What can we do NOW to get next trials for SMT C1100 (utrophin upgregulator) in the US in the future?

http://www.ox.ac.uk/media/news_stories/2011/110505_1.html

 

Considering that BioMarin has scrapped their similar program, and that Dr. Davies is at Oxford, I'm very concerned that the next phase of trials for SMT C1100 will happen in Europe or elsewhere outside the U.S.

 

In my opinion when this happens the FDA uses it as an excuse to take "wait and see" approach and for those of us with boys in their teens, that is of great concern.

 

My son in particular will likely not benefit from exon-skipping or other mutation-specific treatments. Utrophin-based treatments appear to be the most directly applicable to him.

 

What can I, as a parent, do NOW to help ensure that he can participate in the first human trials? We just don't want to wait and see any more.

 

-David

 

 

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So the original question remains - Is there anything a parent can do to encourage this progress specifically with respect to this compound?

Do we lobby the FDA? Do we fund specific organizations? Do we try and get our clinic to engage with Summit? Do we engage Summit directly?

While it is exciting to see such great variety of DMD trials, only certain trials hold promise for my son. The mouse research is so encouraging ... but find I it difficult to feel good about just writing a check to PPMD or MDA and wait. Can I do more to progress this specific treatment?

 

Hi David,

YES!!  You certainly can lobby now, but your local reps will get you further than attempting to contact the FDA.  Contact your representatives and make sure they know to keep an eye on Duchenne issues as they cross their desks.  You can, and should, attend the winter PPMD conference in DC.  They will train you on how to approach your reps and what to say.  After the winter conference you can continue to contact your reps throughout the year as important legislational issues crop up and PPMD is key at letting you know the best way to do this. 

 

You can also contact Pat Furlong directly and ask her what more can be done.  Pat is well entrenched with our federal government and maintains a good relationship with key people at the FDA.  Thank God she has already laid the foundation for what needs doing. 

 

And please, don't discount the power of writing a check.  If you speak to Pat about this she will confirm that any donation you make via PPMD can be earmarked toward a specific therapy that would benefit your son.  She runs a transparent financial sheet and will make sure your $ goes where YOU want.  For example:  Labs have costs not related to cures such as keeping the lights on, paying rent, ect...If you wish your donation to go toward the purchase of mdx mouse or another researcher, or anything specific she will direct your funds then babysit them to make sure they are spent where designated.  Not sure what other organizations offer this but for sure PPMD does.  You could donate to Summit if you wish.  But it's difficult for individuals to monitor and PPMD carries more clout.   

 

I know your frustration of waiting.  My son won't benefit from exon 51, which is coming first.  Unfortunately waiting is part of the horror of Duchenne.   It's a special kind of hell for parents.  Please know we are all in this together, no matter what mutation. 

best,

cheryl

 

 

PPMD will allow you direct donations to specific research?  I asked this a couple of years ago - and it was specifically for utrophin upregulation - and was told no.  So, I went to another organization...

 

I know from personal experience that directed contributions can become a nightmare to manage, so I understand why they're discouraged, but it is nice from the donor side.

 

I think one of the greatest challenges is trying to get a handle on what compounds have the greatest chance of success.  I simply lack the information and background to evaluate whether Summit/C1100, Tivorsan/Biglycan, or PTC/xxx has the edge in terms of science, path forward, and timeline for Utrophin up regulation.

 

I am waiting for Sharon Hesterlee and Pat to weigh in once they get the chance to process all the information.  Hopefully they can give the community an overall 'read' on the situation during the Advocacy meeting in July.   Once I get the jist for them I am planning to go 'ALL IN' on the one I sense is the most promising for the boys - always realizing there are no guarantees.   By 'ALL IN' in mean I am planning to liquidate my IRA's, 401k's, and any stocks and place my bet on a hopeful treatment for my son.   I am hoping since these Utrophin upregulators will help all the boys that there will be some other parents willing to go 'ALL IN' and help drive a Utrophin upgregulator to market.  

 

Perhaps I am too optimistic but it seems to me all the work done by Pat and her organization, the subset of active parents, industry, the CDC/NiH, and the FDA have the proverbial ball inside the 20 yard line.  I am hoping to help in a meaningful way to get more time on the clock for each of the boys (an interim treatment) and utlimately the ball in the endzone (an ultimate treatment).   I am further hoping active parents dig even deeper and the wait-and-see parents spark to life, get engaged, and help move the ball to the cure.

