A few weeks ago, I received a call asking about ‘my favorite drug’ for clinical trial. My answer is pretty simple. There are a number of publications suggesting certain approved drugs may have benefit in Duchenne and they run the gamut from anti-inflammatory agents to well, I cannot think of something that starts with a Z, but you get the picture.

I don’t have a “favorite.” Rather, I think you have to be thoughtful and act; do a literature review, asking questions related to the significance of the publication and scientific method (not all mdx studies are the same) and determine whether there is sufficient and compelling data to suggest that xyz drug will make a significant difference. And it is not as simple as saying, ‘let’s try x’, because trials, first and foremost, are difficult for boys and families and require: frequent trips to the clinic, blood work, time off work/school, changes in the family routine, and are emotionally draining. Just ask one of the families in the PTC124 trial.

Developing a trial protocol takes time and requires IRB (institutional review board) approval and may require regulatory authorities as well. And, given the fact that we have some new chemical entities coming forward, targeting mRNA or substitution of Dystrophin (124, exon skipping, potentially the biomarin drug utrophin upregulation, Prothelia’s integrin upregulation compound, Fallon’s biglycan) we have to think critically about what goes into trial and when. You cannot start one trial and jump to another that looks more interesting or more hopeful. We are working with Treat NMD and currently organizing small groups of individuals to review current publications on approved drugs, prioritize what should go into trial, identify what needs additional work (large animals, etc), and develop a timeline to move. The next meeting is scheduled for June.

When you think about it, the idea of clinical trials seems quite simple. Figure out what you want to test, develop a protocol, and recruit for the trial. Sounds simple enough, but actually not so simple at all. Before taking one single step, you have to figure out what drug you are testing. If the drug is ‘approved’, that is, already on the market for another indication, you must go back to the sponsor (drug company/biotech) to understand their position and if they are willing or interested in testing the drug in this population. Off the top of your head, you might think that it is a no brainer, that they would definitely sign on because it extends their market. But it is not always the case. If the sponsor has a successful product and has concern about risk – What if the drug does not work? What if it causes some serious side effects? – the sponsor may just say NO out of fear that data from the trial might negatively impact their market. Of course, you can do the trial in an academic setting and say something (i.e., positive trend, works, does not work, causes serious side effects), but the insurance company does not have to cover it because it is not ‘approved’ for Duchenne.

And speaking of approved drugs – Thomas Meier (Santhera) is on the Executive Committee of Treat NMD. Thomas’ company is moving Idebenone forward for Duchenne. It is now approved in Canada. I spoke with Thomas about the need to have Deflazacort approved in the US. Two years ago, we did some work to understand the possibility and process involved in order to seek approval in the US. We developed a portfolio of information about the original sponsor (Aventis), indications for us, countries where it is currently approved, and patent status. Thomas suggested Santhera may be interested in pursuing approval and is willing to review the portfolio. It would be a great step forward to get Deflazacort approved in the US. We will keep you posted.

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