The family of a child with a rare disease is fighting for access to a medicine that could save their son’s life. But the company that owns the medicine, Shire Pharmaceuticals, isn't willing to administer the drug.
Six year old Jack Fowler suffers from MPS II (Hunter Syndrome), a rare and fatal condition seen in only 2,000 people worldwide. Children with MPS II lack an enzyme in their blood that breaks down cellular waste. As a result, cellular waste builds up in the sufferer’s body and eventually kills them.
In the past, children born with MPS II had no hope of being cured. Shire Pharmaceuticals is changing that. The company is in the late stages of clinical trials for a drug with incredible potential. The drug, which does not yet have a name, is injected directly into the brain of an MPS II sufferer. It contains the enzyme that patients with MPS II lack, and enables their bodies to break down cellular waste as a healthy body would.
Shire has completed testing phases I/II for the drug with great success. They are currently in the midst of phase II/III trials. According to FDA guidelines, drugs in the II/III trial phase can be administered to outside patients if “the potential benefit justifies the potential risks of the treatment.”
For patients in the clinical trials, the drug has literally been a life saver. Children with MPS II have regained motor and cognitive functions once lost to their condition. Deb Purcell, whose son Trey has been administered the drug in trials, says it has saved his life.
“To a parent who hasn’t had to face the death of their child, it is hard to explain,” Purcell said. “This treatment has given my son a new lease on life; a second chance. It’s the hope we’ve been searching for.”
The Fowler family wants nothing more than for Jack to experience the same life-saving benefits of the drug, but Shire is refusing to grant him access to it.
“Our son is dying,” mother Jamie Fowler said. “Knowing that there is a treatment that can save my son but having it denied to him is heartbreaking and devastating. It’s cruel for Shire to sit idly by while my son slowly dies when they know they can save him.”
To aid their cause, the Fowler’s enlisted the help of the Isaac Foundation, a research and advocacy organization specializing in MPS and MPS related diseases. The foundation’s director, Andrew McFadyen, is calling on Shire to use common moral sense and grant Jack access to the drug.
“Shire has the ability to step in and save this child,” McFadyen said. “FDA regulations allow for expanded-use of investigational drugs in cases like this. As a company developing life changing and life-saving medications, it’s their responsibility to provide those medications to those that most need them. To do otherwise is inhumane and shows a callous disregard to human life.”
For more information on the case, check out this video created by the Isaac Foundation and the Fowler family: