Researcher Andrés París, at the IdiPAZ-CNIO Paediatric Oncohematology Joint Clinical Research Unit. /Laura M. Lombardía. CNIO
This is the first time a CAR-T therapy has been used for this autoimmune disease. The case is presented in the 'Med’ medical journal.
"Research at the Spanish National Cancer Research Centre (CNIO) has enabled us to analyse and understand the immunological changes in the patient and therefore apply what has been learnt to future cases", says the CNIO researcher, Andrés París.
The girl has been in remission for a year; her motor condition is gradually improving and she can breathe on her own during the day.
The IdiPAZ-CNIO Paediatric Oncohematology Joint Clinical Research Unit, headed by the oncologist Antonio Pérez Martínez, is carrying out research “to solve the problems that arise in the clinic”, according to researcher Andrés París. This week, the group has announced how it has successfully addressed the case of a paediatric patient with anti-MDA5 dermatomyositis, a very rare autoimmune disease (one case per million inhabitants) that affects the immune system and has a mortality rate of around 60%.
The patient has been treated at the public La Paz University Hospital in Madrid. The team of oncologists and researchers decided to treat her with a CAR-T CD19 therapy, more specifically with the ARI-0001 advanced cell therapy, produced by the Hospital Clinic in Barcelona. The therapy was administered for compassionate use, as there were no other therapeutic options and the girl was in intensive care.
“This is the first time a CAR-T therapy has been used for this rare autoimmune disease” explains París, the lead author of the publication that presents the case in the Med medical journal. “It is a relevant case because anti-MD5 dermatomyositis is one of the most complicated diseases in medical practice, and has no specific treatments”.
An “academic” CAR-T
CAR-T therapy had never previously been used for this pathology. It is a type of therapy used in oncology to make the patient’s defence cells – lymphocytes – to target and destroy tumour cells. The CAR-T CD19 therapy used in this case is ARI-0001, produced by the Hospital Clinic in Barcelona and the first CAR-T developed entirely in Europe to be approved by a regulatory agency.
The strategy succeeded in safely restarting the part of the immune system that caused the disease and enabled the patient to be maintained without the need for other treatment. The girl has been in remission for a year; her motor condition is gradually improving and she has gone from breathing on mechanical ventilation and ECMO (extra-corporeal ventricular assist ventilation) to breathing on her own during the day.
Research to apply what has been learnt to future cases
“The CNIO research has enabled us to delve deeper at a molecular and cellular level in this case,’ says París. ‘It has enabled us to go beyond the positive clinical outcome and to analyse what immunological changes have occurred in the patient over the course of this year at a molecular and cellular level, so that future cases that are likely to benefit from CAR-T autoimmune therapy can use this data”.
This result has been possible thanks to a multidisciplinary team from different paediatric services at La Paz University Hospital; researchers from the La Paz Research Institute (IdiPAZ) and the CRIS Advanced Cancer Therapies Unit; the Niño Jesús Hospital; the Immunology Service at the Hospital Clinic in Barcelona; and, of course, the CNIO.
Reference article
París-Muñoz A, Alcobendas-Rueda RM, Verdú-Sánchez C, Udaondo C, Galán-Gómez V, González-Martínez B, Menéndez JJ, Martínez-Romera I, Minguillón J, Pertíñez L, de Manuel-Gómez C, de la Cruz-Benito A, Sanz-Rupérez A, Remesal A, Cámara C, Sánchez-Zapardiel E, Del Pino-Molina L, Gómez-Zamora A, Serrano-Olmedo MG, Español-Rego M, Ruiz de Valbuena M, Climent F, Dorao P, Ríos-Blanco JJ, Andrade JD, Ruiz-Zurita G, Fernández-García MA, Pérez-Martínez A. CD19 CAR-T cell therapy in a pediatric patient with MDA5+ dermatomyositis and rapidly progressive interstitial lung disease. Med. 2025 Apr 25:100676. doi: 10.1016/j.medj.2025.100676.