Jack Fowler is a 6 year-old boy, full of energy, love, and laughter. But Jack is dying. He suffers from MPS II (Hunter Syndrome), and the disease has begun to ravage his body. His cognitive abilities are declining rapidly, and will continue to do so until he is in a vegetative state. His only hope is to be granted permission from Shire plc to use their investigational drug, which puts the enzyme Jack is lacking directly into the brain.
This treatment has been studied in a clinical trial setting for the past 3 years. During these past 3 years, the therapy has proven very safe, with no adverse events recorded. Indeed, Shire is set to move into a Phase II/III trial because the treatment is proving safe and effective. It is saving kids, pure and simple.
Jack qualifies for this treatment through a program developed through the FDA that provides pre-approval access to investigational therapies (click HERE to see the breakdown of that program). The guidelines are very straightforward, and easily accessed with a quick application from Shire.
We have a genetics specialist – one of the best in the world – willing to provide the treatment to Jack. We have an independent review board that we are confident will approve the treatment, and a location for that treatment to take place. Funding has been made readily available to do baseline testing and to ensure all relevant safety data is collected. Everything is in place for Jack – with one exception. Shire pharmaceuticals is refusing to allow Jack to access their drug. Their rationale is weak, and their decision is akin to handing down a death sentence for little Jack. Read about our final conversation with Shire HERE.
Please post your comments below and send Shire a message. Tell them to honour their stated values creedo, which calls on them to “Be As Brave As The People We Help”. Call on Shire to do the right thing and #SaveJack.