 

p.s

.

1) I just wanted to remind folks how easy it is to use the www.ParentProjectMD.org/Amazon link to get PPMD a check for approx. 5% of the sale amount.   It's just so, so easy.  I am hopeful parents, family, friends, and coworkers could just keep this in mind to help fund PPMD's work.  It works every day.  All year around.

 

2) Also, don't forget the United Way drives coming this Fall.   Get PPMD setup now in your local United Way or figure out how to help your local Unitd Way get funds to PPMD.   30 minutes on the phone now can generate thousands in the near future.

Keith,

The information I received about this issue came directly from Pat aprox 3 years ago.  Not sure who you spoke to about direct donations but perhaps if you e-mail Pat she can clairify.  Not sure if there has been a change or if maybe, they will handle direct donations above a certain amount.  Either way it should be easy to find out from Pat. 



Keith Van Houten said:

PPMD will allow you direct donations to specific research?  I asked this a couple of years ago - and it was specifically for utrophin upregulation - and was told no.  So, I went to another organization...

 

I know from personal experience that directed contributions can become a nightmare to manage, so I understand why they're discouraged, but it is nice from the donor side.

 

Bob - would like to private message you. Please accept my friend request.

Keith

Dear Friends,   I wanted to respond to the questions about SMT C1100.    We have a proposal from Jon Tinsley currently under review.   As you know, Biomarin terminated the study some time ago.   Since that time, considerable work has been done on the compound and it appears that the issue is one of formulation.   We have gotten several opinions from PharmD on re-formulation of the compound as well.   We are anxious to help move SMT C1100 forward.  Jon Tinsley has sent us a draft Q/A about SMT C1100 which will be posted in the next day or so.   Like you, we are anxious to see compounds that upregulate utrophin move into trial - in the US and Europe - and will do whatever we can to help.    

In addition, Project Catalyst has a compound that upregulates Utrophin moving forward and we expect to announce a clinical candidate before the end of this year.   With luck, the SMT C1100 compound and the catalyst compound may be synergistic.  

There was also a question about earmarking funds. We are happy to earmark funds for projects/programs we are considering funding.  Please let me know if you have questions.  Sincerely, Pat

Very Cool!

Thank you Pat. You'd be amazed at the relief a parent feels when an email comes into their inbox with "Pat Furlong has replied to your post..."

 

Do you expect regulatory hurdles for utrophin upgregulation therapies on par with other novel treatments like exon-skipping? How aggressive should parents be in "preparing" their FDA representatives for these therapies, assuming that PPMD and others can get the trials kick started?

Hi David, I can comment specifically on Summit’s proposed drug, SMT C1100. Because our drug is a classic small molecule chemical, there should be no unexpected hurdles with regulatory authorities.  The development of small molecule drugs is a well-trodden path for the FDA/EMA that has produced many marketed drugs over many decades.

Summit understands all the steps required to satisfy the regulatory authorities the product is safe and well tolerated.

Hope this helps, Jon

I am not sure I understand this question. Why are we talking about FDA approval when these drugs are not even in phase 1 trials? Not sure what path this drug or the next will take but we all can see clearly now how many years and how much money it takes to bring a drug from Phase 1 to 3, assuming that it is safe and effective.Even something like Idebenone for example took many years since Phase 1 started and the end date of Phase 3 is December 2013!

 

Shouldn't we concentrate on the path to bring this or other drugs to Phase 1 for now? I would say the first step is to secure funding or a partnership with a bigger pharmaceutical company. I would think it is a little more difficult for SMT C1100 since BioMarin already had a deal in place and they decided that it is not worth to continue development. I am still very confused about this. Why haven't they continued to work with this drug if the formulation was the problem? Why haven't they reformulated it and conducted another phase 1 study if this drug was promising?

David said:

Thank you Pat. You'd be amazed at the relief a parent feels when an email comes into their inbox with "Pat Furlong has replied to your post..."

 

Do you expect regulatory hurdles for utrophin upgregulation therapies on par with other novel treatments like exon-skipping? How aggressive should parents be in "preparing" their FDA representatives for these therapies, assuming that PPMD and others can get the trials kick started?

Hi Jon, can you comment on the chances of the clinical trial(s) to be held in US? What would be the determining factor?

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