Tarin Gayles looks on in the day hospital as a sleeping Shafon is treated with Naglazyme. They fly from Pomona, Calif., to Oakland every week for treatments.
CHILDREN'S STORIES
Children's helps new drug for kids with genetic disease get FDA approval
Children with a rare genetic disorder, like Shafon Seeden, 15, pictured at left with her mother Tarin Gayles, may soon get help thanks to a new drug recently used in clinical trials at Children's Hospital & Research Center Oakland.
The U.S. Food and Drug Administration (FDA) approved a first-ever drug therapy for the rare genetic disorder, Maroteaux-Lamy syndrome, abbreviated as MPS VI.
Children's Hospital was the world's only Phase 1 clinical trials site for the drug, Naglazyme (galsulfase), and the only United States site for Phase 3 trials.
"It's the best thing they've ever done for us," said Tarin Gayles, mother of MPS VI patient Shafon Seeden, 15, who participated in the trials at Children's. "Shafon has developed more, she breathes better, walks better, has more energy and just feels better about herself." Once a week mother and daughter fly from Pomona, Calif., to Oakland for Shafon's treatments.
MPS VI patients lack an enzyme required for breaking down a complex sugar, glycosaminoglycan, into simpler molecules. Without the enzyme the sugar stays in cells, accumulating in tissues and eventually causing severe organ damage. Kids born with MPS VI have short stature, joint stiffness, respiratory and cardiac problems, and suffer vision and hearing loss.
Naglazyme, made by Novato-based BioMarin Pharmaceutical Inc., uses Aryplase, a synthetic enzyme, to mimic the body's own mechanism for breaking down the sugar. The drug is delivered during a four-hour infusion given once a week. Six patients with this rare condition were treated in the trials at Children's. There are about 1,000 known cases of MPS VI in the world.
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