In vitro results showed a significant decrease in the formation of toxic vitamin A aggregates by 7 fold (top right figure).
Animal receiving ALK-001 showed 70% reduction in the accumulation of toxic deposits called lipofuscin (bottom right fig.) and in 80% reduction of vitamin A aggregates (bottom center fig.) in their eyes. This trend continued after 6 and 12 months of treatment.
To measure visual function, electroretinogram (ERG) is used, measuring electrical response of the retina to light signals: after 12 months of treatment, untreated mice showed gradual losses of visual function. Mice receiving ALK-001 had significantly better responses showing that ALK-001 could prevent loss of visual function in Stargardt mice(bottom left fig.).
No side effects were noted in these experiments and the animals were safely administered the drug for a period of up to 12 months.
The results of these experiments show that preventing the aggregation of vitamin A in the retina can result in preserved visual function in mice with Stargardt disease.