CAR-T therapy could treat severe autoimmune diseases, shows study

For the first time, a CD19 CAR-T cell therapy has shown high efficiency in managing three different severe autoimmune diseases.

The study, conducted on one female patient, demonstrated achievement of remission, with no further treatment required a year on after being given the cell therapy.

These results offer significant potential in this indication because the patient previously required daily blood infusions to manage their severe autoimmune haemolytic anaemia (AIHA), immune thrombocytopenia (ITP) and antiphospholipid antibody syndrome.

They had already had nine different lines of therapy, including antibody treatments, steroids, and immune-suppressing medications. These did not provide long-term relief.

However, three weeks post-treatment with the CAR-T cell therapy, the patient’s haemoglobin levels doubled and normalised and their platelet counts stabilised. Additionally, their antiphospholipid antibodies, associated with life-threatening blood clots, reduced to negative levels, which was maintained.

Results also indicate the CAR-T therapy reset the patient’s immune system, evidenced by a majority of naive B cells.

After more than ten years of illness, the patient’s blood counts normalised within just a few weeks. The speed and depth of the response was remarkable”

Corresponding author, Fabian Müller said: “After more than ten years of illness, the patient’s blood counts normalised within just a few weeks. The speed and depth of the response was remarkable.

“We believe that using CAR-T therapy earlier for patients with severe autoimmune disease could help prevent complications from years of ineffective treatments.”

Müller explained that the therapy likely had a high efficacy because the CAR-T cells could penetrate different tissues and eliminate the dysregulated cells, both in mature and developing states.

The findings were published in Cell.

Another recent advanced therapy win likewise also showed promise in enabling a functional cure for a severe blood disorder.

While the patient population was larger than in the CAR T study, most patients given reni-cel also saw a rapid improvement in key blood cells, with levels recovering within a month post-treatment. The phase I/II data for Editas Medicine’s renizgamglogene autogedtemcel (reni-cel) were presented in the NEJM.