Silver Spring Woman Becomes First Adult Sickle Cell Disease Patient in Maryland to Receive FDA-Approved Gene Therapy
Jessica Ceja, a patient liaison at a Washington, D.C. hospital, received the cells by infusion on April 27. She is the first adult patient with sickle cell anemia in Maryland to receive the gene therapy, called Lyfgenia, which was approved by the U.S. Food and Drug Administration (FDA) in December 2023. A 12-year-old boy from Clinton, MD., was the first person in the U.S. to be treated following commercial approval.
Sickle cell disease is an inherited blood disorder that affects about 100,000 people in the United States and millions worldwide. More than 5,000 Marylanders are living with the disease, and the state ranks fifth nationally in prevalence. The condition most commonly affects people of African descent but also occurs in Hispanic and Middle Eastern populations.
Patient Jessica Ceja with her medical team at the University of Maryland Medical Center
“We are very excited about this transformative, potentially curative therapy for patients with severe sickle cell disease,” said Jean A. Yared, MD, a hematologist and Medical Director of Clinical Operations at the University of Maryland Marlene and Stewart Greenebaum Comprehensive Cancer Center (UMGCCC) and Professor of Medicine at the University of Maryland School of Medicine (UMSOM). “Bringing gene therapy for sickle cell disease to Maryland has been a major institutional effort over the past few years. Our goal is not only to offer this therapy, but to develop a sustainable program that expands access to more patients across the region and gives them a realistic chance at a cure.”
Yared, the medical director of the Transplantation and Cellular Therapy (TCT) Program’s day hospital, and Jennie Y. Law, MD, Associate Section Chief for Classical Hematology and Sickle Cell Disease at UMGCCC and Associate Professor of Medicine at UMSOM, partnered to lead the development and clinical implementation of the sickle cell gene therapy program, which focuses on providing supportive, compassionate care and achieving the best possible outcomes for patients.
“Ms. Ceja’s disease has significantly impacted her quality of life with frequent hospital admissions to manage severe pain crises and reliance on regular blood transfusions for disease management. Given that she does not have an available donor for a stem cell transplant, she was an ideal candidate for gene therapy consideration,” said Law, Ceja’s hematologist.
In sickle cell disease, a mutation in hemoglobin causes red blood cells to become crescent-shaped and obstruct blood flow, leading to severe pain episodes known as vaso-occlusive crises as well as progressive organ damage. This new therapy uses a patient’s own stem cells, which are collected and genetically modified in a laboratory 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.
The therapy is approved for patients aged 12 and older who have severe, recurrent vaso-occlusive events that are not adequately controlled with standard treatments.
The launch of the gene therapy program at UMGCCC represents the culmination of a multidisciplinary effort to build the infrastructure required to safely deliver complex cellular and gene therapies. This included developing clinical protocols, coordinating care across transplant, classical hematology, apheresis and inpatient teams, and establishing long-term follow-up pathways. The program builds on UMGCCC’s established leadership in cellular therapies, including CAR T-cell therapy, and expands its capabilities into curative gene-based treatments.
In clinical trials for the approval of Lyfgenia and a second gene therapy called Casgevy, 88 to 93 percent of sickle cell patients who received the gene therapy were free from pain crises following treatment. The therapy is now being covered by major insurers and Medicaid in many states, including Maryland, through developing outcome-based payment models.
‘Excited Us as a Family’
Ceja was diagnosed with sickle cell disease at age 2 and has been treated over the years with pain medications, blood transfusions and hydroxyurea, a drug that reduces pain episodes and transfusion needs. She described the possibility of a life without the disease as both remarkable and difficult to imagine.
“This treatment was something that excited us as a family, with the prospect of my being cured using my own stem cells and not having to look for a donor,” she said.
Ceja, an only child, was unable to find a suitable donor among family members to have a stem cell transplant, which has been shown to cure sickle cell disease.
She said she looks forward to being more present for her family. “Not to have so many hospital visits, not to miss out on family events, not to miss out on my children’s activities, their lives, and to be able to plan ahead and participate,” she said. “It’s all of the things that I’ve felt that I have missed out on.”
She and her husband, Darwin, have three children, ages 5 to 18.
“For the longest time, the only medication people with sickle cell disease had available was hydroxyurea. To have something like this, with the potential for such meaningful outcomes, is truly remarkable,” she said.
Ceja expressed deep appreciation for her medical team and her family. “I couldn’t be here without the support of my husband, my parents, and my kids. It’s not an easy process. I’m glad that I found the right place that was able to present this option to us,” she said.
