In a recent news release from biotechnology company Cartesian Therapeutics, the company shared that the first patient was dosed in a Phase 2b study evaluating Descartes-08 for generalized myasthenia gravis (gMG). Altogether, 30 patients will enroll. Within the study, Cartesian Therapeutics hopes to better understand the therapy’s safety, efficacy, and tolerability. Additionally, the study hopes to determine how Descartes-08 might reduce markers of disease severity.

The Descartes-08 website, which highlights info regarding the clinical trial, explains that Descartes-08 is:

a T-cell therapy that is custom-made from a patient’s own blood [in which the] cells are engineered with an mRNA to redirect them to find and kill antibody-producing plasma cells. It is hoped that these T-cells, when administered back to the patient, will find and kill the aberrant plasma cells that produce the pathogenic antibodies in MG.

This first-in-class, investigational rCAR-T cell therapy is administered over a six-week period. Patients do not need to undergo any preconditioning to prepare for Descartes-08 treatment. So far, studies have shown that Descartes-08 shows durable and sustained responses. It will be interesting to see what further studies show.

Generalized Myasthenia Gravis (gMG): An Overview

Myasthenia gravis (MG) is an autoimmune neuromuscular disorder; the name literally means “grave muscle weakness.” MG is believed to be an antibody-mediated disease in which the body mistakenly targets proteins necessary for nerves and muscle cells to communicate. This causes muscle weakness and fatigue that worsens with activity. While myasthenia gravis refers to “grave” muscle weakness, the prognosis is fairly positive. Only around 10% of people will experience serious or life-threatening respiratory symptoms. Myasthenia gravis may be ocular or generalized, the latter of which affects ocular, arm, leg, and respiratory muscles.

Symptoms of gMG can include:

• Slurred speech
• Drooping eyelid(s)
• Double or blurred vision
• Difficulty chewing and/or swallowing
• Debilitating fatigue
• Changes in gait
• Impaired speech
• Shortness of breath
• Weakness of the upper and lower extremities
• Respiratory failure

Treatment options include corticosteroids, acetylcholinesterase inhibitors, immunosuppressants, plasma exchange, and IVIG. A small group of patients do not respond to therapy, leading to refractory gMG.