Health Report: A breakthrough in hemophilia treatment

Hemophilia – once a death sentence, is now on the verge of having a cure.

An inherited disorder, mostly affecting boys, happens when blood doesn’t clot. Hemophilia can cause spontaneous and severe bleeding following an injury.

20 years ago, there were only treatments to stop the bleeding. Now, gene therapies hold even more promise of one day curing this disease.

“They look alike. Their baby pictures are identical. They act nothing alike,” said Melody Ward.

The Ward brothers may have different interests, but Jadon and Roan have more in common than you can see.

“Hemophilia is when you have a lot of bleeding,” said Roan Ward. “My blood doesn’t clot like the average human,” said Jadon Ward.

Both were born with hemophilia.

“Definitely, a bleed is the biggest threat to them,” said Melody Ward, Roan and Jadon’s Mother

“When I started 50 years ago, most of the patients with hemophilia had some kind of deformity or disability. You know, some were in wheelchairs, some wore braces, some were in crutches,” said Regina Butler, RN, hematology clinical manager at Children’s Hospital of Philadelphia

Regina butler has treated five generations of melody’s family, including her father, uncles and now her sons, who take shots every few weeks to prevent bleeding.

“The treatment has evolved so rapidly. We kept getting better and better products,” Butler said.

Now, a new gene therapy has been approved by the FDA for hemophilia B. Through a one-time IV infusion, Hemgenix instructs the body to create functional factor nine genes that will help the blood to clot.

“It’s remarkable to me. I feel like I’ve come full circle in my career with hemophilia,” Butler said.

Hemgenix can only be administered once, but its impact can last for years, making the injections the Ward boys take, obsolete.

“I didn’t think that there would be a time where I would say there was a cure for hemophilia,” Melody went on to say.