PPMD Provides Travel Grant for Clinical Trial

By now you have probably seen the latest news from our friends at AVI BioPharma, announcing it has initiated dosing in a Phase 2 study of eteplirsen, their lead exon-skipping therapeutic candidate for the treatment of Duchenne. Parent Project Muscular Dystrophy has been a long-time believer in the work being done at AVI and was happy to provide a $50,000 travel grant to help patients participating in this study. If you attended AVI's presentation at this year's Connect Conference in Baltimore or if you listened in on the webinar AVI hosted in early summer, you know that this company, led by CEO Chris Garabedian, is determined to help us end Duchenne.


Though Chris is relatively new to the company, he has been quick to embrace this community and made our sons' health a top priority: "We are excited to announce the initiation of our Phase 2 clinical trial of eteplirsen and appreciate the generous support that PPMD is providing toward patients’ travel costs. We look forward to continuing our evaluation of this promising therapeutic candidate in the longer Phase 2 clinical trial, which will prepare us to initiate a pivotal registration study. Our continued focus at AVI is to develop eteplirsen as quickly and efficiently as possible with the hope of achieving our goal of delivering a safe and effective treatment to patients."


There are many ways to support research to help projects maintain their momentum. If helping to shoulder the burden of travel expenses for patients participating in these trials keeps research moving forward as quickly as possible, we are happy to participate.


It is because of you that we are able to provide grants like this. Whether it is your involvement in our monthly giving program STIR, Coach To Cure MD, Run For Our Sons, hosting a fundraiser, or texting a $5 donation, you have given to this community and have made it possible for us to support critical research like this Phase 2 study.


Stay tuned for more announcements this fall regarding PPMD's commitment to funding the most promising research in the therapeutic pipeline. And as always, thank you for your commitment to this community and your commitment to helping end Duchenne for all of our sons.

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Comment by amit gupta on August 19, 2011 at 10:32am
Thanks, Pat and Sharon. Parents of non-exon-skipping boys have their hopes on ACE31 and Summit, and neither is coming up with any news.
Comment by Keith Van Houten on August 18, 2011 at 7:10pm

Sharon & Pat - thanks for your response.  I hope you don't take my question the wrong way.


I believe very strongly in what Sharon stated - ' every dollar spent is one we can't spend elsewhere '.  That's exactly why I asked the question.  I also believe that those that need help with travel expenses should get it - because we need a fully enrolled trial, and we need it fast.  


Your answers about IRB's and ethics make sense.  I don't see how paying reasonable travel expenses becomes "unethical" in anyone's book - but you're certainly a lot closer to it than I am - and I know that a lot of this stuff doesn't fall into the common sense category.


Comment by Sharon Hesterlee on August 18, 2011 at 1:58pm

Keith, your question about why PPMD is funding travel expenses for a for-profit company is perfectly valid.  We have never seen the point in funding something that could easily be funded through another mechanism, but that's exactly why travel expenses for clinical studies are a good funding target.  It turns out the the Institutional Review Boards (IRBs) at clinical sites often limit the total amount of reimbursement that a company can provide to families participating in studies and they can limit they types of things to be reimbursed--for example, they may allow hotel bills to be paid but not babysitting expenses for other children.  The IRBs have these restrictions because excessive financial benefits might be considered unethical inducements...stay with me here--I didn't make the rules.  Basically anything we can do that will help companies recruit on time actually reduces the overall cost of a study and makes it more likely that companies will get involved in DMD research and more likely that they can afford to develop drugs for DMD.  For a full explanation of the economic strategy see http://www.parentprojectmd.org/site/PageServer?pagename=Advance_ind...


Also, somone asked about the Summit utrophin-upregulating drug.  We have spent many hours and involved many experts in evaluating this drug to better understand the pros and cons of this molecule.  We are under a very tight confidentiality agreement with the company but I can say that we have given them enough funds to make a reformulated version of the drug.  We don't anticipate providing them with further funding, but we've worked actively with other DMD groups who may still choose to do this.


As always, every dollar we spend in one place represents a dollar we can't spend elsewhere so we do quite a lot of "diligence" around every funding opportunity (but also try to avoid the "paralysis by analysis" scenario).  We want all of our dollars (which are your dollars) to work very hard and productively.

