Partial Wind in My Sails

The wind in my sails may be coming back.

Two things have occurred that provide me with some hope that Ataluren may still be viable.

One was the conference call PPMD held with John Crowley. John Crowley is a dad with two children diagnosed with Pompe’s disease. His story was the inspiration for the movie “Extraordinary Measures” starring Harrison Ford, Brendan Frasier and Kerry Russell. John Crowley is also the CEO of a bio-tech firm. John made some very interesting points about drug development that hit home with me. Drug Trial planning is very difficult. If the drug does not meet their chosen end points, it does not always means that the drug is not viable, harmful or bad. It could be as simple as the dosage may not be correct.

The other thing that provided both my husband and I some hope was the Summary of Ataluren Phase 2b Clinical Trial Results presented at the American Academy of Neurology Meeting on April 16, 2010. These results indicated that the low dose of Ataluren was the dose that showed actual benefit in some of the trial participants.

Both of these things have caused me to sit back and think about some of the complexities of Duchenne. Hitting the expected targets the first time out in genetic disease trials are very difficult. This caused me to think that endpoints like the 6 minute walk test and other tests that are reliant on a multitude of factors may not be the best end points for Duchenne. For example: toe walking; vs flat feet; pelvis strength; thigh strength; spine strength; steroids or not. I’m not sure that you can use such an end point and still be able to calculate all the different variants, to come up with an average of how the kids are performing during this test and whether or not a benefit has been achieved. Then on top of these factors, let’s add that some kids may not like the taste of the drug. A parent’s goal while participating in these trials is to make sure that their child takes the drug. Therefore a parent may be mixing the drug with something that might cause the drug to become ineffective without realizing it. All of these factors make analysis extremely complicated.

It’s not surprising to me the analysis has taken a little time. I believe that PTC as a company has not given up on the drug and that they are still regrouping to figure out the next steps forward. I hope PTC can determine the next steps soon. We are hoping that the trail will be resumed using the low dose and possibly different targets for analysis.

Therefore the wind is slowly coming back. The waiting game is always difficult.