6 lupus patients effectively cured by a cancer therapy, and doctors say they are 'blown away'

• A half dozen patients in Germany have been functionally cured of lupus — for now, at least.
• Their doctors used a blood-engineering technique that's usually reserved for fighting aggressive cancers (CAR-T).

Doctors in Germany have found a way to effectively cure the most common form of lupus using a novel and pricey blood-infusion technique typically reserved for treating cancer.

A group of immunologists based in Erlangen, Germany announced in the journal Nature Medicine on Thursday that all five systemic lupus erythematosus (SLE) patients who they treated with CAR-T cells during an investigational, compassionate use trial were in remission by three months after their treatment, and remained so for at least eight months longer.

Lead researcher Georg Schett told STAT that the treatment outcomes were "miraculous," and the fact that such a therapy course could be used so effectively to combat lupus "blew us away."

"We were really surprised how effective it was," he said.

The find, though still preliminary, suggests that the CAR-T method could one day have a major impact curing the inflammatory, autoimmune disease — if the fix holds in these patients long-term.

For now, the research team is still waiting to see whether the six patients remain "basically healthy" without any more treatments, and "whether there's really no relapse," Schett told Insider.

His latest work builds on an initial research letter that Schett and his colleagues published last year in the New England Journal of Medicine, detailing how one other 20-year-old woman was functionally cured of lupus in the same way. She's been feeling good for at least 18 months.

In CAR-T, a patient's blood is trained to fight disease

CAR-T for lupus isn't without drawbacks. It's both pricey and labor-intensive to produce, meaning this strategy will likely never be a viable treatment option for all 200,000 Americans living with the disease.

Still, "if you fail on the conventional drugs, and you 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 could potentially be used to treat other severe autoimmune diseases, including multiple sclerosis.

Today, CAR-T immunotherapy is usually reserved for treating aggressive cancers like 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 bespoke, specific to each patient and their disease.

First, blood is extracted from the patient, then it is taken to a lab to have the immune-boosting T cells inside re-engineered with special proteins called chimeric antigen receptors (CARs). Finally, those protein-infused T cells are re-injected into the patient, where they work to kill a target disease (for lupus, the T cells were engineered to fight back against CD19.)

Remarkably, these German patients' immune systems remained robust during their CAR-T therapy, with no serious infections or toxic side effects reported. That's unlike what can happen in cancer patients given CAR-T, who may develop neurological issues, or life-threatening cytokine storms.

"This would seem to be the holy grail of treatment," Dr. Mark Leick, who was not involved in this lupus study, but who works on CAR-T therapies at Massachusetts General Hospital, told STAT.

The patients are back to riding horses, dancing, and studying

Schett said one of his young patients is riding horses again, another is DJing in clubs, and a third has resumed her studies — activities they all had to largely forgo when they had lupus, as a result of chronic, debilitating fatigue.

For now, CAR-T remains a highly investigational, pricey, experimental fix for just a lucky few lupus sufferers. The typical recommended treatments 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 ease symptoms, or, in the most serious cases, expensive monoclonal antibodies.

But, "if you can really wipe out the disease with a single shot of CAR-T cells and have remission for a long time, I think it's not that expensive," Schett said, explaining that in his non-commercial trial doctors spent tens of thousands, not hundreds of thousands, treating every sick patient (they saved on costs by manufacturing their CAR-T cells in house.)

"It's a young person having their whole life in front of them," he said. "If you can wipe out a disease when you're 20 and you don't get it again, well, then it can cost a little bit and still be economical over the long run."