Conventional methods for treating diseases, including the 200 or so cancer varieties that are known to science, normally involve taking drugs or undergoing treatments that do what your own body cannot. Gene therapy has come along and, despite being a nascent medical field, has changed everything.
Through a range of different methods, the genes of the patient’s own cells are edited with breath-taking precision using synthetic viruses or CRISPR; these augmented cells are then reintroduced back into their bodies and their immune systems do the rest of the work. Trialled all over the world, this type of therapy is shown to cure children of seemingly incurable blood cancer, nullify someone’s sickle cell disease, and more.
Take, for example, the recent story of the two young children with incurable acute lymphocytic leukemia. After being given a novel type of gene therapy, one that used cells from healthy donors, their aggressive cancers disappeared. In fact, it was so effective that it suggested that a universal therapy for this type of cancer - one that could be used on older children, adults and everybody else - may not be as far off as some have previously imagined.
Although there are still questions about potentially dangerous long-term effects of this soon-to-be-approved procedure, this decision is a landmark one, opening up a brand new world in the fight against cancer, and giving hope to thousands of children around the world.
Up until this point, gene therapy like this has remained experimental, meaning that it was only available to people taking part in a clinical trial. As reported by The New York Times, however, this is to no longer be the case: the Food and Drug Administration (FDA) is about to officially give the go-ahead for gene therapy to be put on the market for the very first time.
In this case, the therapy will be used to treat people suffering from an aggressive type of blood cancer named B-cell acute lymphoblastic leukemia. It will be made available to children and young adults aged between three and 25 who have been temporarily cured of the disease only to find that it has returned with a vengeance.
The treatment will be patient specific: Their own cells will be shipped off to be altered, frozen, then sent back to be reintroduced into the patient at a later date.
Gene therapy treatment has been used on a range of children across the globe to cure them of this form of leukemia. Although they suffered from quite severe side effects, they were not life-changing and, most importantly, these kids are cancer-free years on.
The family of the first child to have ever been treated in this way, Emily Whitehead, were present at the FDA panel discussing whether or not to authorize the treatment for mainstream medical use. Apart from individual testimonies, a key study showing that this method pushes 82.5 percent of patients into remission was also considered.
The panel was apparently convinced that this gene therapy works where other treatments fail and that thousands of children’s lives will be saved if it’s approved, so they unanimously recommended that it should be available. It’s now certain that the FDA chiefs will green light the treatment.