CAR-T Therapy for Multiple Sclerosis Explained by Neurologist

About this Video

• Video Title: CAR-T Therapy for Multiple Sclerosis Explained by Neurologist
• Channel: Dr. Brandon Beaber
• Speakers: Dr. Brandon Beaber
• Duration: 00:16:25

Introduction

This video explains CAR-T cell therapy, a treatment initially developed for cancer, and its potential application in treating multiple sclerosis (MS) and other autoimmune diseases. Dr. Beaber discusses the process of CAR-T therapy, its mechanism of action, current clinical trials, potential benefits, and significant side effects.

Key Takeaways

• CAR-T cell therapy: Harvests, genetically modifies, and reinfuses a patient's own T-cells to target specific antigens. It's expensive (up to $500,000).
• Mechanism of action: Engineered T-cells express a chimeric antigen receptor (CAR) with an antigen recognition domain (e.g., targeting CD19 on B lymphocytes), a hinge region for flexibility, and CD3 Zeta for intracellular signaling. Co-stimulatory proteins (e.g., OX40) enhance the T-cell's activity.
• Current applications & research: FDA-approved for certain B-cell cancers. Research is ongoing for MS, lupus, myositis, systemic sclerosis, and myasthenia gravis. Trials use anti-CD19 or anti-BCMA CAR-T cells.
• Clinical trial data: Small case series show promising results in lupus, with remission in all five patients. Results in MS are less conclusive; some patients experienced improvement, while others had stable or worsening disease. Significant side effects were observed.
• Side effects: Cytokine release syndrome (CRS) causing rash, wheezing, fever; Immune effector cell-associated neurotoxicity syndrome (ICANS) causing brain swelling and neurological symptoms; low B-cell counts and increased infection risk.