Photo Credit: iStock.com/Nemes Laszlo

A team of physicians recently reported the successful treatment of refractory autoimmune neuropathies through the infusion of autologous anti-CD19 CAR T-cells.

In a first-in-human effort reported in The Lancet Neurology, physicians at Ruhr-University Bochum infused autologous anti-CD19 chimeric antigen receptor (CAR) T-cells into two patients with treatment-resistant autoimmune neuropathies—a motor variant of chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) and an anti-neurofilament protein (NF)155 paranodopathy. According to the report, the engineered lymphocytes expanded briskly and eliminated circulating B-cells, the presumptive drivers of disease.

“Anti-CD19 chimeric antigen receptor (CAR) T cells have shown promising therapeutic potential in autoimmune conditions through substantial and sustained B-cell depletion,” wrote report corresponding author Jeremias Motte, MD, Ruhr-University Bochum. “Here, we report treatment of two patients with severe, treatment-refractory autoimmune neuropathies using autologous anti-CD19 CAR T-cells.”

Rapid Functional Recovery

According to the authors, both patients experienced grade 1–2 cytokine-release syndrome that resolved with tocilizumab and corticosteroids, and one transient iliopsoas bleed was successfully embolized.

“Surprisingly, we saw early signs of neurological improvement within days of infusion,” stated Dr. Motte in a news release.

According to the authors, six months post-procedure, the 72-year-old patient who had been intermittently immobile could perform squats and pull-ups, while the 54-year-old patient regained unaided ambulation for 200 m. Objective markers echoed the clinical gains, the authors noted: compound muscle action potentials rose by up to 200%, serum neurofilament-light levels normalized, and pathogenic anti-GM1 immunoglobulin M and anti-NF155 immunoglobulin G antibodies became undetectable.

Durable Remission Sans Ongoing Immunosuppression

Prior regimens—intravenous immunoglobulin, plasmapheresis, high-dose steroids, cyclophosphamide, rituximab, and even proteasome inhibition—had failed for both patients, according to the report. Yet a single CAR-T infusion permitted complete discontinuation of all concomitant immunotherapies.

“The safe production and use of this innovative therapy for autoimmune disease signal a new era for hematology and neurology,” said co-author Roland Schroers, MD, of Ruhr-University Bochum, who directed the cell-manufacturing program.

Mechanistic Insights

The authors reported that deep B-cell aplasia, sustained for up to ten months, paralleled the disappearance of autoantibody titers, underscoring the importance of CD19-directed plasmablast depletion. Electrophysiological gains—faster nerve-conduction velocities, shorter distal motor latencies, and F-wave times—suggested actual remyelination rather than mere symptomatic relief.

Bringing Hope

While complement inhibitors and neonatal Fc receptor antagonists require lifelong dosing and remain costly, CAR T-cell therapy offers a one-time intervention with the potential for long-lasting remission, according to the authors.

“Our results show that precision medicine can become reality in neuroimmunology, bringing hope to patients whom conventional treatments have failed,” concluded co-corresponding author Ralf Gold, MD, of Ruhr-University Bochum.

The team added that prospective trials are now planned to confirm the efficacy, durability, and safety of the treatment across a broader spectrum of immune-mediated neuropathies.