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China leads global trials for in vivo CAR-T therapies with 82% of pipeline

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China leads global trials for in vivo CAR-T therapies with 82% of pipeline

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China has become the global leader in developing and trialling in vivo CAR-T cell therapies, accounting for 82% of the 140 drugs tested in animals and humans worldwide. The country's large population, fast-track regulatory pathways, and government support have driven this dominance. Early trials show promising results, with the US FDA approving the first China-developed in vivo CAR-T therapy for clinical testing this month.

Key Facts

  • Of the 140 in vivo CAR-T cell drugs being tested globally, 82% are developed by groups in China.
  • The first in-human trial of in vivo CAR-T therapy for autoimmune disease reported improved symptoms in five people with treatment-resistant lupus after three months.
  • Earlier this month, researchers in China reported improved symptoms in 16 people with autoimmune diseases after in vivo CAR-T therapy.
  • The US FDA approved the first China-developed in vivo CAR-T therapy for blood cancers for clinical testing this month.

China's Dominance in In Vivo CAR-T

China has become a hotspot for developing and trialling innovative therapies that could transform the treatment of cancer and autoimmune diseases. The combination of the country's large population, fast-track regulatory pathways and resources have helped it to dominate therapies that produce disease-fighting immune cells inside the body, say researchers. In vivo CAR-T-cell therapies instruct the body to alter immune cells so that they attack cancerous cells or, in the case of autoimmune diseases, wipe out cells that attack healthy tissue. In vivo therapies are appealing because they are cheaper and easier to develop than ex vivo CAR-T-cell therapies, in which immune cells are collected from a person's blood, edited in a laboratory and then reintroduced to the body.

Government Support and Early Results

China's progress in developing these therapies has been building for at least the past five years, says Emily Blyth, a physician and cellular therapy researcher at The Westmead Institute for Medical Research in Sydney, Australia. There is a huge volume of work, a lot of it supported by the Chinese government, Blyth says. Early trial results seem promising: the first in-human trial of in vivo CAR-T-cell therapy for an autoimmune disease reported results in September last year. Five people with treatment-resistant lupus showed improved symptoms just three months after treatment, with no serious side effects. Earlier this month, researchers in China reported that symptoms for 16 people with autoimmune diseases improved after in vivo CAR-T-cell therapy.

Global Impact and Future Outlook

The benefits will almost certainly extend beyond China, as this month the US Food and Drug Administration approved the first China-developed in vivo CAR-T-cell therapy for testing in clinical trials. The therapy is for blood cancers. Successful clinical development in China could also accelerate innovation, reduce treatment costs and generate valuable clinical evidence that benefits patients worldwide, says Xiaoyuan Chen, a chemist at Shandong Cancer Hospital and Institute in Jinan City, China, who is developing an in vivo CAR-T-cell therapy for cancer and fibrosis. But researchers caution that it's still early days for such therapies.

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