Gene therapy has fallen short as a practical human medicine, in part because of the lack of safe ways of transporting genes into cells to treat diseases. In lab tests, scientists at Queen's University Belfast used a so-called Designer Biomimetic Vector to deliver a toxic gene into breast cancer cells.
The group described the vector as "a nanoparticle 400 times smaller than the width of a human hair." Once the vector delivers the gene "iNOS" into breast cancer cells, the gene promotes the production of nitric oxide, which either kills the cells or makes them more vulnerable to existing cancer therapies. Insofar as this approach can target breast cancer cells while leaving healthy cells unscathed, it might someday help patients avoid the toxic side effects of standard cancer treatments.
"In the long term, I see this being used to treat people with metastatic breast cancer that has spread to the bones, ideally administered before radiotherapy and chemotherapy," Helen McCarthy, of Queen's School of Pharmacy, said in a statement.
Still, the group says that the approach needs additional preclinical testing and potential human trials are about five years away. The research was published Feb. 28 in the International Journal of Pharmaceutics.
The gene therapy field has been called things like "snake-bitten" over the years because the treatments have caused side effects in humans. Yet recently the field has been re-energized as scientists at firms like Cambridge, MA-based Bluebird Bio have made progress in providing the therapies safely.
- here's the Queen's University Belfast release