Chinese study finds new CAR-T therapy may be safe, low-cost option

Three patients with autoimmune diseases successfully treated with general purpose version of a therapy used to treat blood cancers

Science

Dannie Pengin Beijing

Published: 3:00pm, 18 Jul 2024

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Chinese researchers have developed a new version of a cell therapy used to treat blood cancers, and they say it has potential as a versatile and much cheaper option than those currently being used.

The treatment, known as CAR-T, is a type of immunotherapy that has taken off in recent years and has also shown promise in treating other conditions such as asthma.

But the cost of the therapy is prohibitive. After it was first approved in the United States in 2017, commercial CAR-T therapies now cost between US$370,000 and US$530,000, not including hospital fees and drugs to treat side effects, according to a March paper in Nature.

CAR-T, or chimeric antigen receptor T-cell therapy, works by genetically engineering a patient’s own T-cells – a type of white blood cell crucial for fighting infections – to recognise and destroy enemies such as cancer cells.

The treatment is highly specialised and personalised, which is why it remains so expensive. It involves extracting a patient’s T-cells, genetically modifying them and then returning them to the patient’s body.

The new version developed by a team of researchers in Shanghai works a little differently. Called TyU19, it uses T-cells from healthy donors then genetically modifies them. The team said it was a universal medicine, so a batch could be used to treat many patients, greatly reducing the cost.

It was developed by scientists from biotech company BRL Medicine, East China Normal University and Shanghai Changzheng Hospital. The results of their study were published in peer-reviewed journal Cell on Tuesday.

According to a statement from BRL Medicine, the “most outstanding advantage” of TyU19 is that it can be manufactured on a large scale, while being convenient, safe, low-cost and with good efficacy.

It said the treatment could also overcome the “pain points” of existing, personalised CAR-T therapies, including a long preparation cycle, high risk of failure and extremely high cost.

For the study, the cell therapy was used to treat three patients with autoimmune diseases – one with refractory immune-mediated necrotising myopathy (IMNM) and two patients with diffuse cutaneous systemic sclerosis (SSc). The scientists said it was the first time autoimmune diseases had been successfully treated using a general purpose CAR-T therapy.

During a follow-up six months after they were treated, all three patients were said to be in “deep remission” with no serious adverse events observed.

IMNM and SSc are both rheumatic immune diseases – a group of diseases that affects more than 8 per cent of the world’s population. IMNM is a rare and severe disease characterised by muscle fibre necrosis and progressive muscle weakness, while SSc is characterised by extensive organ fibrosis.

George Gao Fu, a leading Chinese virologist and immunologist, said the new treatment was promising.

In a post on Chinese social media account “BioArt” on Tuesday, Gao noted that the sample size of the study was small. He said they were high-risk and difficult-to-treat cases but the fibrosis had been reversed for some of them.

“These results show the potential of this therapy, which deserves to be further validated in larger clinical trials in the future,” said Gao, former head of the Chinese Centre for Disease Control and Prevention.