We described two cases as individual healing trials with immune neuropathies, which were treated with CAR T-cells, CD19 targeting CAR T-cells. And we see very exciting results in these patients. We never believed before we did that. They fully recovered in their disease activity and before they were very severe disease course. And we see that not only in the clinical improvement, we see also biomarkers which improved very well, SNFL as well as the electrophysiological measurements of this patient recovered very well...
We described two cases as individual healing trials with immune neuropathies, which were treated with CAR T-cells, CD19 targeting CAR T-cells. And we see very exciting results in these patients. We never believed before we did that. They fully recovered in their disease activity and before they were very severe disease course. And we see that not only in the clinical improvement, we see also biomarkers which improved very well, SNFL as well as the electrophysiological measurements of this patient recovered very well. So that’s for us a new step for neuroimmunological diseases to treat with. From the safety perspective we see very mild side effects. The main side effects of CAR T-cells are CRS and ICANS, syndromes of cytokine release and neurotoxicity, and we see only mild effects, very manageable. And the long-term side effects, as we see, is a hypogammaglobulinemia, but it doesn’t affect the patients very hard up to now. There was one pneumonia in the follow-up of one year. It’s a very important question, how to bring this innovative therapy to the patients, because we speak about very rare diseases in our cases but also in other neuromuscular diseases where we treat patients with this approach and it’s very expensive for the companies and also for the healthcare system when we do in every distinct indication and clinical trial. So that’s the discussion we have to make in the next years. Is the procedure to approve or is every indication to approve from the agency like FDA or EMA? And I think that’s very important to bring this treatment to the patients. As we know, there are individual cases and I think it’s very discriminating for the rare diseases for the patients with rare diseases if we make in every case an an individual trial, because I think that would not happen.
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