Tiny particles carrying a killer gene can effectively suppress ovarian tumor growth in mice, according to a team of researchers from MIT and the Lankenau Institute.
The findings could lead to a new treatment for ovarian cancer, which now causes more than 15,000 deaths each year in the United States. Because it is usually diagnosed at a relatively late stage, ovarian cancer is one of the most deadly forms of the disease.
The new treatment, reported in the Aug. 1 issue of the journal Cancer Research, delivers a gene that produces the diphtheria toxin, which kills cells by disrupting their ability to manufacture proteins. The toxin is normally produced by the bacterium Corynebacterium diphtheriae.
Human clinical trials could start, after some additional preclinical studies, in about a year or two, says Daniel Anderson, research associate in the David H. Koch Institute for Integrative Cancer Research at MIT and a senior author of the paper.
Currently ovarian cancer patients undergo surgery followed by chemotherapy. In many cases, the cancer returns after treatment, and there are no good therapies for recurring and advanced-stage tumors.
Anderson and others from MIT, including Institute Professor Robert Langer, along with researchers from the Lankenau Institute, led by Professor Janet Sawicki, found that the gene-therapy treatment was equally as effective, and in some cases more effective, than the traditional chemotherapy combination of cisplatin and paclitaxel. Furthermore, it did not have the toxic side effects of chemotherapy because the gene is engineered to be overexpressed in ovarian cells but is inactive in other cell types.