CLINICAL TRIAL – No easy choices

CLINICAL TRIAL – No easy choices

In DMD

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On a Saturday morning at 9:00am I received a call from our Neurologist at UCLA. He called to tell me the news that there is a clinical trial at UCLA that Alexander could qualify for. After a 45 min conversation, I was left hopeful but at the same time anxious and a little sad. The good news is that the Pfizer is committed to a trial of a myostatin suppressing drug administered through an IV. Myostatin is a protein in our bodies that inhibits muscle differentiation and growth in the process known as myogenesis. It was discovered that animals lacking myostatin or animals treated with substances that block the activity of myostatin have significantly larger muscles. This has been studies in human cells at John Hopkins University for over a decade and they did identify one human being that they know of that has suppressed myostatin. This baby was born with extraordinarily large muscles and is very strong. Pfizer has taken on this treatment which could have enormous potential in millions of people. If suppressing myostatin increases strength and muscles, then it could help everyone from muscles diseases, to cancer patients to even delay aging. is ready to launch this into a human trial around the world in 105 Duchenne boys. Alex could be one of them. The downside is that it is a double blind placebo trial that will be for two years, which means for one year – he may not even be getting the drug. The drug or “antibody” is administered once a month for 2 years via a 2-hour IV infusion with extensive blood work the same day. Throughout the trial, there is extensive external and internal testing as well to be done on separate days, and of course, there is no guarantee that this drug will even have an effect. Hence, it is called a “trial.” It is a lot for a 9 year old to go through. But when I think of all the children that are going through chemo treatment for cancer at 3 and 4 years old, I feel grateful.

These are some of the hard decisions that we parents have to make. After much thought and conversation with different scientists, it looks like we will be going to have him screened in May to see if he qualifies, and then move forward. We either do this, or do nothing and wait for another drug to go into trial in a couple years. We don’t have the luxury of time to wait. Alex is already 9. Some boys walk in to their teens, others have stopped at 12.
I pray to God for guidance.

Keep everybody posted.

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