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2 mins read
For years, multiple sclerosis has been treated as a disease that can only be slowed, not reversed. However, recent research has identified two experimental compounds—K102 and K110—capable of repairing damaged myelin and restoring neurological function in disease models. The finding, the result of more than a decade of scientific work and published in Nature Scientific Reports, opens the possibility of developing therapies that not only halt progression but also reverse existing damage.
Multiple sclerosis is a chronic autoimmune disease in which the immune system mistakenly attacks the myelin sheath, the layer that protects nerve fibers and enables efficient transmission of signals between the brain and body. Its deterioration causes visual disturbances, loss of coordination, and, in more severe cases, paralysis. Although current treatments can slow disease progression, no therapy yet exists that can repair the damage already done.
The new study, born of more than a decade of collaboration between the University of California and the University of Illinois, shows progress that could change that outlook. Researchers tested more than 60 indazole chloride derivatives before identifying the two most promising compounds: K102 and K110, both designed to selectively activate the estrogen receptor beta (ERβ), a key molecular pathway in nervous system repair.
Why is this receptor important? ERβ is present in brain and spinal cord cells that produce myelin. Activating it stimulates the maturation of oligodendrocytes, the cells responsible for regenerating the myelin sheath, without causing hormonal side effects. In experimental models, K102 not only promoted remyelination but also reduced inflammatory cytokines (such as TNF-α and CXCL10) and improved visual and motor function by restoring the integrity of nerve fibers.
K110, meanwhile, showed complementary results: it promoted myelin repair and mitochondrial health, though its action seems better suited for spinal cord injuries or traumatic brain lesions. Both compounds demonstrated safe pharmacological profiles and remarkable cellular protection capacity.
The development program for these molecules was adopted by biotech company Cadenza Bio, which now leads preclinical studies and aims to move toward human clinical trials. “Our goal is to go beyond slowing the disease: we want to reverse the damage and improve patients’ quality of life,” said Carol Curtis, the company’s CEO.
The results mark a paradigm shift: moving from therapies focused on controlling inflammation to treatments that drive active regeneration of nervous tissue. If clinical trials confirm their efficacy, K102 could become the first drug capable of rebuilding myelin and usher in a new era in the treatment of multiple sclerosis and other neurological diseases.