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Experimental drug reverses paralysis and vision loss in multiple sclerosis model

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Experimental drug reverses paralysis and vision loss in multiple sclerosis model

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A drug developed by a UVA Health scientist has not only stopped but reversed paralysis and vision loss in a mouse model of multiple sclerosis. The results are striking because existing MS treatments are designed to reduce future inflammatory attacks, not restore neurological function already lost.

The drug, Kamuvudine K-9, is a derivative of HIV drugs already approved for use in people. It was developed by Jayakrishna Ambati, MD, founding director of UVA's Center for Advanced Vision Science and the DuPont Guerry III Professor in the UVA School of Medicine's Department of Ophthalmology.

In Ambati's new lab results, K-9 preserved nerve fibers and their myelin insulation that MS destroys. The drug also halted the rise in neurofilament light chain (NfL), a blood biomarker of nerve damage. In the same experiments, animals treated with an approved MS drug did not recover nearly as much function as those receiving K-9.

Ambati said the case for the therapeutic potential of K-9 is bolstered by the apparent MS protection offered by the HIV drugs K-9 is derived from, called nucleoside/nucleotide reverse transcriptase inhibitors, or NRTIs. NRTI use in patients, Ambati's team reports in a new paper, is associated with a 41% reduction in the risk of developing MS and a 36% reduction in the annual relapse rate among patients with MS. The researchers drew this conclusion based on an analysis of health insurance data from more than 3 million people.


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