This week, the University of Miami Health System and the Miller School of Medicine (both FL, USA) announced the launch of the first clinical trial to harness the potential of CAR-T therapy against autoimmune conditions (clinical trial number NCT04146051). The groundbreaking trial will utilize Descartes-08 CAR-T cells, a product from Cartesian Therapeutics (MD, USA), to target the cells that act as a source for the antibodies that lie behind the autoimmune condition myasthenia gravis.
Myasthenia gravis is a rare, long-term, autoimmune condition that disrupts the neuro-muscular junctions and leads to muscle fatigue and weakness in those affected. While some of the condition’s effects can be minor, such as drooping eyelids and a difficulty chewing, the severity can fluctuate within a patient, becoming life threatening in extreme cases where the weakness can result in respiratory difficulties. Currently, the only available treatments for myasthenia gravis are the removal of the thymus or a selection of non-selective immunosuppressive that can become toxic with long-term treatment. Researchers hope that the new CAR-T product will target the long-lived, antibody-producing B cells which are believed to be behind the condition.
“Cartesian’s CAR-T technology selectively targets the primary culprit in the disease: antibody-producing plasma cells. Such selective targeting would be a first in GMG and could help patients discontinue use of chronic immunosuppressive therapies,” commented the principal investigator, Volkan Granit (University of Miami Miller School of Medicine). “The strategy of targeting for elimination, the aberrant long-lived plasma cells that produce these autoantibodies with CAR-T cells, is a novel and very promising approach.”
The Miller School of Medicine is the home to the largest myasthenia gravis clinic in the region and has a collection of experts dedicated to the treatment and research of myasthenia gravis. If successful, the trial could pave the way for an array of trials looking into other autoimmune conditions.
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