First successful case of CAR-T therapy against lupus: "It opens a possibility of long-term cure for patients"

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Last week the 'father' of CAR-T cell therapy, the immunologist

, declared to this newspaper his confidence that this treatment, until now used for certain hematological cancers, also works in non-oncological diseases, such as multiple sclerosis. multiple or lupus.

Science corroborates this today with the publication in

of new data from a study carried out in Germany that shows remission of systemic lupus erythematosus (SLE) in five patients.

CAR-T therapy is built from the patient's own T lymphocytes;

once extracted, they are modified, inserting a molecule (chimeric antigen receptor, CAR) so that they eliminate B lymphocytes, targeting certain proteins on their surface.

With this strategy, he has managed

in patients with certain types of leukemia, lymphoma and multiple myeloma in which B cells are involved.

Given the success achieved, with very striking survival rates in some cases, it was a matter of time trying to

where B lymphocytes also act. This is the case of SLE, a

that, according to data from the Spanish Society of Rheumatology

.

This disease, of unknown cause, can produce outbreaks, more or less mild, that cause inflammation, damage to joints, muscles and other organs such as skin, kidneys, heart and lungs, among others.

Current treatments can achieve prolonged remissions in patients, but for some

.

This was the case with the young Thu-Thao V., one of the patients recruited in this pilot study whose latest data is presented today, who was diagnosed with lupus in 2017 at the Erlangen University Hospital (Germany).

The student has reported that in the worst moments of the disease she had to take almost 20 pills a day to deal with joint pain, fluid retention due to kidney failure, as well as intense palpitations and hair loss.

First patient with autoimmune disease successfully treated with CAR-T

After trying various immunosuppressive therapies without success, doctors turned to CAR-T cell immunotherapy.

Thu-Thao has become the first patient with a rheumatological disease to receive the CAR-T treatment.

Six months after the infusion of the therapy, when the hospital reported the first results in a letter in

, the young woman assured that she had returned to playing sports.

Today,

.

The patient is one of five (four of them women, with a mean age of 22 years) who have received CAR-T therapy.

In addition to not responding to conventional treatments, these patients had severe lupus lupus, which especially caused kidney damage.

After the infusion of the cells, the

has confirmed that all of them experienced an improvement in their symptoms, including the remission of the involvement of internal organs, as well as the disappearance of the autoantibodies related to the disease, without the need to continue receiving conventional therapies.

'Chance of long-term cure'

The internal medicine and rheumatologist

, director of the Department of Internal Medicine 3-Rheumatology and Immunology at the Erlangen Hospital, and one of the main researchers of the study, highlights to this medium that "the patients not only rapidly improved their symptoms, but also who

SLE-associated autoimmunity.

Professor Schett is

about the potential application of this therapy.

"We expect it to have substantial implications for patients with severe forms of the disease. A single infusion of CAR-T cells abolished the disease for more than a year, and the patients did not require any type of immunosuppressive therapy, not even glucocorticoids. Therefore, If patients remain in remission without treatment, this therapy could open up a long-term cure for patients," he writes in an email.

With these results, he believes that the therapy could benefit "severe patients with organ involvement, especially those who have failed conventional treatments."

There are several CAR-T therapies approved by regulatory agencies to treat hematologic diseases.

However, the German team chose to develop

, similar to those already on the market, explains Schett.

Common

associated with haematological cancer treatment, such as the so-called cytokine release syndrome (a systemic inflammatory response), were mild in lupus patients (with symptoms such as fever).

This is probably due, the researchers argue in their paper, because "

in patients with SLE than in patients with active B-cell neoplasms."

Neither neurotoxicity nor treatment-associated infections were observed.

A therapy with a great future ahead

CAR-T therapy has only just made an appearance in autoimmune diseases, but Schett is clear that this is only the beginning, since the studies that are already being designed in this field "will provide more information on the safety and efficacy of the treatment, and will the way for the possible approval of this treatment for SLE and other autoimmune diseases".

Specifically, if all of these safety and efficacy steps are confirmed, "we hope that patients with autoimmune diseases such as autoimmune myositis or multiple sclerosis may also benefit from CAR-T cell therapy."

Specialists from the Spanish Society of Rheumatology (SER) consulted to assess the impact of this research in clinical practice are cautious, given the

.

The rheumatologist

, spokesman for the SER and specialist at the Ramón y Cajal University Hospital in Madrid, comments on the research that "the experience reported in this article should move us to

,

and, of course ,

. The results are excellent, but the length of follow-up and the number of patients is limited."

For his part,

, spokesperson for the SER and specialist at the University Hospital Complex of Vigo, recalls that "current treatments for SLE can lead the patient to remission, which can be prolonged, even maintained for several years" .

And he clarifies that, however,

, in which the patient is free to a greater or lesser extent of the symptoms and of any clinical manifestation of lupus; you can also have normal tests, even in patients treated only with antimalarials. That's a clinical remission, but not a cure."

In statements to SMC,

, Professor of Immunology at the University of Navarra, CIMA researcher and co-director of the Department of Immunology and Immunotherapy at the University Clinic of Navarra, also indicates that the study of this therapy must be extended to more patients, "but

in terms of risk-benefit and in these five cases it appears to be effective."

The immunologist adds that "the data is solid in the sense that this B-lymphocyte elimination time is active against severe cases of systemic lupus erythematosus with poor response to conventional therapies. This is pioneering work that opens a path for adoptive cellular immunotherapy in autoimmune diseases".

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