In a study published in Science Translational Medicine in March 2024, researchers from Aarhus University, Denmark, achieved a breakthrough in treating myasthenia gravis. Their study showed that a new chemical molecule named NMD670 improved muscle functions in rats. Their experiments which extended to 12 people with myasthenia gravis also showed promising results, indicating a significant advancement in the treatment of the condition.
In a statement, Dr Thomas Holm Pedersen, associate professor, Department of Biomedicine, Aarhus University said, “People in the trial became stronger because the new treatment improves the communication between motor nerves and muscle fibres.”
NMD670 targets and blocks an ion channel involving chloride ions in muscles. These ions play a crucial role in triggering muscle activity upon receiving the signal from the neurons. The study found that NMD670 improved neuromuscular transmission and restored muscle function in rats and humans. The good thing about the chemical is that it can be administered orally—like a pill – and has no known side effects.
When nerves and muscles lose communication
Myasthenia gravis is a debilitating neuromuscular condition with impaired crosstalk between nerves and muscles. The junctions between motor neurons and muscles are specialised junctions or synapses. When specific ions flow into neurons, the neurons release neurotransmitters. These chemicals bind to receptors in the muscle membrane, facilitating the transmission of electrical signals to the muscle fibres. The electrical signal causes the muscles to contract, triggering their action.
In myasthenia gravis, antibodies are produced in the body, which act against the neurotransmitter receptors at the junction. This leads to disruption in the communication between nerves and muscles, which prevents their contraction and results in muscle weakness. People with this condition struggle to perform their day-to-day activities. In severe cases, it can be life-threatening when the muscles involved in breathing are affected.
Currently, no effective muscle-specific treatment is available for the condition; it can only be managed by immunosuppressants and medicines to improve muscle activity. The current research offers hope for restoring muscle activity.
Their previous studies found that ClC-1 chloride channels at the junction of nerves and muscles, are crucial for muscle activation. “This led to the idea of using these channels as a new treatment point for conditions where the neuromuscular connection is compromised, including myasthenia gravis,” according to the statement.
When the new chemical molecule was given to rats, it improved their grip strength and movement. In the next step, the compound was given orally to 12 participants with mild myasthenia gravis in a randomised clinical trial. The participants’ grip strength and muscle functions improved across different muscle groups.
Future course
The breakthrough could result in significant improvement in the quality of life for people with myasthenia gravis, but it is also a big leap forward in understanding and treating other neuromuscular conditions. “We expect that the benefit would be even more pronounced in people experiencing moderate and severe symptoms, alone or in combination with conventional or newer myasthenia gravis treatments,” Dr Pedersen told Happiest Health.
Currently, the team is scaling their experiments in people with severe myasthenia gravis, spinal muscular atrophy, and Charcot-Marie-Tooth (CMT) disease. They are also attempting to find the optimal dosage for maximum benefits, said Dr Pederson.
Editor’s note: In a recent advancement, FDA has approved phase 2b trials of the compound.
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