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lupus
This technology has allowed advancements in target therapies such as CART-cell therapy, which has shown promising results when used for cancer treatments. Last year we saw the FDA approval for Carvykti, the second therapy treatment approved for the later stages of blood cancer. We’ve also seen recent success in personalised therapies for diseases such as <anchor>lupus</anchor>, showing a promising and wider use of CAR-T cell therapy.
CAR T Cell Therapy Reaches Beyond Cancer
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# CAR T Cell Therapy Reaches Beyond Cancer

## Penn Medicine researchers laud early results for CAR T therapy in lupus patients, point to broader horizon for the use of personalized cellular therapies

## November 23, 2022

![Carl June and Daniel Baker](/news/-/media/images/pr news/news/2022/november/carl june_daniel baker teaser.ashx?h=263&w=400&la=en)

Carl June, MD, and Daniel Baker

PHILADELPHIA— Engineered immune cells, known as CAR T cells, have shown the world what personalized immunotherapies can do to fight blood cancers. Now, investigators have reported highly promising early results for CAR T therapy in a small set of patients with the autoimmune disease lupus. Penn Medicine CAR T pioneer Carl June, MD, and Daniel Baker, a doctoral student in Cell and Molecular Biology in the Perelman School of Medicine at the University of Pennsylvania, discuss this development in a commentary published today in Cell01372-1).

“We’ve always known that in principle, CAR T therapies could have broad applications, and it’s very encouraging to see early evidence that this promise is now being realized,” said June, who is the Richard W. Vague Professor in Immunotherapy in the department of Pathology and Laboratory Medicine at Penn Medicine and director of the Center for Cellular Immunotherapies at Penn’s Abramson Cancer Center.

T cells are among the immune system’s most powerful weapons. They can bind to, and kill, other cells they recognize as valid targets, including virus-infected cells. CAR T cells are T cells that have been redirected, through genetic engineering, to efficiently kill specifically defined cell types.

CAR T therapies are created out of each patient’s own cells — collected from the patient’s blood, and then engineered and multiplied in the lab before being re-infused into the patient as a “living drug.” The first CAR T therapy, Kymriah, was developed by June and his team at Penn Medicine, and received Food & Drug Administration approval in 2017. There are now six FDA-approved CAR T cell therapies in the United States, for six different cancers. The therapies have revolutionized the treatment of certain B cell leukemias, lymphomas, and other blood cancers, putting many patients who otherwise had little hope into long-term remission.

From the start of CAR T research, experts believed that T cells could be engineered to fight many conditions other than B cell cancers. Dozens of research teams around the world, including teams at Penn Medicine and biotech spinoffs who are working to develop effective treatments from Penn-developed personalized cellular therapy constructs, are examining these potential new applications. The commentary from June and Baker comes in response to the first significant clinical report of success from these efforts: a paper in Nature Medicine from German researchers on the use of CAR T therapy against the autoimmune disease lupus (systemic lupus erythematosus).

Researchers say lupus is an obvious choice for CAR T therapy because it too is driven by B cells, and thus experimental CAR T therapies against it can employ existing anti-B-cell designs. B cells are the immune system’s antibody-producing cells, and, in lupus, B cells arise that attack the patient’s own organs and tissues.

In the German study, the patients — five young adults — did not benefit from standard lupus treatments. Yet, all went into remission, and were able to stop taking their lupus drugs within three months of a single, relatively small dose of CAR T therapy, which essentially removed their existing B cells. (Cancer patients subsequently require infusions of purified human antibodies from healthy volunteers, to maintain some antibody immunity.) Even more remarkably, all the patients remained in remission during the follow-up period of up to a year, and unlike cancer patients, the lupus patients experienced the return of their B cells, which are naturally replenished from blood stem cells in bone marrow.

Baker and June note in their commentary that, although the German study’s results need to be confirmed with larger studies and longer-term follow-up, they are highly promising — indeed, they suggest that lupus could turn out to be an easier CAR T target than B-cell cancers.

“Disease-driving B cells are much less numerous in lupus,” Baker said. “Thus, effective CAR T treatment of this autoimmune disease may require a much lower dose that greatly reduces the problem of immunological side-effects.”

##### Topic:

Penn Medicine is one of the world’s leading academic medical centers, dedicated to the related missions of medical education, biomedical research, excellence in patient care, and community service. The organization consists of the University of Pennsylvania Health System and Penn’s Raymond and Ruth Perelman School of Medicine , founded in 1765 as the nation’s first medical school.

The Perelman School of Medicine is consistently among the nation's top recipients of funding from the National Institutes of Health, with $550 million awarded in the 2022 fiscal year. Home to a proud history of “firsts” in medicine, Penn Medicine teams have pioneered discoveries and innovations that have shaped modern medicine, including recent breakthroughs such as CAR T cell therapy for cancer and the mRNA technology used in COVID-19 vaccines.

The University of Pennsylvania Health System’s patient care facilities stretch from the Susquehanna River in Pennsylvania to the New Jersey shore. These include the Hospital of the University of Pennsylvania, Penn Presbyterian Medical Center, Chester County Hospital, Lancaster General Health, Penn Medicine Princeton Health, and Pennsylvania Hospital—the nation’s first hospital, founded in 1751. Additional facilities and enterprises include Good Shepherd Penn Partners, Penn Medicine at Home, Lancaster Behavioral Health Hospital, and Princeton House Behavioral Health, among others.

Penn Medicine is an $11.1 billion enterprise powered by more than 49,000 talented faculty and staff.

## Contacts

Meagan Raeke

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