First treatment utilizing donor cells induces remission in autoimmune diseases

Novel Therapy Using Donor Cells Induces Remission in Autoimmune Disorders

First treatment utilizing donor cells induces remission in autoimmune diseases
Following treatment with bioengineered and CRISPR-modified immune cells, two men and one woman suffering from severe autoimmune diseases have achieved remission. These three patients from China are the first to receive such therapy utilizing modified immune cells derived from donors rather than autologous cells. This milestone represents a significant step toward the mass production of these innovative treatments.

Among the recipients is Mr. Gong, a 57-year-old resident of Shanghai. He is afflicted with systemic sclerosis, a condition that damages connective tissue and can lead to organ impairment and skin hardening. Just three days into the treatment, he reported being able to move his fingers and open his mouth again, along with feeling a relaxation in his skin. Within two weeks, he returned to his job. More than a year after the therapy, he noted, "I feel very good."

In the United States, about six products featuring engineered immune cells, known as chimeric antigen receptor-T (CAR-T) cells, have received approval. These cells have demonstrated considerable potential in treating blood cancers and may also be effective against autoimmune conditions such as multiple sclerosis and lupus, which involve rogue immune cells producing autoantibodies that attack the body’s tissues. However, the personalized nature of the therapy, which typically relies on an individual’s own immune cells, results in significant costs and lengthy preparation times.

The trial represents the first publication of findings related to autoimmune disorders and is led by Xu Huji, a rheumatologist affiliated with Shanghai's Naval Medical University. The results were shared last month in the journal Cell. After their treatment, the participants remained in remission for nearly six months. Xu reported that an additional two dozen individuals have undergone the donor-derived therapy, which includes a slightly modified product, with most responses being positive.

"The clinical outcomes are phenomenal," says Lin Xin, an immunologist at Tsinghua University who is conducting a separate trial with donor-derived CAR-T cells for lupus.

Typically, T cells used in CAR-T-cell therapy are collected from the patient receiving the treatment. Following enhancement with CAR proteins that specifically target B cells, these immune cells are reintroduced into the patient’s body.

Upon injection, the CAR-T cells activated, proliferated, and successfully targeted and eliminated all B cells, including those associated with autoimmune diseases. The bioengineered T cells remained in the recipients for several weeks before primarily disappearing. Eventually, new, healthy B cells began to regenerate, while no pathogenic ones re-emerged. A similar reaction has been observed in autoimmune patients treated with CAR-T cells derived from their own cells.

According to Nature, a generic CAR-T product, if proven successful, could allow pharmaceutical companies to enhance production capacity significantly, leading to lower costs and reduced production timelines while catering to a larger patient population. However, it remains uncertain whether patients treated with donor-derived CAR-T cells will encounter additional risks.

Navid Kalantari for TROIB News