By Ana Chrysa Maravelias
According to the World Health Organization, cancer is the second leading cause of death globally. For years, people thought that it could not be cured completely without surgery, but could be monitored and reduced. Although the first use of gene therapy on a patient was on September 14 of 1990, on August 30, 2017, gene cancer treatment created by the pharmaceutical company Novartis, was approved by the Food and Drug Administration (FDA) for treating Acute Lymphoblastic Leukemia (also known as ALL). Gene cancer therapy is a breakthrough creation, however, it still has a long way to come.
We all have genes. We are created out of them. They are passed on from one generation to the next and are what determine if we have red hair or blue eyes or are average in stature. Genes are made up of DNA. They are located within the nucleus of the cell, which is the cell’s ‘control center.’ Clusters of genes make up chromosomes – half of which we acquire from our maternal side, and the other half from our paternal. All in all, genes make us, us. Cancer cells are not normal cells however. They have faults which cause the genes in them to split too often, creating and growing tumors. When the idea of using our genes – the structures of our being – to cure cancer, it was revolutionary.
Cancer is a big problem. As of 2018 an approximated 9.8 million people died as a result of it. An estimated one out of every six deaths worldwide is due to cancer. These startling statistics fueled a drive for battling it. Gene therapy uses patients’ own genes to combat diseases that they have. Gene therapy has been clinically approved for some cancers, however, it is still experimental when considering the treatment of other types of diseases.
The gene therapy created by Novartis will be sold under the name Kymriah. It was created to specifically target and kill the cancer cells that are associated with ALL – a childhood cancer that is often deadly. This kind of gene therapy is called CAR-T which specifically uses patients’ T cells, which are a kind of immune cells. These cells are distilled from patients and are changed to include a protein which sends the T cells towards the leukemia cells to kill them. This protein is known as Chimeric Antigen Receptor (CAR). These altered cells are then given back to the patient via a shot. When this treatment was tested on 63 kids, 79% of the children tested survived.
Although the results of this treatment are quite amazing, there are a variety of setbacks. First, the cost is quite great – $475,000 for one treatment. This amount is a lot for many families especially ones that are already burdened with hospital bills. Also, as of the summer of 2017, Novartis had only one location where these genes were altered. This means that patients had to send there genes to a lab (in New Jersey) and had to wait for an average of twenty two days for the samples to be altered. This is risky, because that period of time could be enough for patients to pass away. There are also possible fatal symptoms as a result of using this therapy. These include neurological problems, as well as an overproduction of immune cells resulting in something called Cytokine Release Syndrome. There is also a possibility of encountering Cerebral Edema which is when the brain swells. Nearly none of these symptoms have arisen in patients after receiving this therapy however, which is promising.
Despite some room for controversy, overall, this fairly new gene therapy treatment is auspicious. As the commissioner of the FDA, Scott Gottlieb said “We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer.”