“I think we are living in a very exciting time for patients with severe autoimmune diseases,” Dr. Schett said.
Looking Forward
More than ten different B cell depleting CAR T cell therapies are slated for clinical trials in patients with SLE. These trials have either already started or should begin soon.2 Also, trials should start soon in patients with systemic sclerosis and myositis. Theoretically, this treatment approach may also be effective in other rheumatic diseases, such as rheumatoid arthritis.6
Interestingly, Dr. Schett’s patients have all been young (age 18–33), but trials such as Novartis’ SLE CAR T cell trial are planning to enroll a broader range of patients (18–65). This approach may affect treatment response or the risk of toxicities.2
Even if scientists eventually demonstrate the safety and efficacy of these therapies in large clinical trials, the implementation hurdles will remain high. Current FDA-approved CAR T cell therapies for blood cancers cost $300,000–500,000, so many patients will face major access challenges.2
Ruth Jessen Hickman, MD, is a graduate of the Indiana University School of Medicine. She is a freelance medical and science writer living in Bloomington, Ind.
References
- Mackensen A, Müller F, Mougiakakos D, et al. Anti-CD19 CAR T cell therapy for refractory systemic lupus erythematosus. Nat Med. 2022 Oct;28(10):2124–2132.
- Mullard A. CAR T cell therapies raise hopes—and questions—for lupus and autoimmune disease. Nat Rev Drug Discov. 2023 Nov;22(11):859–861.
- Taubmann J, Müller F, Boeltz S. Long term safety and efficacy of CAR-T cell treatment in refractory systemic lupus erythematosus—data from the first seven patients. Ann Rheum Dis. 2023;82(S1):93–94
- Bergmann C, Müller F, Distler JHW, et al. Treatment of a patient with severe systemic sclerosis (SSc) using CD19-targeted CAR T cells. Ann Rheum Dis. 2023 Aug;82(8):1117–1120.
- Müller F, Boeltz S, Knitza J, et al. CD19-targeted CAR T cells in refractory antisynthetase syndrome. Lancet. 2023 Mar 11;401(10379):815–818.
- Schett G, Mackensen A, Mougiakakos D. CAR T-cell therapy in autoimmune diseases. Lancet. 2023 Nov 25;402(10416):2034–2044.
- Anolik JH, Barnard J, Owen T, et al. Delayed memory B cell recovery in peripheral blood and lymphoid tissue in systemic lupus erythematosus after B cell depletion therapy. Arthritis Rheum. 2007 Sep;56(9):3044–3056.