Asset 2: Treatment of Ewing Sarcoma

Medical need: Ewing sarcomas are an aggressive type of tumors in children and young adults that belongs to the rare diseases. About a third of patients with metastases die within the first five years after diagnosis. The genetic causes for the growth of an Ewing sarcoma are well known: There is a genetic change that causes the formation of a new oncogenic factor, the so-called fusion protein EWS-FLI1. This changes the cells so that they grow uninhibited. This factor and of Ewing tumor cells have already been inhibited in the laboratory. This did not work in the patient, the EWS-FLI1 factor was considered “undruggable”, so there is no specific help for the young patients.

Drugging the undruggables: We established a nanocarrier BBB-002 made of and IGF1R antibody capable of detecting and entering Ewing tumor cells.
As a result, the nanocarriers formed by IGF1R-antibodies, along with the protamine and the siRNAs against the EWS-FLI1 bind to the Ewing cells and are internalized. Inside the cell, the siRNAs can then prevent the translation of the EWS-FLI1 oncoprotein and thus deprive the Ewing tumor cells of the basis for their uninhibited growth.