Gene therapy, experimental therapeutic option

If someone sells you gene therapy as a proven treatment against diseases, including cancer, without telling you that it is an experimental treatment, be wary because gene therapy is still essentially an experimental therapeutic option. This is the conclusion of two Brazilian researchers (Giulliana Goncalves and Raquel Paiva) at Hospital Israelita Albert Einstein of Sao Paulo, who reviewed the latest advances in gene therapy.

There are two primary approaches in gene therapy. First is the correction of mutated genes which cause genetic disorders. Second is specific modification of target sites to increase accessibility to treatment.

The most widely studied technique today is the so-called “recombinant DNA technology” wherein a therapeutic gene is inserted into a “carrier” (e.g. virus, plasmid or nanostructure), which has a known capability to invade human cells and introduce the genetic material. The most commonly tested carrier is a virus, because it is a highly efficient mechanism in invading human cells.

The challenges today involve the acquisition of reliable and dependable information on the specific cells that will be targeted to determine their accessibility, on the capability of these cells to distribute the therapeutic gene materials, and on the clear mechanism of genetic bonding between therapeutic cells and host cells. It is in these areas that current research efforts had to be poured.

These target cells can be the stem cells or somatic ( bodily) cells. In stem cell gene therapy, the genetic modifications made are inherited in the bloodline, while in somatic gene therapy, these changes stay in the target cells and are not inherited. However, both approaches can be combined, such as in the treatment of hepatitis B or C wherein induced pluripotent stem cells are used to increase liver cell multiplication, while reinfection resistant genes are added through a virus.

Nonetheless, it was Wallace Stegner who was attributed for saying, “If you avoid the killer diseases and keep the degenerative ones under control with sensible diet and exercise and whatever chemotherapy you need to stay in balance, you can live nearly forever.” I hope the reality of mortality can be that hopeful, though.