A 41-year-old Silver Spring woman has become the first adult patient with sickle cell disease in Maryland to receive gene therapy treatment with Lyfgenia at the University of Maryland Medical Center. Jessica Ceja, a patient liaison at a Washington, DC, hospital, received the genetically engineered stem cells on April 27. This milestone follows FDA approval of the therapy in December 2023.
Sickle cell disease is an inherited blood disorder affecting about 100,000 people in the United States and millions worldwide. More than 5,000 Marylanders live with the disease, which most commonly affects people of African descent but also occurs in Hispanic and Middle Eastern populations. The condition causes red blood cells to become crescent-shaped, obstructing blood flow and leading to severe pain episodes called vaso-occlusive crises and progressive organ damage. Ceja endured debilitating pain crises and frequent hospitalizations throughout her life, requiring pain medications, blood transfusions, and hydroxyurea treatment.
The therapy works by collecting a patient’s own stem cells and genetically modifying them to produce an anti-sickling form of hemoglobin. Patients receive high-dose chemotherapy to clear diseased stem cells from the bone marrow before the modified cells are reinfused. Clinical trials showed that 88% of Lyfgenia patients were free from pain crises 6 to 18 months after treatment. Ceja was an ideal candidate because she did not have an available donor for a traditional stem cell transplant, which has been shown to cure sickle cell disease.
The launch of the gene therapy program at the University of Maryland Marlene and Stewart Greenebaum Comprehensive Cancer Center represents a multidisciplinary effort to build infrastructure for complex cellular and gene therapies. The therapy is now covered by major insurers and Medicaid in many states, including Maryland. Ceja expressed gratitude for her medical team and family support, looking forward to fewer hospital visits and greater participation in her children’s lives.
This article is an AI-assisted summary. All facts and figures are drawn from the original report: https://www.newswise.com/articles/silver-spring-woman-becomes-first-adult-sickle-cell-disease-patient-in-maryland-to-receive-fda-approved-gene-therapy/?sc=rsmn