First patient in the world treated for muscle-weakness disease in clinical trial at HonorHealth Research Institute

(Press-News.org) SCOTTSDALE, Ariz. — June 16, 2025 — 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.

For more about Neuroscience Research Division clinical trials at HonorHealth Research Institute: call 602-258-2863; or email neuroscienceresearch@honorhealth.com.

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About the HonorHealth Research Institute
HonorHealth Research Institute is an international destination that is at the forefront of providing patients with a better quality of life through its clinical trials and innovative treatment options. Headquartered in Scottsdale, Arizona, the institute’s team of physicians and researchers collaborate with experts from across the nation to offer life-changing therapies, drugs and devices. At HonorHealth Research Institute, patients have access to tomorrow’s health innovations, today. Learn more at: HonorHealth.com/research.
 

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