There is NO question that intrathecal drug is working wonders for Trey. He is learning, he is happy, and he is living. In my previous blog post, I noted that criteria for the upcoming Phase III intrathecal trial needs to be narrow in order to get this drug approved, which is the ultimate goal. To get the drug approved. I can see that the argument for broadening the criteria to save a few of my friends’ kids lives is not in the best interest of the greater good. Okay. However, there is another option, one that doesn’t risk approval, and one that saves ALL our kids lives. Compassionate access. With other drugs, compassionate access has been granted once the drug has been proven safe in the Phase I/II trial and the Phase III trial has begun. Well, Phase III is about to begin.
If you were told your child is dying, what risk would you take to save his or her life? When Trey qualified for the trial, Dr. Muenzer made me repeat back to him that there is a possibility Trey could die as a result of the trial. He asked me if I was willing to take that risk. I know he had to ask, but for real? Trey has a severe form of MPS II. Without treatment, he would be dead by 20. Without a doubt. With IT drug, there was a ‘chance’ of him dying. Definitive death or possible death? EASY choice.
When I answered Dr. Muenzer’s question two years ago now, we didn’t know what the drug would do for Trey, but we still took that chance, the chance for life. There are now all sorts of data growing about IT efficacy. Due to the broad criteria in our phase of the trial, not all boys have had the same results because they began IT drug at varying stages of disease, but I can tell you I’ve not heard one report of progression. All of the boys have either maintained the level of skill they had when they began drug, some have continued to learn and gain skills although not at the same rate to maintain IQ, and some have even maintained IQ or IQ has increased since beginning IT drug.
I have heard that compassionate access might not be granted for Phase III of the Intrathecal Elaprase trial. I need to ask why not? When this drug is safe, when my son is living and learning and other boys are waiting and declining, why would compassionate access not be granted? If everything goes smoothly, we’re looking at drug approval in three to five years. With an average lifespan of twelve to fifteen years, our boys don’t have time to wait until approval. This is not a group of impatient parents. This is a group of desperate parents whose sons will die waiting for drug approval. They need compassionate access NOW (in reality, most of them needed the drug years ago). What risks would you take to save your child’s life?
Pictured here are boys with MPS II who did not qualify for Phase I/II and may or will not qualify for Phase III of the trial. These boys have moms and dads, brothers and sisters, grandmas and grandpas. Look at these boys and imagine their moms and dads reading about Trey’s progress and gains while they watch their child lose their language, lose the ability to hold cutlery, lose the cognitive function to play and to want, while they watch their children lose the light in their eyes. It is killing them. All these families want is a chance to access a drug that is available to other boys and will save their son’s lives. Compassionate access is not in the realm of the impossible. It is a decision that rests in the hands of a group of people who get to play God to these boys. If this was your son, what would you do?
Please speak up. We need to hear your voice!
