There is a breakthrough on the battlefield that is leukemia treatment, and it goes by the name Kymriah, a gene altering therapy created by Novartis. Last August 30th, 2017, the Food and Drug Administration (FDA) approved the first ever CAR-T cell therapy for children and young adults under 25 who are suffering from an acute lymphoblastic leukemia (ALL) in the United States.
CAR-T sheds light on a revolutionary approach to treating cancer cells for the field experts and medical practitioners, as each dose is customized for every patient. Unlike pills, radiation and chemotherapy which weakens the natural defenses of a human body, this particular treatment gathers the T-cells and white blood cells of a patient (the immune cells) and trains them to have the ability to recognize and later on fight cancer cells.
Through a special blood filtration process, these immune cells are removed from a patient’s body, brought to a laboratory, and encoded genetically to include a new gene which contains a unique and special kind of protein (a chimeric antigen receptor or CAR) to help identify cancer cells. These re-engineered T-cells will then be transfused back to the patient where they can soon after begin fighting cancer cells. This tedious process takes 22 days, according to Novartis.
The Price Tag for Being Well
Kymriah, which is to be conducted to a patient one time, costs $475,000 — the fee only covering the role of Novartis in the treatment and excluding other expenses such as travel, hospitalization, and any additional drugs needed for the side effects. The creators reiterate that this is a bargain already, as the Wall Street analysts projected the price of the treatment between $600,000 and $750,000, similar to a stem cell transplant treatment.
Given that the patients are below 25 years of age, they are likely to be covered by the health insurance of their parents or get aid from the government-sponsored health plan, Medicare. It was said that Novartis is currently in discussion with the government in an aim to create a system that will only require payment for patients who responded well to the treatment within the span of one month.
Side Effects of the Kymriah Treatment
To test the safety and effectiveness of the treatment, Kymriah was demonstrated to 63 patients in a multicenter clinical trial. The remission rate in three months was 83 percent. Side effects, however, are inevitable. One of the most commonly identified side effects was a severe inflammation called cytokine release syndrome, an adverse reaction to CAR-T. Experts said that it might be fatal in some patients but can generally be controlled with immunosuppressant drugs.
Furthermore, during the treatment, patients may experience high fever, flu-like symptoms, and neurological events, the latter of which was said to be life threatening. In addition to these, the treatment may also cause serious infections, low blood pressure (hypotension), acute kidney injury, fever, and decreased oxygen (hypoxia) — all of which might appear within the first 22 days after the patient is infused with Kymriah.
Due to the identified risks of the treatment, Kymriah was approved by the FDA with a Risk Evaluation and Mitigation Strategy (REMS), with the inclusion of elements to assure safe use (ETASU). REMS specifically states that the patients should be informed of the possible side effects of the treatments and to emphasized that they should return to a medical facility should they develop any of the said adverse reactions.
Moreover, hospitals and clinics which offer the treatment are required to have special certifications which have to include mandatory training for the team of people who are directly involved in the administering, dispensing, and prescription of Kymriah. Furthermore, to ensure that the treatment is only given to the right patients, medical establishments are directed to have a strict set of procedures such as securing the availability of tocilizumab (an immunosuppressive drug), before the administration of the treatment. This will make it easier for the health professionals to subdue severe reactions should there be any.