Best Life: Gene therapy shows promise for treating Hemophilia

PHILADELPHIA, Pa. (Ivanhoe Newswire) – 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. Twenty years ago, there were only treatments to stop the bleeding.

Now, gene therapies hold even more promise of one day curing this disease.

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.

“My blood doesn’t clot like the average human,” said Jadon.

Both were born with hemophilia.

“Definitely, a bleed is the biggest threat to them,” said their mother, Melody Ward.

“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, a hematology clinical manager at the Children’s Hospital of Philadelphia.

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,” said Butler.

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,” said Butler.

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,” said Melody.

The treatment is currently approved for patients 18 years and older, but doctors hope in the future to be able to treat children as young as 12.

The most common adverse reactions associated with Hemgenix include liver enzyme elevations, headaches, and flu-like symptoms.

Contributors to this news report include: Marsha Lewis, Producer; Kirk Manson, Videographer; Roque Correa, Editor.

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