Huji Xu’s team was on tenterhooks after delivering the first treatments. “We couldn’t sleep, because all these cases are very sick patients,” says Xu, a rheumatologist at the Naval Medical University in Shanghai, China, who published the first results of a revolutionary cellular therapy for autoimmune diseases in September (X. Wang et al. Cell 187, 4890–4904; 2024).
Two weeks after receiving engineered immune cells, the first patient — a woman with a debilitating disorder characterized by extreme muscle weakness — told nurses that she had regained enough strength to lift her arms and comb her hair. Two other recipients, both men, with a different condition, said that their symptoms began fading within days. More than six months later, all three recipients were in remission, according to Xu. “We are a little bit more relaxed” now, he says.
The engineered cells are known as chimeric antigen receptor (CAR) T cells and have been designed to hunt down and eliminate B cells, a type of immune cell that sometimes runs amok in people with autoimmune disorders. CAR-T-cell therapy is widely used to treat blood cancers involving malignant B cells, but it has also shown some promise for autoimmune diseases.
Last year, teams in Germany revealed that they had used CAR T cells to treat at least 15 people with several autoimmune conditions, with stunning success. Xu’s trial differs from these because it used cells taken from an independent donor, whereas the German teams used cells taken from the person being treated. If successful, the donor strategy could allow for mass production of CAR-T-cell treatments, reducing their costs and extending their reach.
Xu trained as a medical doctor in Shanghai. In 1990, he moved to Adelaide, Australia, to start a PhD in immunology and rheumatology, looking at the role of a specific antibody in rheumatic diseases — inflammatory conditions that affect the joints, muscles and bones. Xu went on to research a broad range of subjects, from the biological mechanism of lupus and several types of arthritis, to sudden infant death syndrome (SIDS) and malaria vaccines.
In 2008, he returned to Shanghai and established a large clinical and research centre for rheumatology. The CAR-T-cell trial was a good fit, says Xu, because of his interest in the underlying causes of rheumatic disease. The woman his team treated had refractory inflammatory myopathy. The men had a type of systemic sclerosis that causes the skin to harden and affects many organs.
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