“Sickle cell disease is a devastating illness that shortens life expectancy by approximately 20 years and significantly affects quality of life,” said Taofeek K. Owonikoko, MD, PhD, Executive Director of UMGCCC and the Kevin J. Cullen, M.D. Distinguished Professor of Oncology at UMSOM. “Our cancer center has been a regional leader in CAR T-cell therapy and other cell-based therapies, and we are pleased to extend these capabilities to gene therapy for sickle cell disease to help eliminate severe pain crises and improve patients’ lives.”
‘Just the Beginning’
Mark T. Gladwin, MD, Dean of the University of Maryland School of Medicine, Vice President for Medical Affairs at the University of Maryland, Baltimore (UMB), and the John Z. and Akiko K. Bowers Distinguished Professor said: “Sickle cells can block blood flow to nearly every organ, causing excruciating pain—like having a migraine throughout the entire body, affecting the bones, back, limbs, and chest. Curative gene therapies, in which patients receive their own genetically modified bone marrow cells, represent breathtaking advances that have captured global attention. Cell-based and gene therapies are the cornerstone of precision medicine, and we are committed to bringing these innovative treatments to the patients who need them most.”
Gladwin has served as principal investigator on numerous sickle cell clinical trials and is currently leading a large international study evaluating red cell exchange transfusion to improve long-term outcomes.
“This is just the beginning,” Yared said. “We have established a comprehensive framework to evaluate and treat patients with gene therapy, and we expect to expand this program in the coming months. Our focus is to ensure that eligible patients have access to these highly specialized therapies while maintaining the highest standards of safety and long-term follow-up.”
Data from clinical studies of Lyfgenia (lovotibeglogene autotemcel) demonstrated elimination of vaso-occlusive events in 88 percent of patients 6 to 18 months after treatment. Among patients treated with Casgevy (exagamglogene autotemcel), which uses CRISPR-based genome editing, more than 90 percent were free from pain crises for at least 12 consecutive months.
UMMC is a qualified treatment center for both gene therapies and currently the only center in Maryland offering these treatments. Click here for more information about the sickle cell treatment program.
About the University of Maryland Marlene and Stewart Greenebaum Comprehensive Cancer Center
The University of Maryland Marlene and Stewart Greenebaum Comprehensive Cancer Center (UMGCCC) is a National Cancer Institute-designated comprehensive cancer center within the University of Maryland Medical Center in Baltimore, the flagship academic hospital of the University of Maryland Medical System. It offers a multidisciplinary approach to treating all types of cancer and has an active clinical and basic science research program through its relationship with the University of Maryland School of Medicine. The center is ranked among the top 50 cancer programs in the country by US News & World Report. umgccc.org
About the University of Maryland Medical Center
The University of Maryland Medical Center (UMMC) is comprised of two hospital campuses in Baltimore: the 800-bed flagship institution of the 11-hospital University of Maryland Medical System (UMMS) and the 200-bed UMMC Midtown Campus. Both campuses are academic medical centers for training physicians and health professionals and for pursuing research and innovation to improve health. UMMC's downtown campus is a national and regional referral center for trauma, cancer care, neurosciences, advanced cardiovascular care, and women's and children's health, and has one of the largest solid organ transplant programs in the country. All physicians on staff at the downtown campus are clinical faculty physicians of the University of Maryland School of Medicine. The UMMC Midtown Campus medical staff is predominately faculty physicians specializing in a wide spectrum of medical and surgical subspecialties, primary care for adults and children and behavioral health. UMMC Midtown has been a teaching hospital for 140 years and is located one mile away from the downtown campus. For more information, visit umms.org/ummc.
About the University of Maryland School of Medicine
The University of Maryland School of Medicine, established in 1807 as the first public medical school in the U.S., continues today as one of the fastest growing, top-tier biomedical research enterprises in the world. The School has nearly $500 million total research funding, 46 departments, centers, and institutes, more than 2,200 student trainees and over 3,000 faculty members, including notable members of the National Academy of Medicine. As the largest public medical school in the DC/MD/VA region, faculty-physicians are working to help patients manage chronic diseases like obesity, cancer, heart disease and addiction, while also working on cutting-edge research to address the most critical generational health challenges. In 2024, the School ranked #12 among public medical schools and #27 among all medical schools for R&D expenditures by the National Science Foundation. With a $1.3 billion total operating budget, the School partners with the University of Maryland Medical Center to serve nearly 2 million patients annually. The School's global reach extends around the world with research and treatment facilities in 33 countries. In Maryland, the School of Medicine is spearheading new initiatives in AI and health computing and partnering with the University of Maryland BioPark to develop new medical technologies and bioengineering ventures. For more information, visit medschool.umaryland.edu.
