Shedding of GD2 ganglioside in patients with retinoblastoma.

Retinoblastoma is a rare tumor of the young child with an intraocular localization that leads to certain problems of diagnosis. With the aim of defining a biochemical marker--which is still lacking for this disease--the gangliosides of a pool of fresh retinoblastoma tumors were analyzed. The ganglioside pattern was shown to have GM3, GM2, GM1, GD3, GD2, GD1b and GT1b as the major components. The occurrence of a high concentration of GD2 in the tumors led us to investigate the possibility of changes in the level of GD2 in the sera of retinoblastoma patients, using quantitative immunostaining with GD2-specific mouse monoclonal antibodies (MAbs). In 9 out of 10 tumor-bearing patients, the serum level of GD2 ganglioside was significantly higher than the average value found in normal individuals. A 2-year follow-up of patients showed that successful treatment resulted in a rapid decrease in the serum level of GD2 down to the normal range, from which a subsequent elevation was seen only in relapsing patients. Although the clinical study needs further development, the results obtained to date suggest that GD2 is shed in the serum of tumor-bearing patients and that the level of GD2 could be a potential serum marker of human retinoblastoma.