed????? Am I missing something, because I've heard nothing!!!

Thank you all for allowing me to vent... it's been a tough week and it's only Tuesday!

Ofelia Marin said:
So "early" 2010, as Prosensa stated couple of months ago, is now July, August or September 2010... Looks like the fact that GSK is planning the trials will postpone the start (?).

Wouldn't it be great if we could do the same with our boy's DMD progression, stop it for 6 months or so? For them 6 months delay might not seem like a big deal but for us it is.



Michelle Gonzales said:
This just in from Dr. Wong.... again too hard to wait. First come first serve.... Looks like if Nicolas is able to be a part of AVI, we wil be there...

"Per Prosensa, the PRO051 trials will be conducted by GSK - timeline - JULY, AUGUST OR September 2010"
And where is the fast track for this fatal disease with high unmet medical need (no disease modifying treatment etc.)? It's been 8 years already since Prosensa started working on exon skipping, now one year wait between the end of Phase II and beginning of Phase III, and that is not due to lack of funding.
Ofelia:
High five on that comment. I'll take stopping the disease for six months - no problems!!!!

Ofelia Marin said:
So "early" 2010, as Prosensa stated couple of months ago, is now July, August or September 2010... Looks like the fact that GSK is planning the trials will postpone the start (?).

Wouldn't it be great if we could do the same with our boy's DMD progression, stop it for 6 months or so? For them 6 months delay might not seem like a big deal but for us it is.



Michelle Gonzales said:
This just in from Dr. Wong.... again too hard to wait. First come first serve.... Looks like if Nicolas is able to be a part of AVI, we wil be there...

"Per Prosensa, the PRO051 trials will be conducted by GSK - timeline - JULY, AUGUST OR September 2010"
I spoke with Dr. Leshner last night who gave a verbal peek into the Prosensa trail. It is scheduled for this summer after results of Dr. Mendell's trials have concluded. Generally trials feed off of previous ones and the results they yield. There is a 99.9% chance that one site for the Prosensa will be Children's National Medical Center here in DC. There is also the possibility that since AVI and Prosensa use different drug compounds, those that participate in AVI's trial this March in Ohio may be excluded from the Prosensa trial. But since the Prosensa trial will be so large with many sites, they may need to look use those kids from Mendell's anyway. That is one major thing I would seriously clarify before agreeing to any trial.


irishgirl said:
Ofelia:
High five on that comment. I'll take stopping the disease for six months - no problems!!!!

Ofelia Marin said:
So "early" 2010, as Prosensa stated couple of months ago, is now July, August or September 2010... Looks like the fact that GSK is planning the trials will postpone the start (?).

Wouldn't it be great if we could do the same with our boy's DMD progression, stop it for 6 months or so? For them 6 months delay might not seem like a big deal but for us it is.



Michelle Gonzales said:
This just in from Dr. Wong.... again too hard to wait. First come first serve.... Looks like if Nicolas is able to be a part of AVI, we wil be there...

"Per Prosensa, the PRO051 trials will be conducted by GSK - timeline - JULY, AUGUST OR September 2010"
Just curious, I've heard 2 different things, are they still only testing exon 51 or going on at the same time starting others? I know they have 53 ready and it is one of top 4 to be done (according to reports). We need 53. Michelle
Hi - I just read your profile and Kevin looks great. I am so surprised that he actually takes all those supplements you offer. I think that is great even though there is no medical proof that those supplements work in the medical setting other than the CoQ10. I would offer them as well, but my son would never get them down. My son just turned 10 last month and carries deletions of 48, 49 and 50, so we are high on the exon 51 skipping trials. Our son was only diagnosed when he was 8 with no obvious symptoms. Today he runs, jumps, rides horses and swims. His doctor says he is a WAY outlier. He should not even be running at his age. The funny thing about MD is the vast array of differences in progression between children. I am uncertain of the exons they are trying to skip next other than 51 and 50. They began with 51 because the majority of DMD boys can be helped skipping 51 - it's 13%, I think. You sound as though you are completely on top of things. The more we stick together and work together - they better off all of our children will be. Keep in mind that a biopsy will be necessary to be included in a trial. Did you mention that you are unaware if your son had BMD or DMD? The exon skipping trials hopefully turn a DMD into a BMD - so if your son had BMD - he won't need a trial - just a cure - we all hope for a cure. That will happen for sure someday.
Hang in there and keep doing all you are doing - it's wonderful. I love to hear your gumption!!!!

Kelvinsmom - Michelle said:
Just curious, I've heard 2 different things, are they still only testing exon 51 or going on at the same time starting others? I know they have 53 ready and it is one of top 4 to be done (according to reports). We need 53. Michelle
What exactly do you mean "one of top 4 to be done?" -- My son also needs 53 and am interested in knowing the exact 4 exons GSK is contracting. Obviously 51, and I think 44 - that leaves two others....just wondering if you read anything that I may have missed that identified the 4...thanks

Kelvinsmom - Michelle said:
Just curious, I've heard 2 different things, are they still only testing exon 51 or going on at the same time starting others? I know they have 53 ready and it is one of top 4 to be done (according to reports). We need 53. Michelle
The press release only mentions 51 and 44. I don't know if the other two have been officially announced.

I would guess the other two will be 45 and 53, which would then give them coverage for the 4 most commonly needed exon skips. Just a guess on my part, though.

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