Abstract: The invention is directed to the use of Smac to sensitize different tumors and self-reactive immune cells to various pro-apoptosic stimuli, in that the cells subsequently undergo apoptosis. Therefore, Smac can be used as a compound for the manufacture of a medicament for the treatment of cancer and autoimmune diseases. Sensitization of the cells is achieved either by applying a cell-permeable form of Smac combined with known anticancer agents or by overexpression of the protein. It is an object of the invention to provide a new method in cancer and autoimmune disease therapy by using Smac agonists for apoptosis regulation. Thus, Smac agonists represent novel promising cancer and autoimmune disease therapeutics to potentiate the efficacy of cytotoxic therapies even in resistant tumors and immune cells.

Abstract: The present invention is, generally, directed to the use of betulinic acid and derivatives thereof for the treatment of neuroectodermal tumors. The present invention is based on the discovery that betulinic acid and its derivatives are potent anti-neuroectodermal agents. As disclosed herein, betulinic acid and its derivatives are useful for the treatment of neuroectodermal tumors, including, due to its distinct mechanism of action, neuroectodermal tumors that are resistant to conventional chemotherapeutical agents. In addition to the new use of known compounds, the invention discloses novel compounds and pharmaceutical compositions for the treatment of neuroectodermal tumors.