Update on my search for comfort and hope…

I told you I emailed the pharmaceutical co developing the drug Ataluren/PTC124, well they actually wrote me back! Here is their email!!

Dear Sarah and Derek,

Thank you for your interest in ataluren. I’m happy to answer your questions where I can but, as you suggested, I can’t answer some of them. We hope to find answers for your first three questions once we have the results of our current trial. As for the others:

Timing: the current trial is still recruiting. It will run for approximately one year after the last patient enrolled receives the last dose. Then it will take several months to analyze the data and submit it to the FDA for approval, which can take up to six months. So probably the earliest is late 2012 or early 2013.

Dosage: Dosing is by body weight so adults will take more drug than children but it is because they weigh more, not because they are older. In the current trial, the dosage is 10 milligrams of drug per kilogram (2.2 pounds) of body weight in the morning, 10 mg/kg at midday and 20 mg/kg at night. Ataluren is a mild vanilla-flavored powder that is mixed with milk or water.

Age: Right now it is only approved for use in a trial for children six years or older, so if the trial is successful and the drug is approved it will be only for that age group. If the trial is successful we will consider doing trials in younger children. Age six was specified because the trial’s primary endpoint – the measure we are using to determine if the drug shows a significant improvement when compared to placebo – is FEV1 as measured by spirometry, and children under age six cannot perform spirometry with reliability. A different endpoint would have to be chosen if we opened a trial for younger children.

Long term: theoretically the drug should remain effective but we will not have the answer to your question until the drug is approved and there is experience with long-term use.

Safety: ataluren has been studied in a variety of clinical trials in healthy volunteers and in patients.  It was given to 38 patients with nonsense mutation Duchenne muscular dystrophy and 77 patients with nonsense mutation CF who participated in the Phase 2a studies and was generally well tolerated.  Adverse events were infrequent and usually mild to moderate in severity.  A few patients with CF sometimes had mild burning with urination but this did not result in therapy being interrupted or discontinued.  There were no safety concerns based on physical examinations, vital sign measurements, electrocardiograms, or laboratory test results.  A Phase 2b pivotal study of 174 boys and young men with nonsense mutation Duchenne/Becker muscular dystrophy was completed late last year. Safety data from the study showed that ataluren was well tolerated and no participants discontinued treatment due to an adverse event.

If you would like to receive reports about ataluren in CF I invite you to join our email update list by following this link to the “contact us” section of our website and signing up: http://www.ptcbio.com/5.0_contact_us.aspx

I hope this information is helpful. Please feel free to contact us again should you have additional questions.

Best,

Diane

Diane M. Goetz | Director, Patient and Professional Advocacy | PTC Therapeutics

So I wrote her this:

Thank you so much for all the information Diane. It really means the world to me that you would write me back so quickly and with so much information. I just feel like the more I can find out the better I might feel. Our little girl , Ginger Blue is 16 months and my life. CF is such a terrible disease. We are so grateful for the work PCT Therapeutics is doing. This drug could possibly save my daughters life. Thank you ALL for what you’re doing. You’re our heroes.
Much Love, Sarah

and she wrote back!

Oh, Sarah – Ginger Blue is precious! I have shared this with my colleagues and a copy of the photo now decorates my office. Thank you so much for sending it and for your kind words. Seeing how our work might help makes us all want to work even harder.

Best,

Diane

This was good… Although I wish they would tell me, “Oh yeah, this will make her all better, forever.” I guess I’m not going to get that answer. I was really surprised and disappointed to hear she will not even be able to take it until she is 6. This was news to me. On the up side it is neat to know that Ginger’s face is in the building that a medication that could save her is being worked on. Makes me smile……

KeEp PrAyInG fOr A cUrE!!

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