Pharma players are also targeting other diseases with cancer therapy

Now that CAR-T cell therapy is working wonders for some cancer patients, research is underway to determine whether it can also help with immune system disorders such as lupus and multiple sclerosis. ‘Some results are remarkable and hopeful.’

For some cancer patients for whom traditional treatments no longer work, CAR-T cell therapy can make the difference between life and death. In personalized treatment, immune cells are taken from the patient’s body to genetically manipulate them. The T cells are grown in a laboratory and then reintroduced into the patient.

What is less known is that CAR-T therapy is also being extensively tested as an experimental treatment for autoimmune disorders, a collective name for diseases in which antibodies turn against the body and cause damage. Think of rheumatoid arthritis, multiple sclerosis or the inflammatory disease lupus.

“The fact that CAR-T therapy is also being investigated for such conditions is a recent evolution,” says immunologist and pulmonologist Bart Lambrecht, affiliated with Ghent University Hospital and the Flemish Institute for Biotechnology. ‘Since 2019, you have also seen many clinical studies in autoimmune disorders. In oncology, a lot of expertise and capacity has been built up around CAR-T on which researchers can build further.’

Moreover, there are similarities between cancer and disorders of the immune system. In both cases, runaway B cells cause damage. ‘You have to imagine those B cells as factories that produce antibodies to kill invaders, such as viruses,’ says Lambrecht. ‘But such antibodies can also target the body. The big difference is that those B cells proliferate in cancer, while they occur in much lower numbers in autoimmune disorders.’

‘For cancer, CAR-T therapy must be administered more powerfully and for longer. This is different with autoimmune disorders. There you have to kill the ‘bad’ B cells to reset your immune system. Those newly created B cells no longer cause damage,’ says Lambrecht.

Several biotech and pharmaceutical companies, including Novartis and the American Bristol-Myers Squibb, are conducting early research into CAR-T therapy as an experimental treatment for the inflammatory disease lupus. ‘Lupus is a very serious disease that causes, among other things, joint pain and severe fatigue. There is medication, but it doesn’t work very well,” says Lambrecht.

Due to the busy field and the fact that other companies are further along, Galapagos recently decided to discontinue its CAR-T candidate for lupus, for which it had yet to start the first clinical trial. The biotech company says it will continue to focus on early CAR-T research in immunology.

“Last month, a remarkable German study on an experimental CAR-T therapy for lupus was published in the New England Journal of Medicine,” says Lambrecht. ‘A group of 15 patients with a persistent form of lupus and other autoimmune disorders appeared to be in remission after a single therapy (where the disease no longer appears active, ed.). Some still did not need any medication even after two years. That is remarkable and hopeful.’

A broader overview in the journal Nature shows that the number of clinical trials of experimental CAR-T therapies for autoimmune diseases has quadrupled since 2021, to forty this year. More than half of those studies revolve around lupus. But there are also studies that focus on the rare muscle disease myasthenia gravis and multiple sclerosis.