The Lankenau Institute for Medical Research (LIMR) announced that in collaboration with the Massachusetts Institute of Technology (MIT) they have performed preclinical proof-of- principle studies showing how nanotechnology can be used to enhance gene therapy for cancer.
The results of the study, led by Janet Sawicki, Ph.D. at LIMR and Robert Langer, Ph.D. and Daniel Anderson, Ph.D. at MIT, were reported as a cover article in a recent issue of the Proceedings of the National Academy of Sciences.
This advance offers an alternative method of gene therapy that has advantages over earlier technology. The nanotechnology-based approach used by the researchers has minimal toxic side effects to normal cells. Moreover, it does not rely upon recombinant viruses, the use of which has been questioned recently due to the serious adverse effects they can have in some patients. Such problems have prompted researchers to find alternative methods for delivering gene therapy.
Dr. Sawicki is an expert in preclinical studies of gene therapy for prostate cancer, and she is excited about the success of the current approach. In this study, the MIT group identified a polymer termed C32 that the Lankenau group demonstrated was capable of delivering genes to cancer cells more efficiently and with less toxicity than other polymers that have been tested in the field to date. C32 works by condensing the DNA in a gene and allowing the resulting nanoparticles that are formed to enter cells through a process called endocytosis. Therapeutic genes delivered to cells in this manner are able to drive cellular production of a gene-encoded protein through normal processes.