or through continental - email malia reed at email@example.com (this is how you request a flight through continental's program called Careforce)
or private planes: maybe angel flight..if close enough or a group called Wings of Hope (google either one of these). I have flown all except Angel flight. Wings of hope is a small private plane. all of these will fly one patient and one parent...
Good. Just wanted to make sure that you know. Did she say when they expect the trial to start? Any other info about how exon skipping works so far? Does she think that this will be approved in 2-3 years?
I hope all is well.
Please take a look at the latest discussion about the exon skipping trial. Someone posted that Dr. Wong told her that they are a site for the Prosensa trial. I think you emailed her a couple of months ago to ask, but I remember she said no at the time (??). So you might want to ask Michelle about it.
About the comment under mine, in Annemieka's last paper or it might have been tonight, she talks about there being a few different variables between the studies and so I agree they could be very similar in outcome until all conditions are made identical.
Hi Ana, I'm sorry I didn't check with DMD community while the chat was ongoing. I didn't think to. When you say you couldn't connect, have any attempts to open the conference room URL worked for you?
What web browser are you running? Internet Explorer? I was unable to record the chat as well, I was booted a couple of times from the room and that lost the recordings I had initiated. Other than that we heard Annemieka well and saw her presentation and her face. I don't think the dozen others got booted out as much as I did. I am only learning and will make sure I check this next time. We had a practice chat last two nights leading up to tonight. Let me know if the room doesn't work when you try it in next couple of days and your OS and I'll ask the software people?
I do not know if it will be safety only. My impression is that it will not be safety ONLY since the AVI ceo says that they plan to use the dose levels found working in the UK trial.
People say that AVI's chemistry is "better" than Prosensa's but if you look at the comparison b/w % dystrophin in the intramuscular trials they seem to be performing the same. What's important is what % is obtained in the systemic trials. Prosensa does not say how much dystrophin they obtained and AVI will let us know end of year...then we can have a better idea if one is better than the other.
Thanks Ana: I did fin out some information.
I just got this email in from the Ohio folks where the AVI trial is slated to take place.
You contacted Lindsay Arnott who used to work in our institution, back in April and she had a chart open for your child. We have his name, date of birth and mutation and he is included in our list of potential candidates.
However, we are not ready to start the trial yet. The sponsor, AVI, will give his “go”, hopefully in a few months, and we will start enrolling patients. We will contact at this time to proceed to enrollment and screening. We would be very happy to have Liam in the trial and we will make sure to contact you back.
In the meantime, if you have questions about the trial, please feel free to email me.
Thank you very much.
Laurence Viollet, PhD
Clinical Research Coordinator
Team Project Leader
Center for Gene Therapy
The Research Institute at Nationwide Children's Hospital
700 Children's Drive
Columbus, OH 43205
Phone: (614) 355-2695
Fax: (614) 355-5247
Ana: Here is what I thought I posted to you...
Dr. Leshner did call at about 6:20 last night. Nothing earth shattering, but he was very, very hopeful with both waves that Prosensa and AVI are making with their drugs and exon 51 skipping. He feels certain that not only will this extend the Life of Liam and others who will benefit from exon 51 skipping for quite some time, but will also clear the path for all DMD children. Now, skipping more than one exon is still farther down the road, but he reiterated once again that in five years, things will be totally different. Last year Eric Hoffman told us to avoid Prosensa trails due to the fact that they were using an older drug, but they somehow changed the sugars in it, among other things, and are starting to deliver it subcutaneously at very high doses - higher than AVI at this time. Nothing has been shown to be toxic at this point and the higher the dose, the stronger the results. AVI is doing the IV delivery. Subcutaneous delivery gets to all parts of the body as well.
He also mentioned Dr. Mendell in Ohio, I think - who is calling for candidates who can possibly be helped by skipping exon 51. It will be a safety study. We already knew this, but this may come to fruition before the close on 2010. That study will not just be taking candidates from Ohio only.
Dr. Leshner added to be mindful of past trial exclusionary criteria including any sort of cognitive problems, behavioral issues, age, previous trial inclusion. This trial is for safety only and so far, both drugs are effective and safe.
In closing, Leshner said this is the most promising treatment he's ever realized.
Oh, on the funny side - at the Dining Away Duchenne fundraiser Tuesday evening, my hubby and a friend of mine were the high bidders for a boat ride along the Potomac River - captained by none other than Eric Hoffman himself. His folks passed away a few years ago and left him this sailboat. He has fixed it up and it is now sea worthy. WHat a great time to hit him up for information, eh??!!!!!
Dr Leshner just got back and said he will call us Wednesday with some very interesting updates. I will see him tonight along with Dr, Hoffman at the Dining Away Duchenne fundraiser in DC, but I don't plan to hit him up for the info tonight. He will call tomorrow at 6 pm so both Matt and I can hear what he's got to say. He's never asked to call us at home before, so this could be interesting. I will let you know what I hear tonight, but will give you the full Leshner update after we talk on Wednesday. I HOPE I have some good news to pass along to you. Hang in there and I will get back to you ASAP. How are things going otherwise?????
Have you heard any more details from the WMS meeting? I am still waiting for Debra or Pat's blog but it doesn't seem to come... I rememeber PAt wrote a blog before the meeting about how great the meeting is going to be etc.etc. but now, after the meeting, I do not see a blog from her. Please let me know if you have more details! My son also needs 51 skipped, he has a deletion of 50.
I do not know about the Prosensa trial sites. I would ask my neuro if I would be you. Since Columbus works with AVI I assume they are not one of the sites in the Prosensa trial...they will be very busy with both skipping 51 and skipping 50 AVI trials. I have an appt with Mendell tomorrow and I will ask what trials will start next year here in Columbus. The good news is that things are moving and hopefully our boy's futures will be different!
Nope, not much more. Dr. Hoffman says things will be speeding up now since they have cleared a few FDA hoops. CNMC just hired a doc in Hoffman's lab who is great in dealing with the FDA. If your son is strong, think hard about jumping onto a "safety" trial. The IV dose at first will still be low and may not prove positive results, but that is expected. If you son gets on an early trial, that may exclude him from the next phase. Has Ryan had a bx yet? He'll need one prior to getting on any trial.
Italy was grand and the summer is hot/humid - yuck!
How was yours?
It is a systemic trial (IV and/or subcutaneous). There will be a placebo groop so no guarantee of what boy gets a certain dose or placebo. This is only the first Phase of the trial, if it is safe and works there will be next Phases enrolling more boys to prove efficay. I think they said that after the trial the boys participating will get the drug (similar to the PTC 124 trial). I am not sure what happens with the extra prclinical work for which they got the grant announced 2 days ago. I really hope it will not delay the start...
Mendell's group hopes to have more info soon to send to everyone... I am not sure how soon that will be.