These are the words that best describe PPMD’s research plan.

  1. We aim to put better drugs into development for clinical trials because we can’t afford the failures.
  2. We apply funding strategically to increase the speed of testing new drugs.
  3. We prioritize and fund the testing in Duchenne of drugs that are already approved for other purposes.

Everything we do goes through this filter to make sure that we are achieving one of these aims and our actions and funding history demonstrate this commitment. 


Better: For example, in the interest of putting better drugs into clinical development, I recently took part in a meeting sponsored by the National Institutes of Neurological Disorders and Stroke to address the reasons why animal data was not translating efficiently into good drug candidates for various diseases. The group concluded that many studies are not conducted with the appropriate degree of rigor and that replication of studies is important. That report, of which I am one of the authors, includes recommendations for increasing the rigor of preclinical animal studies—it appears in today’s issue of Nature. As I write this blog we are also in the process of adapting PPMD’s grant applications to request that applicants address the issues highlighted in this report even more explicitly than they have done in the past.

In addition to increasing the rigor of our reviews to improve the quality of drug candidates, we’ve used a multi-pronged strategy that includes collaborating with the TREAT-NMD TACT expert committee for review, a strategy that we’ve been invited to present at the upcoming FasterCures partnering meeting. And we continue to fund the laboratory of PPMD Scientific Advisory Committee Chair Dr. Lee Sweeney to replicate findings published in the scientific literature by others. This strategy has proved increasingly important as more and more groups are reporting that the vast majority of studies published in the scientific literature cannot be replicated and should not be relied upon as the basis for human clinical trials (see Believe it or not: how much can we rely on published data on potential drug targets? and Drug development: Raise standards for preclinical cancer research).

Faster: We also want to see better drugs approved faster. To increase the efficiency of clinical trials we have provided travel awards to sites involved in both the Shire/Acceleron study and the Sarepta Eteplirsen study to help cover costs, such as childcare, that may not be allowed by institutional review boards. We also developed the DuchenneConnect patient registry, which sends out notifications to those eligible for clinical trials at the request of companies and academic investigators.  


Now: Finally, to take advantage of the potential of drugs that are already approved, we have vigorously investigated the possiblity of repurposing drugs approved for other uses by directly supporting clinical trials of approved drugs Cialis/Viagra and Eplerenone, based on animal studies funded by PPMD that showed these drugs were promising (see our Investigator grant to Stan Froehner, Ph.D. and Bridge grant to Jill Rafael-Fortney, Ph.D.), and by forming a working group with the purpose of reviewing all the data on Duchenne published to date to prioritize other approved drugs for testing in Duchenne.

We know that the fight to end Duchenne is a race against time. But we also understand that there is little to no room for error in this journey to treat people with Duchenne. That is why we will continue to instill this philosophy of Better, Faster, Now any time we consider supporting potential therapies.

Sharon Hesterlee, Ph.D.
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Comment by Sharon Hesterlee on October 15, 2012 at 12:24pm

Hi Neil--the participants from the Ataluren phase 2a and 2b study have all been rolled over into an "open access" study where they continue to receive the drug, but this isn't a phase III study.  The company is planning to announce something soon about next steps for this drug.  Also, keep in mind that it's really impossible to say whether or not the drug is working unless you have access to all of the drug data collected from all participants.

Am to speak with you more about this offline (

Comment by Neil McLaughlin on October 13, 2012 at 8:42am
Hi Sharon,
You mentioned that PTC are understandably concentrating on Ataluren. Do you know if the phase 3 trials are bringing positive results. information on this is so hard to come by. The phase 3 has been on going in America since 2010 and just started in europe. My sons consultant tells me it doesnt work. Any feed back or info would be very much appreciated.
Comment by Sharon Hesterlee on October 12, 2012 at 9:00am

Hi Keith--

We funded PTC to screen for new drugs actually, and yes, at least three active candidates have come out of that screen.  The company is understandably focused on Ataluren right now, but they have a utrophin upregulating drug that is in the final stages of preclinical evaluation for a lead-up to a clinical trial.   As for approved drugs, we have funded the RADD group, referenced above, to review all of the published literature and all of the gene expression data generated at Children's National Medical Center to try to identify the cellular pathways that are most significant to the course of the disease.  With the gene expression data the group at CNMC was able to apply software that overlays all the approved drugs that affect these pathways -- the data is still being analyzed (there's a lot of it!) but the goal is to prioritize some approved drugs for testing in Duchenne.  These could include cardiac drugs, anti-inflammatory drugs or anti-fibrotic drugs or maybe even something we haven't thought of yet that will fall out from this data review.  So we are still working on that, but testing things like tadalafil and eplerenone in the meantime.

Comment by Keith Van Houten on October 12, 2012 at 8:55am

Question on the "now" section.  Didn't PPMD fund PTC to look at a panel of FDA approved drugs for potential effectiveness with DMD?  Whatever happened with that?  I thought there were compounds identified that looked promising.  I know others are doing this type of work as well.  Cialis moved forward - what about others? 

Thanks,  Keith

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