The goal of the projects financed by BMBF and ROSNAUKA was to investigate the feasibility of developing a new therapeutic approach based on a relative new technology termed RNA-interference for the treatment of leukemia patients. In this approach, a specific gene that is deregulated in the leukemic cells is "silenced" by using small interfering RNAs (siRNA). These sequences bind to the RNA produced by the gene, leading to their degradation. For this approach to work, two requirements have to be fulfilled:
- The chosen gene has to be a "lynchpin" in the cancerous cell, in that its loss will lead to the collapse of the cell.
- The siRNA has to be reach the vast majority of the cancerous cells and be stable long enough to silence the gene.
In the funding period, it has been able to demonstrate using animal models that a gene called RUNX1, which is deregulated in circa 20% of human acute leukemia, fulfils the "lynchpin" function. In this study viral vectors were used to introduce the siRNA into the cell – an approach that may not be feasible in the clinic.
Thus, the Russian collaborators are investigating methods by which the siRNAs can be delivered directly and stably to the leukemic cells. This approach would then be a promising alternative to current therapeutic strategies with relatively poor overall success rates in adults.
