Little Hunter Miller's motor is always running. Like most toddlers, he's sometimes one step away from trouble, but for Hunter, being rough and tumble can have serious side effects. Hunter has severe hemophilia.
Three days after he was born, a routine circumcision caused a major scare.
"You know, a baby gets up in the morning and their diapers are just full," said Hunter's grandmother, Tina Miller. "Well, his was full, but it was full of blood."
Doctors diagnosed Hunter with hemophilia A, which means his blood is missing a protein, known as clotting factor VIII. When he gets hurt, doctors need to inject the clotting factor to stop the bleeding. He's had eight emergency room visits in 19 months.
"Him falling, bumping his head too hard, little cuts. He cut the roof of his mouth with a tortilla chip and that was a hospital trip," said Heather Frederick, Hunter's mother.
Dr. Katherine Ponder studies gene therapy treatment for hemophilia and other blood disorders. Her lab treated hemophilia A in animals, but she said the therapy isn't quite ready for humans – yet.
"I think that the big question is going to be the safety," said Katherine Ponder, hematologist at Washington University School of Medicine in St. Louis.
But gene therapy has proven effective for some patients with hemophilia B. Researchers at St. Jude's Children's Research Hospital and University College of London have added the missing protein -- factor IX -- to a specially-engineered virus, which travels to the patient’s liver and transfers the gene.
"This modifies the disease from a situation where they might bleed once a week to a situation where they hardly ever bleed," Ponder explained.
Until there's a cure, Frederick walks a fine line between hovering and helping Hunter develop like any two-year old.
"You don't want to put him in a bubble," said Frederick.
Frederick said Hunter's doctors may soon put a port in his chest that would allow her to treat him with clotting factor at home.
Ponder said regular treatments of clotting factor can help keep hemophilia under control but are often costly. Those treatments can run upwards of $300,000 a year.
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