Skip to navigation

What we're up to

News Friday, September 15, 2017

Enzyme replacement therapy a success

Patient with hypophosphatasia shows drastic improvement

Knee before and afterDevon is your typical 11-year-old boy – he plays with other kids, and runs and jumps, and can basically do whatever he wants. However, a few months ago this active lifestyle was not possible. Devon has hypophosphatasia (HPP), a rare disorder that causes his bones to be soft and requires him to be more careful when physically active. He now has the physical abilities of other children his age since starting a new therapy, and his family couldn’t be happier with the results.

This enzyme-replacement therapy was studied in clinical trials at the Center for Metabolic Bone Disease and Molecular Research at Shriners Hospitals for Children — St. Louis, and approved in 2015 by the FDA. “He’s like a normal 11 year old now, it’s awesome,” says dad Bela. “He used to get tired very easily and could not jump much at all. That’s all changed now. He’s a more confident, happier kid.”

When diagnosed with HPP at six months of age, Devon’s parents searched the internet for a treatment option. They found Michael P. Whyte, M.D., and his team at the St. Louis Shriners Hospital Research Center. Other than monitoring his calcium intake, Devon’s parents did not have any other option to help strengthen his bones. Then they learned that some HPP patients were involved in a clinical trial for a new “biologic” that could help treat their son’s symptoms.  Although Devon was too young to be included in the trial, once the drug was approved by the FDA his parents sought treatment.

Devon injects Strensiq® (asfotase alfa) under his skin six days per week. In the few months since he started, he has already experienced a significant change in his muscle strength and endurance. Improvements in his X-rays show that the medicine is working to heal his bones. His father says Devon is overall a happier child with so much more energy than before taking Strensiq®, and they appreciate the role Shriners Hospitals for Children has played in his care.

“Shriners is awesome, everybody here is professional and caring. It’s done so much for us,” says Bela.

Devon may take the drug until he is an adult and perhaps afterwards. The Research Center currently has 22 patients taking Strensiq® and all have seen improvements. The patients live throughout the United States and range from age 4 to 21.