17 December 2024
Press
- Autoimmune and auto-inflammatory diseases
The CRI-IMIDIATE network, with F-CRIN accreditation, presents an innovative therapeutic approach and reminds us of the crucial role of National Diagnostic and Care Protocols (PNDS) for rare and autoimmune diseases. These themes will be at the heart of its 8th Research Day, organized jointly with the FAI2R rare disease network, on January 15, 2025 in Paris.
A new weapon against autoimmune diseases
Autoimmune diseases, which affect nearly 3 million people in France and up to 10% of the world's population, are caused by a disturbance in the immune system. These pathologies, which affect a large majority of women (80% of cases), include more than 80 conditions such as lupus, multiple sclerosis, rheumatoid arthritis and scleroderma. They can have a profound impact on patients' quality of life, and often require lifelong medical follow-up. As most of these diseases are multi-factorial, they require considerable research and early diagnosis to ensure the best possible treatment for patients. Despite advances in biotherapies over the past 25 years, which have put a growing number of patients into remission (up to 70% in the case of rheumatoid arthritis), some diseases remain refractory to current treatments. What's more, while these biotherapies can effectively target the cytokines or circulating cells involved in these diseases, they sometimes struggle to reach the tissues affected by the disease. With World Rare Disease Day just a few weeks away,the CRI IMIDIATE network wishes to highlight an innovative therapeutic approach and emphasize the importance of PNDS to better manage inflammatory and autoimmune diseases.
CAR-T cell technology
CAR-T cells(T lymphocytes carrying a chimeric antigen receptor), true "killer cells", represent a major advance in the treatment of autoimmune diseases, offering the hope of lasting remissions for patients previously refractory to conventional treatments. These laboratory-modified white blood cells are designed to specifically target and destroy the cells responsible for autoimmune reactions and inflammation found in the blood or tissues (joints, brain, muscles...). Initially developed to treat certain blood cancers, this approach has already shown spectacular results, notably in the treatment of severe systemic lupus, where lasting remissions have been observed. These remissions, which suggest a resetting of the immune system, pave the way for promising therapeutic developments. Other autoimmune diseases, such as lupus, inflammatory myopathies, scleroderma, Goujerot-Sjögren syndrome, or rheumatoid arthritis could benefit.
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Although their production is costly and complex (harvesting the patient's T lymphocytes, which are then genetically modified in the laboratory to integrate the specific "CAR" receptor, and reinjected into the patient), these treatments are very well tolerated in the short term and could transform the management of autoimmune diseases in refractory patients: "It's our role to disseminate information, to really identify centers with expertise in these diseases so that we can actively participate in the recruitment of these refractory patients who could benefit from this new approach. The technique is really interesting and will be the focus of new academic and industrial projects. This complex but short treatment could be a revolution in the management of these diseases and transform the way we care for these patients" explains Pr Bruno FAUTREL, Co-coordinator of the CRI-IMIDIATE network (F-CRIN) and Head of the Rheumatology Department at Pitié-Salpêtrière (AP-HP).
National Diagnosis and Care Protocols (PNDS): an essential guide for rare diseases
In the field of rare diseases, the small number of patients often makes it difficult to carry out randomized clinical trials.NDPs therefore play a central role. These protocols, drawn up by experts, provide clear, standardized recommendations on the diagnosis, follow-up and treatment of these complex pathologies. They harmonize medical practices and reduce geographical disparities in care. They enable access to treatments that do not yet have marketing authorization. The FAI²R (Filière de santé pour les maladies auto-immunes et auto-inflammatoires rares) and the CRI IMIDIATE network have worked hard to raise the level of expertise in patient care.
In the case of very rare diseases that can lead to serious sequelae (articular, neurological, etc...) in patients, as is the case with interferonopathies, (complex and little-known pathologies, which group together disorders linked to excessive activation of the immune system, often detected in infants: 1-2 years old), the diagnosis must be made quickly. Therecent PNDS, which can be consultedhere, enables rapid recognition of symptoms, effective referral to reference centers and access to innovative treatments, such as inhibitors of the interferon pathway, while improving patients' quality of life.
The PNDS are now the reference for the diagnosis and treatment of rare diseases. "We've been working with patients and experts in these diseases for 10 years, contributing to the PNDSs to improve the management of auto-inflammatory or auto-immune diseases. It's not widely known. Genetic research is going to take some time to come to fruition. In the meantime, it's important to make doctors, patients and patients' families aware of the existence of PNDSs to better manage these complex diseases" adds Pr Fautrel.
An unmissable event
The 8th CRI-IMIDIATE/FAI2R Research Day, to be held at the Pitié-Salpétrière Hospital on January 15, 2024, will be an opportunity to discuss these advances. This event organized by FAI²R (Filière de santé pour les maladies auto-immunes et auto-inflammatoires rares) and the Réseau CRI-IMIDIATE - labellisé F-CRIN will bring together healthcare professionals, researchers, patient associations and industrial players to foster exchanges and accelerate innovation.
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