An experimental compound derived from approved HIV medicines reversed paralysis and vision loss in mice with a laboratory model of multiple sclerosis, according to work from UVA Health. That distinction matters: current MS therapies are built to prevent future inflammatory attacks, not to bring back neurological function that has already been lost.
The drug, Kamuvudine K-9, 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 the lab experiments, K-9 protected nerve fibers and the myelin sheath that MS strips away, and it stopped the rise of neurofilament light chain, a blood marker that tracks ongoing nerve damage. Animals given an already approved MS drug regained considerably less function than those on K-9.
Ambati's team also looked at real-world data to see whether the parent drugs behaved the same way in people. Analyzing health insurance records from more than 3 million individuals, the researchers found that use of nucleoside/nucleotide reverse transcriptase inhibitors, or NRTIs, was linked to a 41% lower risk of developing MS and a 36% lower annual relapse rate among people who already had the disease.
The combination of an animal result and a large human database signal is more persuasive than either alone, but neither amounts to a clinical trial. Insurance-claims analyses show association, not cause, and a mouse model of MS is a simplified stand-in for a complex human disease. K-9 itself has not been tested in people with MS.