Comment by Pat Furlong on August 18, 2011 at 1:57pm

Hello Amit,  PPMD is interested in and working hard to accelerate therapies for ALL individuals with Duchenne.  This has been our mission from day 1..  My comments were in response to questions from Ofelia and Keith, specifically related to the block grant for the Nationwide/AVI study. 

   Our research support includes Utrophin upregulation, Myostatin, PDE5's (clinical comparison study underway), research review and prioritization of approved drugs for clinical studies, replacement compounds for steroids, gene therapy (follistatin study), exon skipping, etc  (please refer to the website).   The comment 'specifically exon skipping' was used because the discussion centered around this specific instance (block grant to Nationwide Childrens for the clinical trial).   We support infrastructure projects such as Imaginig DMD (in an effort to avoid repeated biopsies), outcomes research, standardization of animal models and other such projects to accelerate the development of therapies   Sharon Hesterlee would be happy to answer your questions about research and programs we support and those is review.

Sincerely, Pat

Comment by amit gupta on August 18, 2011 at 1:23pm
Pat, could you elaborate on why PPMD is 'specifically' interested in accelerating exon-skipping development (benefitting only a subset of DMD population) but only 'generally' interested in others that mght benefit 100% of DMD boys? And as a followup question - is Summit's proposal still under consideration or a decision was made to not provide support to them? Thanks.

Comment by Pat Furlong on August 18, 2011 at 11:12am

Keith and Ofelia, hi

  At some point in the future, access to expensive therapies will be a global advocacy issue, but to date, no one is making money because there are NO approved therapies.   In some cases, industry (especially publically traded companies) is unable to provide travel expenses as it can be perceived as 'promoting'.    Industry has fairly stringent rules about what they are able to say and do and the specific corporate culture will vary from company to company  (keep in mind we provided block grants for the Acceleron/shire (ACE 031)  I am not privy to MDA's contract, but would guess it is very specific.     I also cannot speak for AVI's financial situation - but I am aware they are working to raise significant VC and foundation money in order to proceed and expand their DMD program.  Many of us have had intense discussions with Chris Garabedian and colleagues about rolling out trials skipping multiple exons, to include rare mutations, duplications, etc.   They understand our urgency and in addition, we have expressed the willingness to help. 

We are all interested in accelerating drug development for DMD (generally) and specifically with regard to exon skipping technology.  It is expensive and time consuming.  Seems to me, this will take a 'village' as there are many issues/barriers (financian and otherwise) to be addressed and overcome if we are to understand the potential impact of this approach and (cross fingers and pray) see approved therapies.  

Trials necessarily screen candidates and not everyone screened will meet inclusion criteria.   This is not the fault of Dr. Mendell (or anyone else in the future)  Candidates for clinical studies and trials have to meet the inclusion criteria.  Compromsing in any way increases the risk of failure of the study.  And studies in rare diseases are difficult and especially in conditions such as Duchenne were there is significant clinical variability across the population.


We have received several notes from families (both avi and earlier acceleron/shire study), saying that without the travel reimbursement, they would be unable to participate.  Every boy/young man with Duchenne deserves a chance.



Comment by Ofelia Marin on August 17, 2011 at 7:47pm
Good question Keith...Parents are also upset b/c they spent thousands for pre-screening travel and were not selected for the trial. Instead of asking why AVI did not pay for it they are upset on Dr. Mendell for asking them to come for pre-screening. The only explanation I heard is that AVI wants to save money for next phase and also investing in scaling up manufacturing.
Comment by Keith Van Houten on August 17, 2011 at 6:28pm

Pat, could you comment on why patient advocacy groups like PPMD and MDA have to put funds into travel expenses?  

AVI stands to make billions on this drug if approved.  Their research expenditures were almost $36 million last year.  They have $52 million in cash, according to their financial statements.  

I agree that financially supporting those participating in the trial is key to quick and full enrollment - but it's in AVI's interest to do so - why aren't they paying for it?  That would free up the $50k that PPMD is putting towards travel expenses, and the $110k that MDA is putting up, for other research.

Just wondering....


Comment by Ofelia Marin on August 17, 2011 at 2:28pm
The financial support not only speeds up enrollment but also takes some of the burden from the families sacrificing a lot to participate in testing these potential therapies. I think it's great.

Comment by Pat Furlong on August 17, 2011 at 2:11pm
my prayers are added to yours!

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