Hospital sees success in novel therapy for patients with autoimmune diseases

Patients with serious autoimmune diseases are seeing new hope at Shanghai's Changzheng Hospital, where doctors have adopted a novel CAR-T cell immunotherapy by using genetically engineered, healthy-donor-derived cells.

Currently, most CAR-T therapy, or chimeric antigen receptor T cell therapy, is specifically developed for each individual patient by reprogramming the patient's own immune cells to target their diseases, mainly cancer. Because the autologous cells need an individualized process for each patient, the process is long and the cost is very high. Moreover, many patients' own immune cells have deficiencies.

So the team of Dr Xu Huji from Changzheng Hospital decided to try T cells from healthy donors.

"The allogeneic cells hold great promise for expanding the accessibility of CAR-T therapy, due to its low cost, batch processing, and good source of immune cells," Xu said on Tuesday.

Researchers genetically engineered healthy-donor-derived, targeted CAR-T cells using genetic editing technology to address the issue of immune rejection and treat patients' diseases. So far, the first batch of three patients, one with refractory immune-mediated necrotizing myopathy and two with systemic sclerosis, have been infused with these cells and all reported very positive results.

"We see deep remission in all these patients, whose symptoms and data all have greatly improved," Xu said. So far, 12 patients with serious autoimmune diseases such as myopathy, sclerosis, and lupus have received the treatment and all have shown good responses. Some patients have even stopped routine medications, experts said.

The cost of autologous CAR-T therapy is about 1 million yuan (US$137,342), while allogeneic therapy only costs some 100,000 yuan as one donor's cells can be made into 200 dosages.

Dr Xu Huji from Changzheng Hospital

"Our research is the world's first report on using donor's T cells to develop allogeneic CAR-T therapy to treatment autoimmune diseases, as our results demonstrate the high safety and promising immune modulatory effect of the off-the-shelf CAR-T cells in treating severe refractory autoimmune diseases," Xu said. "It provides a new solution and thought to the clinical practice and will offer positive effects on medical decisions, treatment processes, and medical costs for such patients.

"The cost of autologous CAR-T therapy is about 1 million yuan (US$137,342), while allogeneic therapy only costs some 100,000 yuan as one donor's cells can be made into 200 dosages. Currently, there are some 20 off-the-shelf CAR-T products in clinical research and ours is the first one to release its effects, drawing attention from both home and abroad."

Over 8 percent of the population suffer autoimmune diseases, which result in high disability, influence life quality, and impose severe financial burdens. It is the world's third-highest killer, following cerebro-cardiovascular disease and cancer. Among the patients, 30 percent suffer refractory conditions, facing poor treatment effects, and a high risk of relapse risk.

The research was published by world-leading journal Cell.

Patients who want to consult or participate in the clinical trial can contact Dr Wang Xiaobing through gale820907@163.com.