First patient in the world treated for muscle-weakness disease in clinical trial

The first patient enrolled in a planned international clinical trial has been treated at HonorHealth Research Institute with a new type of immune therapy for those with a rare muscle-weakness disease known as Myasthenia Gravis.

In an odd medical twist, this auto-immune disease—in which antibodies interfere with the connection between nerves and muscles—most often strikes young women in their 20s and 30s, and older men in their 60s and 70s, though it can affect others.

The disease affects fewer than 200 in every 1 million people. Symptoms range from droopy eyelids, problems chewing, general fatigue, difficulty swallowing and even problems breathing when the muscles surrounding the lungs are weakened. Patients often find it difficult to conduct basic hygiene and grooming; things as simple as brushing their teeth, taking a shower and getting dressed.

"It's kind of exciting," said Anne Hatch, D.O., Principal Investigator for this international clinical trial (NTC06744920), which will consider the safety and effectiveness of a drug called Remibrutinib, manufactured by Novartis Pharmaceuticals of Basel, Switzerland.

Dr. Hatch explained that, for the first time in this disease, the drug will target B cells, a type of white blood cell that makes antibodies. While usually an important part of the body's immune system, she said, in Myasthenia Gravis malfunctioning white blood cells interfere with the messaging between the nervous system and muscles in what is known as the neuro-muscular-junction pathway.

"This new drug blocks a different part of the pathway than before," Dr. Hatch said. "Hopefully, with this new mechanism of action, the patient's symptoms will improve."

Aiming for FDA approval

If successful, this Phase III clinical trial could lead to the drug being approved by the FDA for Myasthenia Gravis . The treatment has already been approved for some types of cancer.

Trial participants must be 18–75 years old with a confirmed diagnosis of Myasthenia Gravis. They must be able to breathe without a respirator and be able to safely swallow the oral medication. A patient's progress could be followed for up to 5 years.

Provided by HonorHealth Research Institute