CAR-T cell, new weapon against autoimmune diseases

CAR-Ts, “killer cells” autoimmune diseases? These white blood cells modified in the laboratory have a mission: to specifically target and destroy the cells responsible for inflammation which are found in the blood or tissues (joints, brain, muscles, etc.). A few weeks before International Rare Disease Day on February 28, 2025, the CRI-IMIDIATE (F-CRIN) network is highlighting this new innovative therapeutic approach.

80 autoimmune diseases: 3 million French people

As a reminder, autoimmune diseases, which affect nearly 3 million French people and up to 10% of the world population, are caused by a dysregulation of the immune system. These pathologies, which mainly affect women (80% of cases), include more than 80 conditions such as lupus, multiple sclerosis, rheumatoid arthritis and even scleroderma. They can have a significant impact on the quality of life of patients and often require lifelong medical monitoring. Being mostly multi-factorial, they require considerable research work and early diagnosis to best treat patients.

Diseases refractory to current treatments

“Despite advances in biotherapies (editor’s note: treatments using biological or biotechnological drugs, generally having a more targeted action than conventional drugs) for 25 years, which have made it possible to put a growing number of patients into remission (up to 70% in the case of rheumatoid arthritis), certain pathologies remain refractory to current treatments”, observes CRI-IMIDIATE. These genetically modified white blood cells, called “T lymphocytes carrying a chimeric antigen receptor”, intend to change the situation.

“Spectacular results” on severe systemic lupus

Initially developed to treat certain blood cancers, this approach has already shown “spectacular results”, particularly in the treatment of severe systemic lupus, where lasting remissions have been observed. These remissions, which suggest a reset of the immune system, “open the way to promising therapeutic developments”, according to this network of excellence in clinical research which aims to improve the management of osteoarticular or systemic inflammatory pathologies. Other autoimmune diseases, such as inflammatory myopathies, scleroderma, Goujerot-Sjögren syndrome or rheumatoid arthritis could benefit.

Identify refractory patients

Although their production is expensive and complex (collection of the patient’s T lymphocytes, which are then genetically modified in the laboratory to integrate the specific receptor « CAR », then reinjected into the patient), these treatments are very well tolerated in the short term and could transform the management of autoimmune diseases in patients refractory to conventional treatments.

A revolution in the management of autoimmune diseases?

“It is our role to disseminate information, to identify centers which have expertise in these diseases in order to actively participate in the recruitment of these people who could benefit from this new approach. The technique is really interesting and will be the subject of new academic and industrial projects. This complex but short treatment could be a revolution in the care of these patients,” explains Professor Bruno Fautrel, co-coordinator of the CRI-IMIDIATE network and head of the rheumatology department at Pitié-Salpêtrière in Paris (AP-HP).

The major role of national diagnosis and care protocols

Another lever to improve the management of these pathologies: national diagnostic and care protocols. “In the field of rare diseases, the low number of patients often makes it difficult to carry out randomized clinical trials (editor’s note: whose participants are distributed randomly. This technique makes it possible to limit bias when testing the effectiveness of a new treatment), he underlines. The PNDS therefore play a central role. » These protocols, developed by experts, provide clear and standardized recommendations on the diagnosis, monitoring and treatment of these complex pathologies. “They harmonize medical practices and reduce geographical disparities in care but also allow access to treatments which do not yet have marketing authorization,” continues the F-CRIN labeled network.

Rapid recognition of symptoms

In the case of very rare diseases that can cause serious after-effects (articular, neurological, etc.) in patients, such as is the case in interferonopathies (complex and little-known pathologies, which include disorders linked to activation excessive immune system, often detected in young children), “the diagnosis must be made quickly”. The recent PNDSallows a “rapid recognition of symptoms by healthcare professionals, effective referral to reference centers and access to innovative treatments, while improving the quality of life of patients”.

Raise awareness among doctors and patients about PNDS

“PNDS are now the reference for the diagnosis and treatment of rare diseases”, insists Professor Fautrel. And yet… “It’s relatively little known. Genetic research will take some time to be successful. In the meantime, it is important to raise awareness among doctors, patients and their families of the existence of PNDS to better manage these complex diseases. he concludes.

An event on January 15 to accelerate innovation

On January 15, the Health Network for Rare Autoimmune and Autoinflammatory Diseases (FAI²R) and the CRI-IMIDIATE network are organizing a conference, within the hospital of the Pitié-Salpêtrière (Paris 13) to highlight these “advanced”. Objective ? Bring together healthcare professionals, researchers, patient associations and industrial players to promote exchanges and accelerate innovation. To participate (for free), simply fill out the registration form before January 12, 2025 midnight. Discover the complete program online.

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