If given the green light by the FDA, a CAR T cell therapy from Kyverna Therapeutics would be the first approved treatment for stiff person syndrome. Scientists are now testing CAR T cell therapy in nearly a dozen autoimmune diseases linked to harmful B cells, including lupus, rheumatoid arthritis and a connective-tissue disease known as systemic sclerosis. Kyverna, the California-based company behind the SPS trial, is also running trials for neurological autoimmune conditions including generalized myasthenia gravis and progressive multiple sclerosis, or MS. CAR T cell therapies are transformative, Konig says. “They’re going to change the field — but there are risks.” · Another risk is that the once-vanquished disease will return. Doctors don’t know all the genetic and environmental conditions that trigger autoimmune disease. In 2017, the FDA approved the first CAR T cell therapies for two blood cancers, acute lymphoblastic leukemia and large B cell lymphoma. Soon after, scientists saw the first tantalizing hints that it might work for autoimmune diseases, too. In some cases, cancer patients with autoimmune disorders reported improvements in both conditions after treatment. At 40, Lynn Montgomery-Haga was diagnosed with stiff person syndrome, a rare autoimmune disease that can cause severe muscle spasms and rigidity. In 2024, she received an experimental CAR T cell therapy for her condition — then her mobility began to come back.Animations by Eric Pio; Edited by Damien S.

A recent publication discusses the management of subclavian and axillary artery aneurysms in patients with connective tissue disease, highlighting the lack of dedicated guidelines for this rare vascular condition. The study emphasizes the therapeutic uncertainties faced by clinicians in treating these aneurysms.

A systematic literature review highlights the importance of early detection and treatment initiation in improving long-term health outcomes for patients with connective tissue disease-associated pulmonary arterial hypertension. Expert consensus emphasizes the need for enhanced screening protocols.

The large dose of brepocitinib, a JAK1/TYK2 inhibitor, showed benefits on a composite score and secondary end points. The FDA has set a PDUFA date for the drug in the third quarter of 2026, according to its developer. Close to 10% (9.95) of the patients randomly assigned to the 30-mg dose of brepocitinib experienced a serious infection during the yearlong trial, compared with 2.5% of the patients assigned to the 15-mg dose and 1.3% in the placebo group. Vleugels noted that brepocitinib was “layered on” other treatments. “For those who are experts in dermatomyositis, this would be something we would expect. The more therapies you lay on, you have to be mindful of infections,” she said. The primary end point was a composite of six measures of myositis activity called the Total Improvement Score. Vleugels said that score has been used in other studies of treatments for dermatomyositis and that it encompasses both the muscle and skin aspects of the disease. The trial design called for a treatment period of 52 weeks. “If you had asked me when I started practicing almost 20 years ago if we would ever have a pivotal Phase 3, global, randomized controlled trial in dermatomyositis, I don’t know if I would have believed it,” co-lead author · Ruth Ann Vleugels, M.D., M.P.H., MBA, a professor of dermatology at Harvard Medical School and director of Connective Tissue Disease Clinics at Harvard-affiliated Brigham and Women’s Hospital, said in an interview with Managed Healthcare Executive. Brepocitinib, the agent assessed in the trial, is an oral drug that inhibits Janus kinase 1 (JAK1) and tyrosine kinase 2 (TYK2), a duality that explains its efficacy because it stifles multiple cytokines implicated in the pathogenesis of dermatomyositis in addition to interferon 1 and 2, Vleugels said. The FDA has put brepocitinib in a category that is supposed to mean a speedier review process. Priovant Therapeutics, the Durham, North Carolina, company that developed brepocitinib, says its product has a Prescription Drug User Fee Act (PDUFA) date — the date on which the FDA is supposed to make a decision on whether to approve a drug — in the third quarter of this year.

A recent study published in PubMed highlights the extended follow-up of CD19 CAR T-cell therapy in patients with connective tissue disease-associated pulmonary arterial hypertension. This research contributes to understanding the potential of CAR T-cell therapy in rare diseases.