Crescent Discovery Series

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One CAR-T treatment worked on three autoimmune diseases

Lupus, systemic sclerosis and myositis are normally treated as separate diseases, each with its own long-term drug regimen.

The Crescent Team17 April, 12:55 PMMüller et al., NEJM, 2024 · University Hospital Erlangen2 min read

Researchers at University Hospital Erlangen gave a single infusion of CD19 CAR-T cells, engineered immune cells originally developed for blood cancer, to 15 patients split across all three conditions, then tracked each patient using the specific response criteria for their disease.

Every one of the 15 patients responded. All eight people with lupus reached remission by standard criteria, all three with myositis met the major clinical response threshold for that disease, and all four with systemic sclerosis showed significant improvement on their disease's activity index, at a median follow-up of 15 months.

The treatment works by depleting the antibody-producing B cells thought to drive all three diseases, then letting the immune system regenerate without the faulty antibodies that caused the original damage. The group was small and came from a single center, and larger multi-site trials are now underway to see whether the response holds at scale and how long remission actually lasts.

Crescent Discovery Series cover: One CAR-T treatment worked on three autoimmune diseases

One reset, three different diseases, each in remission.

Source

  1. Müller, F., Taubmann, J., Bucci, L., Wilhelm, A., Bergmann, C., Völkl, S., Aigner, M., Rothe, T., Minopoulou, I., Tur, C., Knitza, J., Kharboutli, S., Kretschmann, S., Vasova, I., Spoerl, S., Reimann, H., Munoz, L., Gerlach, R. G., Schäfer, S., . . . Schett, G. (2024). CD19 CAR T-Cell Therapy in Autoimmune Disease — A Case Series with Follow-up. New England Journal of Medicine, 390(8), 687–700. https://doi.org/10.1056/NEJMoa2308917
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