What if you could transcend the current car-repair-like maintenance of your body and health – changing out parts damaged from age and/or wear – and heal yourself at the source, the genetic level?
Gene therapy is making medical headlines – but what is it and what can it do for us?
Gene therapy is the treatment of disease by replacing, altering, or supplementing a gene that is absent or abnormal and whose absence or abnormality is responsible for the disease. Gene therapy may use the genetic material, DNA, itself as the means of treatment.
Although still in its infancy, gene therapy is undergoing rigorous scientific scrutiny to treat and prevent disease. “Doctor Future” may be able to toss out drugs or surgery and opt for inserting a healing gene directly into the patient’s cells.
What exactly is a gene anyway? A gene is “a sequence of DNA that carries the instructions for making a sequence of RNA, which in turn instructs and assists in the creation of proteins.”
Proteins, in turn, “perform important tasks for the cell functions or serve as building blocks. The flow of information from the genes determines the protein composition and thereby the functions of the cell.”
Perhaps you’ve heard someone say, “My beautiful blue eyes are in my genes.” This is literally true. A parent or further ancestor had blue eyes and passed the chance of developing them along to younger family members who followed.
But along with the lovely traits come the less savory ones. The long list of diseases inherited from parental family members includes:
• Cardiovascular disease (CVD)
• Autoimmune disorders
• Psychiatric illnesses
• Alzheimer’s disease (dementia)
The US National Library of Medicine’s Genetic Home Reference Guide lists three scientific approaches to gene therapy:
• Replacing a mutated gene that causes disease with a healthy copy of the gene.
• Inactivating, or “knocking out,” a mutated gene that is functioning improperly.
• Introducing a new gene into the body to help fight a disease.
Nobody wants to hear their doctor say, in that sympathetic, “there, there” tone of voice, “You inherited your dad’s diabetes. Let’s outline your course of therapy.”
Fortunately, science fact is catching up to science fiction. Like any comic book hero with super strength or other enhanced physical attribute, modern gene therapy could be used not only to repair diseased cells, but to enhance normal ones.
Something happened last August 2017 that could change all our lives profoundly and forever.
Novartis AG got approval from the US Federal Drug Administration (FDA) for its CAR-T cell therapy. It is the first gene therapy available in the country.
According to MarketWatch, “CAR-T, or chimeric antigen receptor T-cell therapy, uses a patient’s immune T-cells and re-engineers them to better fight cancer.”
Sold as Kymriah, it is “a prescription cancer treatment used in patients up to 25 years old who have acute lymphoblastic leukemia (ALL) that has relapsed (went into remission, then came back) or refractory (did not go into remission with other leukemia treatments).”
MarketWatch indicates that “each dose of Kymriah is customized to the individual patient’s T-cells through genetic modification.”
Novartis tells us that “Kymriah is an innovative immunocellular therapy that is a one-time treatment” made from your own white blood cells.
Cancer immunotherapies use the body’s own immune system to fight cancer cells. Kymriah removes malignant cells, reengineers them, and then puts them back where they came from in the body to attack cancer cells. Pretty nifty, eh?
But the price is rather daunting: that single treatment will set you back a cool $475,000. So it’s not surprising that, according to Labiotech.eu, sales for the first American gene therapy are lagging behind investor expectations. Do you suppose that could that be due to the astronomical price tag?
Despite its promise to restore health and well-being, the National Center for Biotechnology Information (NCBI) is not impressed with the state of bio-cellular genetic engineering happening today:
“Gene therapy is an elegant concept crudely executed.”
Still, the ability to cure genetically inherited diseases on demand, using the body’s own genes, is a compelling endeavor. Keep your eyes on the health horizon and don’t be surprised to see the good ship Affordable Gene Therapy hove into view.