Eleven-year-old girl with a scarce autoimmune condition receives successful CAR-T treatment.
Madrid Breakthrough: 11-Year-Old Girl Recovers from Rare Disease using Groundbreaking CAR-T Therapy
In a remarkable medical feat, Madrid's public healthcare system has successfully treated an 11-year-old girl suffering from a rare and life-threatening autoimmune disease using CAR-T therapy. This milestone marks the first worldwide use of this treatment for this type of condition in a pediatric patient.
The girl was diagnosed with anti-MDA5-positive dermatomyositis, a condition characterized by rapid and severe damage to the lungs. This disease has a high mortality rate (between 30% and 66%) in the first six months after onset, making it incredibly rare, with only one case per million inhabitants.
The groundbreaking therapy was applied on a compassionate basis when all other treatment options had failed. The girl, who was critically ill in the Intensive Care Unit (ICU), has been in remission from the disease for a year and no longer requires immunosuppressant treatment. Not only that, but her motor capacity is improving, and she can now breathe on her own without needing mechanical ventilation or extracorporeal ventricular assistance.
Rosa Alcobendas, the coordinator of the Multidisciplinary Unit for Rare Diseases at La Paz Hospital, shared the improvements the girl has made. "She is now 11 years old, but at 10 years old, she was hit with a rare disease, anti-MDA5-positive dermatomyositis, which caused severe skin and lung damage that left her dependent on a machine, despite having received multiple lines of immunosuppression," Alcobendas explained.
The therapy used on the girl, known as CAR-T therapy, is usually employed in cases of leukemia. However, in this case, it was used to target the deeper tissues involved in this autoimmune disease. Andrés París, a researcher at IdiPaz, explained, "This therapy seems to be able to deeply reset a part of the immune system, specifically B lymphocytes, which play a fundamental role in this disease."
The success of this treatment is a testament to the collaboration between Madrid's public health system and other Administrations, including the Hospital Infantil Universitario Niño Jesús, the Hospital Clínic de Barcelona, and the National Cancer Research Centre. A multidisciplinary team of healthcare professionals from various services at La Paz Hospital worked together, including Rheumatology, Hematology-Oncology, Endocrinology and Nutrition, Neurology, Pulmonology, Immunology, Internal Medicine, Rehabilitation, and ICU, as well as staff from the IdiPaz Research Institute and the Advanced Therapies Unit of the CRIS Foundation against Cancer.
While the specific details about the treatment and the medical team involved in this case are not fully outlined in available search results, a recent study has reported on the use of CD19 CAR-T cell therapy in a pediatric patient with aggressive MDA5+DM-RPILD (rapidly progressive interstitial lung disease). This therapy targets CD19+ B lymphocytes, which are suspected to be present in deeper tissues, and has shown promise in treating complex autoimmune diseases refractory to multiple immunosuppressant treatments.
This pioneering effort in using CAR-T cell therapy for complex autoimmune diseases could pave the way for future breakthroughs in treating various rare and life-threatening conditions.
- This groundbreaking CAR-T therapy, initially used for leukemia, was employed to target the deeper tissues involved in chronic diseases like autoimmune disorders, demonstrating potential for health and wellness improvements in such cases.
- The success of the experiment on the 11-year-old girl with anti-MDA5-positive dermatomyositis shows promise for therapies and treatments aimed at managing complex medical-conditions like chronic diseases, particularly autoimmune disorders.
- The collaboration between Madrid's public health system and other Administrations resulted in the use of a novel CD19 CAR-T cell therapy for a rare and life-threatening autoimmune disorder, indicating the potential for this treatment in dealing with chronic diseases and medical-conditions.
