Treatment with ST266 linked to increased survival of retinal ganglion cells in patients with multiple sclerosis.
Noveome Biotherapeutics recently announced positive results from a preclinical study of an investigational secretome, ST266, for the treatment of multiple sclerosis (MS).
The intranasal drug was observed to have anti-inflammatory activity, and prevented retinal ganglion cell loss in the optic nerve. These findings show the drug’s potential as a treatment for optic neuritis, a common symptom of MS, according to a press release.
Optic neuritis involves inflammation of the optic nerve, whichcan result in pain and temporary vision loss. While the condition may get better naturally, steroids are typically used to reduce inflammation.
ST266 is a complex solution of active molecules secreted from cells, and induces changes in nearby cells through paracrine signaling, Noveome reported. This alters cells to control inflammation, speed wound healing, promote bone growth, restore nerve function, regenerate cells, and restore balance to cells.
In the preclinical study, ST266 was found to reach the central nervous within 30 minutes of administration. The investigators discovered a higher concentration of the drug in the vitreous and optic nerve compared with the brain, which suggests it may effectively target the eye.
Early treatment with ST266 was observed to prevent neuronal damage by reducing the loss of retinal ganglion cells, suppressing inflammation in the optic nerve, and limiting demyelination caused by optic neuritis, according to the study.
Similarly, ST266 was found to reduce neuronal damage and demyelination in late-stage optic neuritis. Treatment was associated with improved vision compared with the control groups.
These findings suggest that the drug increases retinal ganglion cell (RGC) survival through activating pathways, such as stimulating SIRT1-mediated mitochondrial function and AKT phosphorylation to prevent cell death, Noveome reported.
“Current therapies reduce inflammation but fail to prevent RGC loss; thus, there is a need for combination treatment options that are able to prevent RGC axon loss for patients with optic neuritis. The unique and diverse biologic molecules present in ST266 were seen to help promote anti-inflammatory and neuroprotective activity in this preclinical model and suggest that ST266 has the potential to mediate neuroprotection through activation of multiple intracellular signaling pathways,” said researcher Kenneth S. Shindler, MD, PhD. “These results are particularly important as the preservation of RGCs has been recognized as a significant factor when treating optic neuritis due to potential permanent visual dysfunction.”
Currently, intranasal drugs are not used to treat ophthalmic conditions. The findings from the preclinical study suggest that this drug delivery method should be explored further.
“We believe this is the first demonstration of a potentially successful therapeutic treatment of the optic nerve using intranasal delivery of large molecular weight biomolecules,” commented Larry Brown, ScD, chief scientific officer of Noveome. “These promising results reinforce the multifaceted potential of ST266 in multiple disease areas, including disorders in the back of the eye. The study also reconfirmed the safety profile and potent nature of ST266 in a preclinical model, which provides encouragement and support for continued research.”