Cancer is one of the utmost debilitating and deadly epidemics of our time. Our ability to treat and/or cure certain types of cancer has significantly improved over the years; however, there remains room for better, more effective methods. The recent approval of gene therapy for leukemia may be an incredible breakthrough in how we will soon, and more successfully, fight the disease.
What is Gene Therapy?
Until recently, gene therapy was only in an experimental phase. The technique uses a patient’s own genes and “teaches” cells how to combat a disorder. It’s a part of the field of immunotherapy, which has garnered successful results—without utilizing surgery or drugs.
There are different ways that gene therapy can be implemented. For one, a mutated gene that causes disease can be replaced with a healthy copy.
Another way the therapy can be utilized is by de-activating the mutated gene that is causing the disease to spread. One other technique is to introduce a totally new gene into the body to help fight the disease.
Approval of Therapy for Leukemia
Many labs around the country are working on perfecting a multitude of gene-therapy studies in hopes of having them approved for clinical use. (According to the FDA commissioner there are over 500 types of gene-therapy studies currently.) Recently, however, the Food and Drug Administration (FDA) approved a therapy called Kymriah. The FDA granted Novartis (the maker of Kymriah) approval for doctors to use this technique on patients with leukemia—making it the first-ever, gene therapy treatment for cancer.
This particular therapy is individualized and customized. T-cells (a form of white blood cells) are removed from the patient’s bloodstream. They are frozen and shipped to the Novartis lab for genetic engineering. They are then multiplied, refrozen, and sent back to the patient’s medical facility.
Once returned to the doctor, the newfangled cells are dripped back into the patient’s bloodstream. The cells have been revamped to recognize the cancer and attack it. Kymriah has been approved for young adults and children with an aggressive form of leukemia, which until now, has been barely treatable.
The Great White-Cell Hope
Gene therapy is expected to transform the way the world has treated cancer and other diseases. Altering a person’s own cells to fight off mutated cells is transformative. Although it’s incredibly hopeful for scientific treatment/curing of cancer, it does not come without its own risks—and costs.
Kymriah will be implemented one time per patient. That comes at a ticket price of $475,000. Novartis representatives pointed out that bone-marrow transplants (a current treatment for leukemia) can cost upwards to $800,000. Novartis also offered that if a patient does not respond to Kymriah within 30 days, they would eliminate the charge.
Another consideration for this type of treatment is that doctors and others in medical facilities need thorough training on the technique. The FDA is actually requiring that hospitals and doctors get certified and that facilities carry particular drugs that combat severe reactions.
Adverse reactions can occur from removal of the T-cells to the replacement of the refurbished cells. Some of them are: dangerously low pressure levels, high fever, lung congestion, and neurological complications.
The Light in Gene Therapy
Even with the risk of life-threatening side effects, gene-therapy can still provide a treatment to save an already ailing life. Gene therapy may also possibly provide a cure. As the FDA, over time, continues to approve more genetically-based treatments, we may actually see a decrease in deaths from diseases such as cancer. As for the present, we can be encouraged that the first gene-therapy has been approved and is now available in the U.S.
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