PHILADELPHIA (CBS) — An exciting new breakthrough for people with hemophilia is coming from a drug company based in Philadelphia.
It’s a rare disease but for people with hemophilia, something like a paper cut or a little scratch could be life-threatening.
Now, gene therapy the new wave of medicine – could also be helping people with this inherited disorder
This holiday season brings a special gift to Curt Krouse: hope, with a potential new treatment for the bleeding disorder his whole life.
“It’s a game changer,” Krouse, an executive director of the eastern Pennsylvania chapter of National Hemophilia Foundation.
The 50-year-old father of two was diagnosed with hemophilia at birth, people with the disease are missing a gene that makes a protein need for blood to clot
“The littlest thing could cause a bleed or I could spontaneously bleed living with hemophilia is a daily challenge,” Krouse said.
Regular injections are needed to help control hemophilia and Krouse also wears braces on his ankles to prevent bleeding.
That could all be changing with a gene therapy being developed by Spark Therapeutics.
It’s a one-time treatment that improves clotting, to significantly reduce the risk of bleeding.
New research shows it’s worked on small groups.
“It’s not a cure but it is a way that you can live a normal life, not having to worry about infusing and taking injections,” Krouse said. “It’s almost overwhelming to think what the end result can be, so it’s exciting.”
Spark Therapeutics is working with Pfizer on this new gene therapy, which is still in the early phases.
Krouse, who’s involved with of the National Hemophilia Foundation, says many people with the disease live in the Philadelphia-area because there are four hospitals with dedicated hemophilia treatment centers.