Ohio State completes first gene therapy for hemophilia B

COLUMBUS, Ohio – Recently, a team at The Ohio State University Comprehensive Cancer Center – Arthur G. James Cancer Hospital and Richard J. Solove Research Institute (OSUCCC – James) completed its first gene therapy for a patient with hemophilia B, the first at an adult medical center in Ohio. Hemophilia B, an inherited bleeding disorder, happens when a gene that helps blood clot mutates or changes.

People who have hemophilia B bleed more than normal and/or longer than normal after being injured, having surgery or having dental procedures. They are also at risk of spontaneous bleeding. Bleeding, in general, can be life threatening if not treated. In more severe cases, patients need infusions on a routine basis to decrease the risk of bleeding. According to the Centers for Disease Control and Prevention, as many as 33,000 men are living with hemophilia B in the United States.

Madison Gregory IV, 53, from Toledo, Ohio, was infused with the gene therapy for his hemophilia B on Oct. 29 at the OSUCCC – James. Gregory is back home and recovering well. He no longer requires routine infusions to prevent bleeds, which he had been doing for many years, and he has not had any bleeding issues since the gene therapy.

“It is so rewarding to see Madison doing so well after this 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 in this population,” said CJ Hansen, BSN, RN, program manager and nurse coordinator of the Hemostasis and Thrombosis Treatment Center at the OSUCCC – James.

After doing some research on his own, Gregory approached his care team at Promedica – Toledo to see if he was a good candidate for the gene therapy. He was, and that’s when his doctors referred him to the OSUCCC – James for the infusion.

On that important day, Gregory was surrounded by several Ohio State care team members, family and even representatives from CSL Behring, the company that produces the gene therapy, all cheering him on during the one-hour-long infusion in an outpatient setting. Gregory stayed for observation and was back home to Toledo the next day – in good spirits and with high hopes for a cure for his life-long battle with hemophilia.

How the gene therapy works to target hemophilia B
People with hemophilia B lack factor 9, meaning their blood does not clot. Gene therapy is an approach to treatment that either introduces a functional gene or inactivates or edits the gene causing the disease. For hemophilia B, this means that an effective gene therapy could introduce a working factor 9 gene and allow patients to achieve long-term relief from the disease.

“For Madison, the infusion we administered introduced a functioning gene using a virus. Now, this new gene is working and producing the factor 9 that Madison was lacking,” said Nicolas Gallastegui Crestani, MD, a physician in hematology at the OSUCCC – James.

The gene therapy, called HEMGENIX®, is produced by CSL Behring.

Ohio State staff who were involved in this gene therapy for hemophilia case have no conflicts of interest to disclose.