Ganglioside administration in retinitis pigmentosa

The retinitis pigmentosa process typically causes variably progressive visual loss due to retinal photoreceptor and pigment epithelial cell deterioration. No effective agent either to retard or stop the deterioration has been known. Because gangliosides have been shown to have a variety of trophic effects on peripheral nervous tissue, we administered a daily 40 mg intramuscular dose in a prospective, double-blind, randomized, placebo-controlled trial in 30 persons with retinitis pigmentosa with or without congenital deafness. When we compared baseline performance on the two principal study outcomes, visual field area and electroretinographic response amplitudes, there was a marginally statistically significant increase in visual field area in the ganglioside-treated group. The subgroup of subjects who had recordable electroretinograms at baseline and who received the drug showed in three of five cases an increase in amplitude to all stimuli. These results appear encouraging; however, the data must be interpreted cautiously because the numbers of individuals are very small. Further study of the possible benefits of ganglioside administration to retinitis pigmentosa patients appears warranted.