Systemic sclerosis (SSc) is a severe autoimmune disease characterized by hardening and scarring of the skin and internal organs. Despite numerous research efforts, the treatment of SSc remains a major challenge, as many patients do not respond adequately to existing therapies. Systemic sclerosis (SSc) is one of the most serious rheumatic diseases, which is why new and effective treatment methods are particularly important.
What is CAR-T cell therapy?
CAR T cells are T cells of the immune system that have been genetically modified so that they can specifically recognize and destroy cancer or autoimmune cells. In CD19-directed CAR-T cell therapy, the so-called CD19 receptor, which is located on the surface of B cells, is used as a target. By specifically eliminating these B cells, the therapy can have a profound effect on the immune system, possibly leading to a long-lasting remission.
Current study situation on CAR-T therapy for SSc
One innovative approach is therapy with BMS-986353, a CAR-T cell product that specifically targets CD19-positive B cells. As part of the phase 1 study "Breakfree-1", BMS-986353 was investigated in patients with severe, therapy-resistant autoimmune diseases, including SSc. The main focus here was on safety and initial indications of efficacy.
The initial results from the SSc patient collective are promising:
- Safety:The treatment was well tolerated. Side effects, such as cytokine storm syndrome, occurred in less than half of the patients and were predominantly mild, short-lived and reversible.
- Skin improvement:Patients with diffuse cutaneous SSc showed a significant reduction in skin thickening. The standardized modified Rodnan skin score (mRSS) improved by an average of 14 points within three months. This improvement remained stable at the last follow-up examination.
- Lung function:Patients with concomitant lung involvement (interstitial lung disease, ILD) experienced an increase in forced vital capacity of up to 7 percent within about six months.
- Less medication:Fortunately, 8 out of 9 patients were able to discontinue all previously used immunosuppressive drugs after CAR-T therapy, indicating a potential permanent remission and a "resetting" of the immune system.
Significance for the future of SSc treatment
The available data show that the CD19-targeted CAR-T cell therapy BMS-986353 is a promising new therapeutic approach for severe, therapy-resistant systemic sclerosis. This therapy could significantly expand the treatment spectrum and in particular help patients who have so far hardly benefited from other therapies.
With the potential ability to bring the disease under control in the long term and without ongoing immunosuppression, this treatment opens up new perspectives for the quality of life and prognosis of SSc patients.
We are following the further development of this innovative therapy with great interest and will keep you up to date on upcoming study results and approvals.
