UCSF Team Tests New Drug That Could Reverse Multiple Sclerosis

Multiple sclerosis is an autoimmune condition that affects an individual’s brain and spinal cord, causing symptoms that include difficulty walking or problems with one’s vision or balance. The condition’s mechanism of action involves using an individual’s immune system to attack the myelin, a protein and fatty substance that protects and surrounds nerve fibers. By damaging myelin, nerves aren’t able to carry electrical impulses from and to the brain. This damage also allows scar tissue known as sclerosis to form.

This chronic disease of the central nervous system may, in some cases, lead to severe disability.

Now, researchers at Contineum Therapeutics and University of Californi at  San Francisco have developed a new drug that could help reverse the damage caused by multiple sclerosis. The drug in question, PIPE-307, has been designed to target the M1R receptors found in the brain and trigger those receptors to mature into oligodendrocytes, which can produce myelin.

Michael Poon, the first author of this study, explained that it was important that the researchers prove that the M1R receptor could be found on the cells that could repair damaged fibers.

This latest study was based on an earlier breakthrough from another study which discovered that clemastine, an antihistamine, could cause remyelination. The pioneering work involved UCSF scientists Dr. Arin Green and Professor Jonah Chan, who discovered that clemastine activated oligodendrocyte precursor cells, allowing them to mature into oligodendrocytes that could produce myelin.

For this latest study, the researchers began by studying clemastine to better understand how it helped regenerate myelin. They discovered that clemastine blocked one of the five muscarinic receptors, noting that since their focus was on M1R, they would develop a drug that blocked this receptor exclusively.

It was here that Poon, a biologist by profession, discovered that the toxin MT7, which is found in the venom of the green mamba snake, could show the exact location of M1R receptors in the brain. The scientists’ objective, Poon reiterated, had been to find indisputable evidence that M1R was present in oligodendrocyte precursor cells found near damage caused by multiple sclerosis. This allowed the researchers to develop a molecularfor M1R that showed oligodendrocyte precursor cells assembling around damaged tissue.

Medicinal chemists at Contineum, led by Austin Chen, then took over to design PIPE-307. The formulation would work to block M1R receptors, trigger oligodendrocyte precursor cells to mature into oligodendrocytes and produce myelin, beginning remyelination.

The researchers observed in their trials that the formulation could cross the blood-brain barrier.

Thus far, the formulation has cleared phase 1 trials with researchers demonstrating its safety. The researchers are focused on testing it in patients with multiple sclerosis during phase 2 trials. The success of this new drug could change how multiple sclerosis is treated.

The study, whose findings were reported in “PNAS,” was partially funded by the National Institute of Neurological Disorders and Stroke.

As many other entities, such as Clene Inc. (NASDAQ: CLNN), devote research resources to seeking effective treatments for this disease, a time may come when patients have many options to obtain the desired clinical outcomes, which isn’t currently possible with the existing MS treatments.

NOTE TO INVESTORS: The latest news and updates relating to Clene Inc. (NASDAQ: CLNN) are available in the company’s newsroom at https://ibn.fm/CLNN

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