Jamie Fowler is one of my closest friends in the world. I feel her in my heart and my soul. I love her. Her partner is Jason and they have two kids, Juliet and Jack. Jack is six years old and has MPS II (a rare and progressive disease) like Trey.
I don’t want to hurt Jamie and Jason in saying what I’m about to say. It’s the elephant in the room. They know it, we know it, everyone knows it, but no one wants to say it. Jack is dying. Jamie’s acknowledged this before, but it hurts so much to even say the words, that most of the time these words are silent. We can’t keep them silent any longer. Not if we are going to save Jack’s life.
Although Trey and Jack both have MPS II, Trey, unlike Jack, isn’t dying. Trey qualified for a clinical trial that puts the enzyme our boys are missing into his brain. Trey has been on this drug for over two years now and is not only no longer dying, he is learning and he is happy. Over three years ago, Jack was excluded from this trial due to hydrocephalus.
For over three years Jamie and Jason have been watching Jack lose his skills. If your child is not dying in front of your eyes, you might not have a clue how excruciating it is, but Jamie’s blog and a new blog written by Andrew McFadyen (which includes a letter Jamie wrote to a mutual friend Melissa and I), will give you a feel.
One of my biggest struggles over the past two and a half years has been how to live as a mom to a child who, as Jamie put it, ‘has a chance at life,’ when Jamie goes to sleep wondering if she’ll hear Jack say mama or smile at her again tomorrow. It makes me sick.
There is an experimental drug out there that has been proven safe (Phase I/II is complete). Although it has not been proven effective yet (we are waiting for Phase II/III to begin), anyone who is involved with the Phase I/II trial and knows and sees our kids, knows it works. Although not all our kids are gaining skills at the same rate, NONE of them are dying. ALL of their progression has been stopped, AT MINIMUM, where it was at when they began receiving the drug.
Looking in from an office at the FDA or the company who makes the drug, Shire HGT, even the doctors in charge of the trial, I understand their hesitation in giving Jack drug. The doctors and drug company (as do I) want to prove the drug is effective so it will be approved by the FDA. Jack may not provide compelling evidence to approve the drug. The FDA doesn’t want to harm anyone.
What the FDA doesn’t realize, is that by not giving drug, they are killing kids.
Jamie and Jason have been fighting since the trial began to get Jack enzyme into his brain. Even when Jack was excluded, they didn’t give up. They have fought criteria, doctors and drug companies every step of the way. Now, however, with Jack about to lose everything, they have enlisted help. The Isaac Foundation has taken over advocacy for Jack to receive compassionate drug.
What it comes down to for me is: There is a kid whose life we could save. Yes, it will involve work, but there is a kid’s life we could save.
So as not to effect approval, Jack’s data could be excluded from the trial data. There is a trial site set up where Jack could receive drug. Jamie and Jason know there is a possibility Jack could die receiving this drug (Jack will certainly die without the drug) and have no problem signing a consent. Funding is not an issue.
There is a kid’s life we could save. Isn’t the rest just details to be worked out?
Jamie and Jason approached The Isaac Foundation because The Isaac Foundation has a track record. They have fought and won every battle they have waged in order to access expensive drugs for kids with rare diseases. These wars have been waged against governments and drug companies.
Jack, we are not giving up. We are currently waiting to hear back from Shire as to whether or not they will grant Jack access to IT Idursulfase. Hopefully they will say yes. If they say no, we are ready and armed. Jack’s life is not something we are willing to lose.
We may need your help. If we do, I will post again. Thank you for checking in. As we have learned, one voice becomes one hundred, becomes one thousand and eventually, the powers that be have no choice but to listen.
