According to Myasthenia Gravis News, CAAR T-cell therapy may be a viable treatment option for those with myasthenia gravis. Researchers from the University of Pennsylvania, Yale Medical School, and Cabaletta Bio created this treatment to bind specifically to damaging B-cells while leaving healthy ones alone. While this therapy has only been tested on mice so far, researchers hope to take the next step to make it available to those who need it.
About Myasthenia Gravis
Myasthenia gravis (MG) is the most common autoimmune neuromuscular disorder. Those affected experience weakness in the voluntarily controlled muscles. Physical activity worsens the symptoms, whereas periods of rest can improve them. Throughout the world, about 20 of every 100,000 people have MG. It is an autoimmune condition, meaning the body attacks itself, specifically the proteins that are necessary for communication between the brain and muscles. This causes symptoms like drooping eyelids, issues with chewing and swallowing, fatigue and weakness in the skeletal muscles, slurred speech, double vision, and a changed gait. For a small percentage of those with MG, weakness in the chest could lead to life-threatening respiratory issues.
Doctors diagnose this condition through a physical exam, evaluation of patient history, blood tests, and EMGs. Like many rare disorders, a diagnosis is not always easy to obtain. The similarity of symptoms to other conditions often results in a misdiagnosis. After doctors have confirmed that one has MG, treatment consists of steroids, plasmapheresis, or surgery to remove the thymus gland. While there is no cure, some medications can greatly improve symptoms or even induce remission.
About CAAR T-Cell Therapy
This immunotherapy is meant to target the malfunctioning B-cells that damage the MuSK protein, while sparing the normal cells. It isolates T-cells so that they target the abnormal B-cells upon being returned to the system.
In-vitro studies showed positive results, so researchers moved on to animal models, specifically mice. The mice had weak immune systems and were given anti-MuSK B-cells before being treated with the CAAR T-cell therapy. After 13 days, the anti-MuSK cells were successfully eliminated. Researchers are excited by these results and plan to conduct more animal trials in the hope of making it to human studies.
As those with myasthenia gravis face limited treatment options, this study is very exciting. With more positive results, this immunotherapy could offer another viable treatment.
