Jack Fowler is a 6 year old boy that lives in my town. For those of you that have children that attend the same schools as my own, that shop at the same grocery stores as I do and that keep up with local news with my town paper as I do, you are likely familiar with him or his family. For those of you that aren't, please allow me to briefly introduce you to his story.
He is one of roughly only 500 children in the United States that suffers from Hunter Syndrome. You likely haven't heard of it because it's so rare. It's a lysosomal storage disorder he was diagnosed with at 16 months when his parents grew concerned that he didn't seem to be hitting developmental milestones when he should be and still didn't sleep through the night. I can't even begin to imagine what it must have been like to have your baby diagnosed with a chronic, progressive, and ultimately fatal disease. Treatment for this disease has been limited due to the fact that it is so rare, but Jack has had them all. He has had therapy, infusions and surgeries to slow down the progression of the disease. His case is severe. His parents are very familiar with the progression of it and have used every therapy available to them to keep their son alive and as healthy as they can.
The most promising breakthrough is coming from Shire Pharmaceuticals, who is based in Massachusetts and has corporate headquarters in Dublin, Ireland. They had an enzyme-replacement drug, Elaprase, approved by the FDA. In 2012, sales of that drug totaled $497.6 million dollars. They now have a new drug in clinical trials, which is a concentrated form of their approved drug, to be administered by intrathecal (into a membrane of the spinal cord or brain) injection. There have been very promising signs from the trial, and one participant's mother has said that this treatment has given her son a second lease on life. Even though Jack is not a participant in the trial, the FDA allows for expanded use outside of the trial in exceptional cases such as Jack's. His family has a physician at Lurie Children's Hospital that is willing to administer the drug and the hospital will allow the treatment. All Shire has to do is agree to provide the compound.
Except they won't.
Jamie and Jason, Jack's parents, amassed 32,000 signatures to present to Shire in support of them providing the infusion to the Fowler family. They have enlisted the advocacy of The Isaac Foundation
who has gone to battle tirelessly for this family in hopes that Shire would agree to provide for expanded use outside of the clinical trial. They finally got their face to face meeting with the CEO Flemming Ornskov and Phil Vickers, which lasted mere minutes instead of the hour it was supposed to. During this meeting, they told the family they are declining to sponsor Jack for expanded use, and that they "don't work with families".
One of the reasons Shire won't sponsor Jack is a lack of safety data. I find this ironic since in 2012, they made $429 million and $1.0298 billion dollars on Adderall XL and Vyvanse, respectively. These are amphetamine medications used to treat ADHD in adults and children as young as 6 years old, the same age as Jack. Both of these medications come with the ominous black box warning on them, which warns consumers that side effects up to and including sudden death can occur. I have scoured online to find solid statistics on sudden death related to these drugs but that has been difficult to say the least. To play out worst case scenario here, we should assume that the highest safety risk of any drug would be death. In Jack Fowler's case, that risk is already inextricably present in his every day life as a 6 year old with a rare, chronic, progressive, and fatal condition. Given the fact that their highest-grossing drug is an amphetamine with a black box warning, I find the "lack of safety data" excuse to be disingenuous at best.
Shame on them.
The only avenue the Fowler family has left at their disposal is public pressure, and they are asking everyone to help them in that regard. If you are so inclined, reach out to them on their personal website. You could reach out to The Isaac Foundation. Shire has been watching the response to this and has pinged their servers daily.
Shire's motto is "To Be as Brave as the People We Help". Its the first thing you see on their website. It's time they earn the right to have that as their motto. If you send only one message, make it to them. You could contact their media department, or if you really want to go big, you could contact Dr. Fleming Ornskov at Shire directly. Drop an email to firstname.lastname@example.org. Tell him that as a doctor, he took the oath to first do no harm. Tell him he has the power to do the right thing and sponsor Jack for expanded use. Tell him that you will continue to bring this injustice to light until he does the right thing. To right a wrong takes bravery, and is exactly what he needs to do to be worthy of Shire's credo.
Addendum: A petition to the White House was created by a local woman on January 30 but has been removed. Jack's original petition has been reopened. Please consider sharing it after signing here.