Breakthrough in Pediatric Autoimmune Disease: CAR-T Cell Therapy Cures Lupus Patient
In June 2023, 15-year-old Uresa received CAR-T cell therapy at Erlangen University Hospital, marking the first use of this innovative treatment to slow the progression of systemic lupus erythematosus (SLE), a severe autoimmune disease. A year later, Uresa feels as healthy as ever, aside from a few minor colds.
Uresa is the first child treated for SLE with immunotherapy at Erlangen University's German Center for Immunotherapy (DZI). The success of this individualized treatment has been published in The Lancet.
Dr. Tobias Krickau, a pediatric rheumatologist at Erlangen University Hospital's Department of Pediatrics and Adolescent Medicine, explained the uniqueness of using CAR-T cells to treat autoimmune diseases. Previously, CAR-T therapy was approved only for certain advanced blood cancers.
After all other medications failed to control Uresa's worsening SLE, the research team faced a challenging decision: should these engineered immune cells be used for a child with an autoimmune disease? The answer was unprecedented, as no one had attempted CAR-T treatment for pediatric autoimmune diseases before.
CAR-T cell therapy involves extracting some of the patient's immune cells (T cells), equipping them with chimeric antigen receptors (CAR) in a specialized clean lab, and then reinfusing these modified cells into the patient. These CAR-T cells circulate in the blood, targeting and destroying autoreactive (harmful) B cells.
Uresa's symptoms began in autumn 2022, including migraines, fatigue, joint and muscle pain, and a facial rash—typical signs of lupus. Despite intensive treatment, her condition worsened, affecting her kidneys and causing severe complications.
In early 2023, after multiple hospitalizations and treatments, including immunosuppressive chemotherapy and plasma exchange, Uresa's condition deteriorated to the point where she required dialysis. Isolated from friends and family, her quality of life plummeted.
The medical team at Erlangen University, led by Professor Mackensen, agreed to produce and use CAR-T cells for Uresa after detailed discussions. This compassionate use of CAR-T therapy was initiated under Germany's drug law and compassionate use regulations.
The CAR-T cell therapy program at Erlangen, led by Professor Georg Schett and Professor Mackensen, has been treating patients with various autoimmune diseases, including SLE, since 2021. Their success with 15 patients was published in the New England Journal of Medicine in February 2024, and they are currently conducting the CASTLE study with 24 participants, all showing significant improvements.
To prepare for CAR-T cell therapy, Uresa underwent low-dose chemotherapy to make space for the CAR-T cells in her blood. On June 26, 2023, Uresa received her personalized CAR-T cells. By the third week post-treatment, her kidney function and lupus indicators improved, and her symptoms gradually disappeared.
The treatment process involved careful coordination to ensure the effectiveness of chemotherapy and the protection of remaining kidney function. Uresa experienced only minor side effects and was discharged on the 11th day post-treatment.
By late July 2023, Uresa returned home, completed her exams, and set new goals for her future, including becoming independent and getting a dog. She was delighted to reconnect with friends and resume a normal teenage life.
Professor Mackensen explained that Uresa still has a significant number of CAR-T cells in her blood, which means she needs monthly antibody infusions until her B cells recover. Dr. Krickau emphasized that the success of Uresa's treatment was due to the close collaboration of multiple medical disciplines at the German Center for Immunotherapy.
Uresa no longer needs any medication or dialysis, and her kidneys have fully recovered. Dr. Krickau and his team are planning further studies to explore the potential of CAR-T cells in treating other pediatric autoimmune diseases.
This landmark case demonstrates the potential of CAR-T cell therapy to provide long-term remission for pediatric patients with severe autoimmune diseases like SLE. The success of Uresa's treatment highlights the importance of early intervention and multidisciplinary collaboration. Further clinical studies are needed to confirm the long-term safety and efficacy of CAR-T cell therapy for children with autoimmune diseases.