By Linda Cako
Kymriah is the first CAR-T cell therapy to be approved by the FDA. It was approved on August 30, 2017, and has been making waves since. The reason why is because up until now, doctors needed a more personalized treatment for leukaemia. Leukemia is the most common childhood cancer, making up 30% of all childhood cancers in America (American Cancer Society, 2016), so an effective treatment was in demand. Acute cancers are cancers that progress very quickly, usually within a few months. Unfortunately, this means that they are often diagnosed in the later stages. Cancers of Acute Lymphoblastic Leukaemia (ALL) are also very aggressive. They exhaust treatments quite rapidly, pushing researchers to develop more methods for targeting cancer. Now that Kymriah has been approved, these aggressive cancers can be treated with more precision for the patient.
It will be used on patients who have relapsed or refractory ALL. This type of treatment will also only be used on patients up to 25 years of age. The company did not specify why this age limit is placed.
Kymriah uses CAR-T cell therapy to target ALL. It does this by, in a way, “boosting” the immune system. It is a type of immunocellular therapy which starts by harvesting the patient’s own T cells. They are filtered from the blood, and then they are modified to target a particular antigen expressed in the patient’s cancer cells. They are modified by having a vector (usually an adenovirus) inject the genetic material into the cells. Then they are grown in-vitro and injected back into the patient. Within weeks the patient’s cancer begins to go into remission.
Using the patient’s own T cells significantly reduces the chances of them from suffering Graft vs Host disease and provides a reliable method for treatment. Previously patients would receive bone marrow transplants, but they were risky. Patients, sometimes, would have to be on a [restrict]waitlist for months, or years, to receive a bone marrow that their body would be likely to reject.
Although Kymriah sounds like a wonder drug, it has shown to cause severe to life-threatening side effects. The most common side effects are Cytokine Release Syndrome (CRS) and Neurological Toxicities. CRS is when the immune system is releasing too many cytokines into the blood resulting in a more severe immune response, which triggers the release of even more cytokines. This results in very low blood pressure, high fever, difficulty in breathing, severe nausea, vomiting, joint pain, etc.
While this therapy is revolutionizing how we can personalize cancer treatments, it is far from perfect. For starters, this drug costs $475,000. Not everyone can afford to pay this kind of money for a therapy that is supposed to be used when other therapies have already been paid for and failed. Due to the age limit, this drug is not even available for people over 25 so it is pushing researchers to develop more treatments for those who do not have access.[/restrict]