Promising Lupus treatment needs more research, study

Dear Doctors: Our daughter, 24, struggled with her health for years before being diagnosed with lupus. I have seen a study with a potential cure called CAR T-cell therapy. How does it work? 

Answer: Lupus is an autoimmune disease. That means the immune system mistakenly identifies the body’s own tissues as foreign invaders and attacks them.

This results in damaging inflammation that, over time, leads to a wide range of symptoms. These can include fatigue, fever, stiffness or pain in the joints or muscles, skin problems, sensitivity to sunlight, headache, dry eyes, hair loss and mouth sores.

Lupus also can cause damage to organs, particularly the lungs and kidneys, and can affect the nervous system and heart. There’s no known cure. 

Treatment involves medications to reduce inflammation, manage symptoms and prevent organ damage.

Now, unlike traditional treatments, which can only ease the effects of the disease, new research on CAR T-cell therapy hints at a path to remission.

But the study was quite small, with just five patients. The long-term success of this approach and any long-term side effects aren’t known.

CAR T-cell therapy is a type of immunotherapy — treatments that harness the body’s immune cells to fight disease.

Five years ago, the federal Food and Drug Administration began to approve CAR T-cell therapy — which uses a type of white blood cell — for use in some blood cancers.

The first step in such a treatment is to collect blood from the patient and extract the T cells. These are then genetically modified so they will act on a specific target.

For the lupus study, researchers “taught” the CAR T cells to target the overactive immune cells that attack the body’s own tissues. Up to 100 million CAR T cells were needed for a single therapeutic dose, so the customized cells were reproduced in a lab. The patients were then infused with their own customized cells. 

About three months later, after the CAR T cells had eliminated the malfunctioning immune cells, the patients’ bodies began to produce new immune cells, which behaved normally.

Each of the five patients has been able to stop taking drugs to manage their illness, and they are considered to be in remission.

But the follow-up period so faris less than two years. That and the tiny sample size mean this approach is still in the realm of investigation. More study is needed for CAR T-cell therapy to be effectively evaluated as a possible treatment for lupus.

Dr. Eve Glazier and Dr. Elizabeth Ko are internists at UCLA Health.