New treatment for Hemophilia B brings hope to patients

Life-changing treatment brings hope to patients with Hemophilia

For most people, something like a bruised knee is a minor inconvenience. But for someone with hemophilia B, it’s a medical emergency. For decades, patients have lived a life of caution, but now there could be new freedom for them through gene therapy.

(FOX 2) - A rare condition that's been around since the 1950s now has a modern treatment creating a promising future for those who've been diagnosed with Hemophilia B.

Big picture view:

For someone with hemophilia B, it’s a medical emergency. For decades, patients have lived a life of caution, but now there could be new freedom for them through gene therapy.

"It was a Band-Aid, that's all it was," said patient Stephen Werner. "If I had an injury, I'd be right back at this. There was no ‘uh oh, I have this for the rest of my life, what am I going to do?’ But it was feeling different."

Werner was diagnosed with Hemophilia B at the age of 2 and has been living with it ever since.

Hemophilia B is a rare genetic bleeding disorder that prevents blood from clotting properly. So those who are affected tend to bleed more than the average person after injury or surgery.

It's life-threatening. 

Dig deeper:

But a new, life-changing treatment is bringing patients like Stephen hope.

"It’s a one-time treatment where you deliver the gene for hemophilia factor IX," said Dr. Indryas Woldie.

It’s placed into a vector, then infused into the patient through an IV.

Karmanos Cancer Institute is becoming the first and only freestanding cancer center to treat hemophilia B with this new gene therapy, and Stephen is the 52nd patient in the country to receive it.

"There had been people who have had this done that hasn't taken so well, so that was my concern that it wouldn't take. But it did!" said Werner.

"He's doing well after treatment. He's no longer using prophylaxis, and the five-year follow-up data from other patients shows 94% of patients don't have to use prophylaxis after this infusion," said Dr. Woldie.

Werner received the hour-long IV infusion at the end of last year and says it's been a game changer, offering him a new lease on life.

"I feel fantastic. It's amazing, really, what it's done, and it's taking some concern away from me," said Werner. "I was in end-stage liver disease because of it, and now I'm perfectly healthy."

What's next:

The new gene therapy treatment is for patients who are at least 18 years old, have severe, life-threatening bleeding, and are on factor prophylaxis.

Karmanos tells FOX 2, although this has been used to treat hemophilia, it could potentially be used to treat certain cancers in the future.