Results from a German CAR-T study, shared Saturday, suggest that the therapy can lead to significant improvements or even remission for patients suffering from lupus, myositis and other autoimmune diseases.
The results, announced at the American Society of Hematology (ASH) annual meeting, are still preliminary, but very positive. Although the study was small – consisting of only 15 patients – after a median follow-up time of 15 months all patients’ conditions had improved and none had required any other treatments.
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Eight of the patients had lupus (also known as systemic lupus erythematosus), four had systemic sclerosis and three had inflammatory myositis, all non-responsive to standard treatments, with many “so weak that they could not walk farther than a few meters.”
The study used chimeric antigen receptor T (CAR-T) cells in a therapy currently being tested for blood cancers, but applied them to severe b-cell autoimmune conditions. Since both sets of conditions are caused by defective blood cells, the theory goes, a tweak in the way the T cells are altered should allow similar results to those seen in blood cancers.
Anecdotally, this does appear to be the case. Leader author of the study Dr. Fabian Müller told the room, “Our first lupus patient was 21 years old and she was so sick that she had a life expectancy of 4-6 weeks; now, she is out jogging five days a week.”
Adverse events were widespread but mild: ten of the patients experienced mild cytokine-release syndrome, one moderate and another experienced vertigo. These compare favourably to autologous stem cell transplant, which has high efficacy in treating non-responsive autoimmune diseases but also carries a high risk of adverse events, including autoimmune disease.
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By GlobalDataCalifornian Immix Biopharma is currently developing a CAR-T therapy that targets B-cells, and whilst the treatment used by this study was unnamed, the mechanism is much the same. Its candidate NXC-201 is currently in Phase I/II trials for amyloidosis and multiple myeloma but it also plans to extend trials to include autoimmune indications including lupus.
There is increasing interest in gene and cell therapies – the category to which CAR-T therapies belong – but GlobalData research suggests that it is being talked about more by small biopharma companies than global hegemons like Pfizer or Roche.
GlobalData is the parent company of Pharmaceutical Technology.
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