RUNX1 Inhibition as Treatment for CNV

Article: New therapeutic approach may help treat age-related macular degeneration effectively
Source: Mass Eye and Ear, in Elsevier, via ScienceDaily  and NEI
Published: February 22, 2021

In this study, researchers affiliated with Harvard Medical School explored the use of a Runt-related transcription factor 1 (RUNX1) inhibitor, with and without the anti–vascular endothelial growth factor (VEGF) aflibercept (Eylea), for the treatment of choroidal neovascularization (CNV) in exudative age-related macular degeneration (wet AMD). RUNX1 has previously been linked with CNV pathogenesis, that is, it seems to be involved in not only angiogenesis but also in the inflammation and fibrosis leading to CNV. Inhibition of RUNX1 thus provides a novel and intriguing approach to address the problem of incomplete response in anti-VEGF therapy. The researchers used a mouse model of CNV to test intravitreal injection of saline, aflibercept, the RUNX1 inhibitor Ro5-3335, and a combination of Ro5-3335 and aflibercept. They found that a single intravitreal injection of Ro5-3335 alone significantly decreased the CNV lesion size seven days after induction of the CNV lesions, and that the combination of Ro5-3335 and aflibercept was more effective at reducing vascular leakage than aflibercept alone. One of the researchers states, “RUNX1 inhibitors hold significant promise to complement or replace anti-VEGF therapies for patients in which anti-VEGF therapy is no longer effective, and with the potential to be administered topically it could be transformative in the field.” Because many eye diseases involve neovascularization, RUNX1 inhibition has great potential to be applicable to diseases ranging from wet AMD and diabetic retinopathy to retinopathy of prematurity and retinal vein occlusion.

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Gonzalez-Buendia L, Delgado-Tirado S, An M, et al. "Treatment of Experimental Choroidal Neovascularization via RUNX1 Inhibition." American Journal of Pathology.  Published online 23 December 2020. https://doi.org/10.1016/j.ophtha.2020.12.009