After enduring over four decades of infusing himself with the blood clotting factor his body cannot produce, Chad Stevens, a 63-year-old from Newdale, Idaho, decided to try a novel gene therapy for his hemophilia B. This condition, caused by a genetic mutation affecting the production of factor 9 protein, has led to severe joint damage over the years. In mid-October, Stevens traveled to Omaha’s Nebraska Medical Center, becoming the hospital’s first patient to receive Hemgenix, the first gene therapy approved for hemophilia B.
Hemgenix works by using a modified virus to deliver a working gene to the liver, enabling the body to produce the necessary clotting factor on its own. Since undergoing the treatment, Stevens has not experienced any bleeds or required infusions of clotting factor. He hopes the therapy will transition him from severe hemophilia to a milder form, potentially requiring infusions only for severe trauma or surgery.
Dr. Alex Nester, a hematologist at Nebraska Medicine, expressed excitement over the treatment, highlighting its significance as a long-awaited advancement in the hemophilia community. The FDA's approval of Hemgenix in 2022 marks a pivotal moment in gene therapy, offering a new lease on life for individuals with hemophilia B. Kim Phelan, CEO of The Coalition for Hemophilia B, emphasized the therapy's potential to reduce joint damage, decrease hospitalizations, and enhance the quality of life for patients.
Despite the high cost of treatment, which can run into the millions, the benefits of gene therapy, including the possibility of a more normalized life and greater independence, are seen as invaluable. Nebraska Medicine's role in administering this therapy underscores its commitment to pioneering treatments for genetic conditions, building on its expertise in bone marrow transplants and cellular therapies. As gene therapy continues to evolve, it offers hope not only to those with hemophilia but also to patients with other genetic disorders, marking a new era in medical treatment.
