Multiple sclerosis (MS) is a debilitating disease that affects the central nervous system (CNS), which is made up of the brain and spinal cord. Optic nerves, considered to be extensions of the CNS, send signals from the eyes to the brain and are commonly affected in MS. Although MS is currently incurable, a newly developed drug may be just the cure we’ve been waiting for.
MS occurs when the immune system mistakenly attacks the nervous system’s myelin sheaths, which are structures that insulate, protect, and maximize the efficiency of nerve cells. When MS attacks myelin sheaths, it disrupts communication between the nervous system and other body systems and potentially causes permanent damage to nerve cells. Symptoms can include muscle weakness, fatigue, and vision problems.
Although MS has no cure, there are other forms of nerve cell damage that can be repaired through means like surgery.
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Current treatments include disease-modifying therapies, which suppress the immune system, reducing its attacks on myelin sheaths and preventing further damage. There are many disease-modifying therapies, including a prescription medicine called, glatiramer acetate, which mimics myelin and tricks the immune system into attacking glatiramer acetate instead of actual myelin. These treatments only slow or stabilize the progression of MS and improve symptoms–they aren’t a cure.
However, a new drug called PIPE-307, currently in clinical trials, may soon change this. PIPE-307 has shown promise in reversing the damage caused by MS, potentially revolutionizing MS treatment.
The creation of PIPE-307 began with the discovery that the drug clemastine stimulates special nerve cells called oligodendrocyte precursor cells (OPCs). OPCs have the potential to develop into myelin-producing oligodendrocytes, another type of nerve cell. Specifically, clemastine blocks chemical sensors in the body called “muscarinic receptors”, and in turn promotes OPCs to form new myelin sheaths that can replace myelin deteriorated due to MS. Clemastine was the first drug shown to repair MS-damaged myelin, making this a groundbreaking discovery–made even more interesting by the fact that clemastine was not even developed for this purpose. It is actually an antihistamine, a type of allergy medication.
However, there was a problem with this discovery: clemastine isn’t effective enough at repairing myelin because it affects multiple pathways and is not specific to the receptor affected by MS, called “M1R“. This broad activity “dilutes” the amount of clemastine that targets M1R, as some clemastine targets other receptors unnecessarily.
To solve this problem, researchers utilized a toxin called MT7 found in green mamba snake venom. MT7 can select for M1R specifically, and thus direct clemastine towards the M1R receptors only. Researchers combined MT7 and clemastine to create the novel drug PIPE-307, a dramatic improvement from clemastine alone. PIPE-307 is much more effective in rebuilding myelin and has even been shown to reverse myelin degradation in mice with MS.
This development brings hope to those with MS. If PIPE-307 is successful, it could potentially restore the quality of life for patients. As we await the clinical trial results in 2025, the future looks brighter for MS patients around the world.
Featured Image: Generative AI
