A Mundelein couple feels stymied in their efforts to get what they believe could be life-saving medicine for their sick toddler son.
Jack Fowler was diagnosed with Hunter syndrome when he was 15 months old. The disease takes away his body's ability to get rid of cellular waste, and as the waste builds up, it causes mental and physical damage.
It's been a year since Jack has been able to tell his parents, Jason and Jamie, that he loves them, but as his motor and language skills fade, the possibility of death is a real possibility.
"To watch him forget who people are and not get as excited about people has been quite challenging," Jamie Fowler said.
A new drug, still in the experimental phase, has already shown promise in children enrolled in a clinical trial. Jack was not eligible for the trial because he has a excess fluid on his brain.
A world-renowned physician at Lurie Children's Hospital agreed to deliver the drug to Jack outside of the trial, but Shire Pharmaceutical denied that option during a brief meeting with the family on Saturday, citing a lack of safety data.
"I screamed and I broke down," Jamie Fowler said of her reaction to the decision. "By not sponsoring him, they're delivering him certain death."
The couple has launched an online campaign, hoping public pressure can help save Jack's life.
A "compassionate exception" from Shire would allow Jack to have access to the drug, and then the FDA would make the final decision on its safety.
"This is not a fight we're going to give up, because we have no other option," Jason Fowler said.
Shire Pharmaceutical issued the following statement to NBC 5:
We remain deeply sympathetic to Jack Fowler and his family. Patients have been, and will always be, at the heart of what we do, and have driven our desire to research potential new therapies for more than 20 years. Guidance from regulatory agencies is that expanded access to investigational medicines and devices outside of a clinical study setting should be based on the existence of compelling evidence of efficacy, or the drug's benefit, and safety. Efficacy is typically studied in later stage trials; these data are not yet available for our investigational treatment as we have only just initiated our Phase II/III study. Shire does support, and has provided, expanded access at the appropriate stage in the development process-when clinically validated efficacy and safety information is available. We remain focused on completing our Phase II/III trial with the hope of making our investigational treatment available to as many Hunter syndrome patients as possible, as quickly as possible.