COLUMBIA, SC: A Trailblazing Step in Gene Therapy
A Summerville man has made history as the first patient in South Carolina to receive a groundbreaking gene therapy treatment aimed at tackling his lifelong struggle with a rare bleeding disorder known as Hemophilia B. The procedure took place at Prisma Health’s Hemophilia Treatment Center in Columbia early Thursday morning, marking a significant milestone for both the patient and state health care.
Meet Brent Maloy
Meet Brent Maloy, a remarkable 38-year-old who has battled Hemophilia B since he was just a baby. This hereditary condition arises from a missing clotting factor—specifically, the Factor Nine protein—leaving individuals like Brent vulnerable to excessive bleeding, even from everyday activities.
“It’s been just a constant thing I had to deal with,” Brent shares, as he reflects on the challenges of having to manage a condition that often demands intensive medical attention. For many patients coping with Hemophilia B, treatment involves frequent and often complex intravenous infusions performed at home. “They typically have to infuse themselves several times a week, sometimes even more,” explains Dr. Stephanie Ambrose, a physician at the center. These infusions can lead to significant medical costs and limited lifestyle choices.
A New Hope: Hemgenix
Fortunately, Brent’s trajectory may soon take a positive turn thanks to Hemgenix, a revolutionary gene therapy that recently hit the market. While the initial price tag for this treatment is a staggering $3.5 million, healthcare professionals are optimistic about the “one and done” approach it offers. If successful, this therapy could stimulate the production of Factor Nine in Brent’s liver, potentially enabling his body to clot blood naturally.
“If everything works well, it means I can finally live my life like a normal person,” Brent says with a hint of excitement, “and not have to take any more injections—unless I have to get vaccines!”
A Cost-Effective Alternative
While the upfront cost of Hemgenix might sound astronomical, Dr. Ambrose is quick to point out that the cumulative expenses of ongoing IV treatments can amount to hundreds of thousands of dollars. “Over a lifetime, Hemgenix could actually be the more economical choice,” she emphasizes.
Future Monitoring and Expectations
For now, Brent will be under close medical supervision as they monitor the therapy’s success over the next few years. “Our hope is to get his Factor Nine levels to a sustainable point, so he won’t have to rely on IV medications,” Dr. Ambrose explains, noting that improved outcomes could lead to a significantly enhanced quality of life for Brent. “He’ll be much better protected against bleeding episodes, which would open up a world of possibilities for a more active lifestyle.”
The Only Center in South Carolina Offering Hemgenix
It’s worth mentioning that Prisma Health’s Hemophilia Treatment Center is a unique facility in the state. It’s the only federally approved center currently providing access to Hemgenix, further highlighting the importance of this treatment not only for Brent but for future patients struggling with similar conditions.
As we celebrate this breakthrough, it serves as a reminder of the incredible advancements in medical science and the hope that treatments like Hemgenix bring to individuals and families facing life-altering challenges.
With the promise of a promising new future ahead, Brent’s journey is just beginning. Who knows what great milestones and memories await as he moves closer to a life freed from the burdens of his condition?
