Gene therapy helps hemophilia patient overcome bleeding concerns

Madison Gregory IV had to get regular IV infusions for years because his lifelong hemophilia B disorder left him at risk of dangerous bleeding episodes.

So, when he learned about a gene therapy to treat the disease, he wanted to know more.

The 53-year-old did some research before reaching out to his doctors at Promedica – Toledo to ask if the treatment would be a good fit.

After determining it would be, they referred him to The Ohio State University Comprehensive Cancer Center – Arthur G. James Cancer Hospital and Richard J. Solove Research Institute (OSUCCC – James), where he received the therapy in October, becoming the first patient to undergo the treatment at an adult medical center in Ohio.

Successful hemophilia B treatment

Since the treatment, he hasn’t had any bleeding issues, and he no longer requires the routine infusions of a clotting factor that prevented bleeding.

CJ Hansen, BSN, RN, program manager and nurse coordinator of the Hemostasis and Thrombosis Treatment Center at the OSUCCC – James, says it’s rewarding to see Madison doing so well following the innovative treatment.

“This is just the tip of the iceberg here at Ohio State, and we hope to bring this new treatment option to many more patients,” Hansen says.

About hemophilia B

Hemophilia B, an inherited bleeding disorder, is caused when there’s a mutation or change to a gene that affects the ability of the body to produce one of the factors (called factor IX) that helps the blood clot, explains Nicolas Gallastegui Crestani, MD, a hematology physician and medical director of the Hemostasis and Thrombosis Treatment Center at the OSUCCC – James.

People who have hemophilia B bleed more or longer than normal after being injured or having surgery or dental procedures. They’re also at risk of spontaneous bleeding.

Left untreated, bleeding can be life-threatening for people with hemophilia. In more severe cases, like Madison’s, patients need factor IX infusions on a routine basis to decrease the risk of bleeding.

How gene therapy targets hemophilia B

The therapy adds a functional gene to the body so it can start producing factor IX.

This helps blood clot and promises to give patients long-term relief from hemophilia B and from the burden of treatment, Dr. Gallastegui Crestani says.

Madison received the gene therapy, called HEMGENIX®, during an hour-long infusion on an outpatient basis. He followed up the next day at the OSUCCC – James before returning to Toledo.

The gene was carried into the body on an inactive virus.

“For Madison, the infusion we administered introduced a functioning gene using a virus,” Dr. Gallastegui Crestani says. “Now, this new gene is working and producing the factor IX that Madison was lacking.”