Five patients with acute lupus who received CAR-T were reported to be in remission according to a recent article in StatNews. Researchers are now encouraged by the prospect of treating a long list of autoimmune disorders.
The current study, led by Dr. Georg Schett and colleagues at Alexander University in Nuremberg, was published this week in Nature Medicine.
Dr. Schett and team’s study follows research they published over a year ago. In that case, the study involved one woman with acute lupus who was treated with CAR-T and reportedly went into remission.
The therapy was given a “compassionate use” designation. That allows the drug to be used to treat patients who are severely ill and have no other option, even though it had not been approved by the FDA.
About Lupus and CAR-T Therapy
Lupus is the most classical model of autoimmune disease where the immune system goes on the attack against the body’s organs and tissues. By definition, autoimmune disease is the immune system’s inability to differentiate between its own or foreign antigens.
T cells are engineered in CAR-T therapy to target specific disease cells. CAR-T treatment, one of the newest therapies, uses our own immune system to target a disease.
Immunosuppressive drugs currently in use may eliminate errant B cells from the blood but do not always penetrate lymph nodes or bone marrow. CAR-T on the other hand clears B cells from the body.
According to the CDC, approximately 200,000 individuals in the U.S. have been diagnosed with systemic lupus erythematosus (common lupus). Researchers have yet to determine the cause of the disease.
When lupus has control of various body systems, an army of immune system agents goes on the attack against a perceived pathogen. But instead, these agents are actually pursuing the body’s proteins.
Where lupus is involved, T cells target a protein called CD19 located on the surface of certain B cells that are associated with disease flareups.
Then B cells join the fight and the body manufactures antibodies that attack itself. The result is a vicious cycle of immune attacks leading to pain, organ damage, and fatigue as well as other symptoms.
Dr. Schett’s Reports from the Current Study
- A lupus patient who received CAR-T therapy eighteen months ago has had no recurring disease symptoms;
- Another patient has been in remission and has not received treatment for over one year.
Dr. Schett noted that the patient’s immune system remained stable in contrast to some CAR-T and various other cancer treatments. Nor were there any serious toxic effects or infections.
Other recent reports from Dr. Schett and his team on the five patients are:
- A third patient could not attend school because the disease caused swelling of her heart muscle. Within a short period of time, this young woman was back in school.
- A fourth patient was able to continue her love of horseback riding, and
- A fifth patient is now a DJ in a nightclub.
Dr. Schett told STAT that he is grateful to be able to help these young women return to normal life.
This may be only the beginning. Other diseases involving B cells such as multiple sclerosis or autoimmune conditions will fall in place. Yet CAR-T therapy is expensive. The cost is approximately $400,000. The FDA has only approved a few CAR-T products for specific cancers. Drug manufacturers are having difficulty keeping up with the demand. Similar issues are anticipated with respect to autoimmune diseases.
There is still a long road ahead. It involves studies with a larger number of participants and ensuring the safety and efficacy of CAR-T.
