- Half a dozen patients in Germany have been functionally cured of lupus, at least for now.
- His doctors used a blood engineering technique generally reserved for fighting aggressive cancers (CAR-T).
- It is unlikely that such a technique will be widespread among people with lupus, due to cost and the lab work required for each patient.
Doctors in Germany have found a way to effectively cure the most common form of lupus using a novel and expensive blood infusion technique usually reserved for treating cancer.
A group of immunologists based in Erlangen, Germany, announced in the journal Natural medicine Thursday the five systemic lupus erythematosus (SLE) treated with CAR-T cells during a compassionate use research trial were in remission three months after their treatment and remained so for at least eight more months.
Lead researcher Georg Schett told STAT that the results of the treatment were “miraculous” and the fact that such a course of therapy could be used so effectively to combat lupus “blew us away.”
“We were very surprised at how effective it was,” he said.
The finding, though still preliminary, suggests that the CAR-T approach could one day have a major impact on curing inflammatory autoimmune disease, if the solution is sustained in these patients long-term.
For now, the research team is still waiting to see if the six patients remain “basically healthy” without further treatment, and “if there really isn’t a relapse,” Schett told Insider.
Their latest work builds on an initial research letter that Schett and colleagues published last year in the New England Journal of Medicine, detailing how another woman in her 20s was functionally cured of lupus in the same way. You have been feeling well for at least 18 months.
In CAR-T, a patient’s blood is trained to fight disease
CAR-T for lupus is not without its drawbacks. They are expensive and labor intensive to produce, which means this strategy may never be a viable treatment option for everyone. 200,000 Americans living with the disease.
Still, “if you fail with conventional medications and have severe disease, I think this will be, in the future, the time to intervene with CAR-T cell therapy,” Schett explained to Insider, adding that the same strategy It could could be used to treat other serious autoimmune diseases, including multiple sclerosis.
Today, CAR-T immunotherapy is generally reserved for the treatment of aggressive cancers such as lymphomas and can cost around $450,000, according to the National Cancer Institute. Part of the reason the treatment is so expensive is because it is highly personalized, specific to each patient and her disease.
First, blood is drawn from the patient, then it is taken to a lab so that the T cells that stimulate the immune system are re-engineered with special proteins called chimeric antigen receptors (CARs). Finally, those protein-infused T cells are reinjected into the patient, where they work to kill a targeted disease (for lupus, T cells were designed to fight CD19).
Remarkably, the immune systems of these German patients remained robust during their CAR-T therapy, with no serious infections or toxic side effects reported. That’s different from what can happen in cancer patients receiving CAR-T, who may develop life-threatening neurological problems or cytokine storms.
“This would seem to be the holy grail of treatment,” Dr. Mark Leick, who was not involved in this lupus study but works on CAR-T therapies at Massachusetts General Hospital, told STAT.
Patients return to horseback riding, dancing and studying.
Schett said one of his young patients is riding horses again, another is DJing in clubs and a third has resumed his studies, activities that all had to largely abandon when they had lupus, as a result of chronic and debilitating fatigue.
For now, CAR-T remains an experimental, expensive and highly researched solution for only a lucky few who have lupus. The typical treatments recommended for the millions of other people with lupus around the world without access to these kinds of special blood engineering programs are still limited to steroids, anti-inflammatories like ibuprofen to relieve symptoms, or, in the most severe cases, expensive monoclonal antibodies.
But, “if you can really kill the disease with a single injection of CAR-T cells and have a remission for a long time, I think it’s not that expensive,” Schett said, explaining that in his noncommercial trial, doctors spent dozens thousands, not hundreds of thousands, treating each sick patient (they saved costs by manufacturing their CAR-T cells in-house).
“He is a young person who has his whole life ahead of him,” he said. “If you can get rid of a disease when you’re 20 and you don’t get it again, well, then it can cost a little bit and still be cheap in the long run.”