Gene therapy is an innovative approach to treating many different cancer types at the molecular level. Our scientists from ACGT have done years of careful research introducing genetic materials into genes to prevent or fight disease. Genes are responsible for every aspect of cell life: they hold the code for proteins that enable cells to grow, function and divide. When genes are not properly functioning or are missing or defective, the goal of gene therapy is to fix the problems at the source using cell and gene therapy.

What Makes a Gene Not Work Properly?

Scientists do not really know what makes a cell malfunction, causing harm rather than performing its natural function. However, with new scientific gene therapy improvements, we can replace abnormal or absent genes with healthy ones that enable cells to produce useful proteins.

How Does Gene Therapy Work?

Cell and gene therapies are revolutionizing cancer treatment by repairing, replacing, or destroying cancerous cells; activating our powerful natural immune systems; re-engineering cells to trigger killer cells to attack; injecting viruses directly into tumors to spark a quick response.

When a cell does not work properly or is absent, a genetically engineered gene is inserted into the cell. In order for proper functioning of the cell the gene needs to be inserted correctly into the target cell to avoid causing any adverse effects.

Genes are often delivered to cells by deactivated or specially modified viruses that target and destroy cancer cells while leaving normal cells unharmed.

Viruses function by delivering their genetic material into the host cell to create more of their virus in the body. The genetically engineered virus is created so that it cannot reproduce but it will use the gene’s DNA to deliver it directly to a cell that is not functioning. This treatment is being used for only certain groups of cells that need to be functioning correctly.

The Future of Gene Therapy

As of now, there are 3 approved gene therapies on the market.

Kymriah was approved in 2017 for pediatric relapsed and refractory acute lymphoblastic leukemia (ALL) and was approved in 2018 for adults with diffuse large B-cell lymphoma (DLBCL). Also in 2017, Yescarta, another CAR T cell therapy, was approved for non-Hodgkin’s lymphoma. Although not for cancer. Luxturna, which treats an inherited form of blindness, is the US approved gene therapy that targets a specific gene mutation.

We still have a long way to go to revolutionize treatments for all types of cancers and for all patients, but as one researcher has said, “we are now in the golden age of cancer gene therapy.”