The trial is testing the effectiveness of two chemotherapy drugs, oxaliplatin and gemcitabine, in combination with radiotherapy, an approach that has been used to treat other aggressive tumours
Recently, the US FDA has granted orphan drug status to two new therapeutics in a row. One is a small molecule inhibitor, PCLX-001, for the treatment of acute myeloid leukaemia; the other is a T-cell therapy, ET140203, for the treatment of hepatoblastoma. Both therapies are currently in relevant trials.
