A new experimental drug has yielded revolutionary results in laboratory models of multiple sclerosis: actually reversing paralysis and vision loss that has already developed in mice. The drug, Kamuvudine K-9, was created by researchers at the University of Virginia and may set researchers on a whole new course of investigation to seek a way to repair the damages of the disease, instead of simply preventing subsequent attacks.
The results were published in the journal Science Translational Medicine. The study was funded by the National Institutes of Health. It was stated that the results of this research are only in their initial stages, animals, and have no proof as yet that this treatment can cure multiple sclerosis in humans.
A Different Approach to Multiple Sclerosis
Multiple sclerosis is a neurological disorder in which the body’s immune system destroys myelin (protective sheath of the nerve fibers). Damage to the myelin may cause a disruption in communication between the brain, spinal cord and the rest of the body resulting in symptoms like weakness, mobility problems and visual problems.
Most MS drugs currently available work on reducing inflammation and minimizing relapses. What the investigators involved in the use of K-9 are interested in is of another approach that targets nerve fiber protection and cellular damage that can follow inflammation.
K-9 is a highly modified nucleoside/nucleotide reverse transcriptase inhibitors or NRTI, used in patients with the human immunodeficiency virus (HIV) or other disease. The design of K-9 was intended to target inflammatory pathways and maintain a better safety profile.
Paralysis and Vision Loss Were Reversed
In the experiments, researchers a mouse suffered with a disease similar to MS and waited until neurological symptoms become apparent. Once this point was reached the animals were treated with K-90 for two weeks.
The findings were striking as the treatment was implemented following the presentation of neurological deficits. K-9 was able to cease the progression of neurological damage and reverse existing paralysis and visual impairment. Experimental animals regained more function with K-9 than with any approved MS drug.
Secondly, evidence was demonstrated that K-9 helped to save the axons and myelin in the spinal cord. Axons are the long thin structures of nerves, which conduct electrical signals and myelin coats them to carry the signals down them more efficiently.
Targeting the Inflammasome
The investigators hypothesize that part of K-9s mechanism of action involves inhibiting activation of inflammasomes (molecular systems mediating inflammatory responses). In this study, the researchers observed the effects on NLRP3 inflammasome and NLRC4 inflammasomes.
That means, over activation of these systems could bring damage of cells. K-9 by modulating the mechanisms seem to be able to prevent nerve degeneration of neurones in the mouse model. K-9 also prevented the rise in neurofilament light chain, a blood biomarker of nerve damage, in the model.
The impact of which, could be critical because, researchers are not just trying to cut out the inflammation but to protect the nervous system from the adverse effects of the inflammation.