By: Janet Yan
The cure to cancer. It is oftentimes thought of as the highest achievement in the medical field. And this year, we’re one step closer.
Pediatric acute lymphoblastic leukemia (ALL) is a blood cancer that affects white blood cells known as lymphocytes, causing them to multiply boundlessly and aggregate in the liver, spleen, and lymph nodes. Every year in the United States, approximately 3,100 patients under age 20 are diagnosed with ALL, and most of these cases are in children aged 3 to 5. Although 90% of children with ALL can be cured, many are unable to respond to standard treatments, which include chemotherapy, stem cell transplant, and radiation therapy.
In August 2017, the Food and Drug Administration (FDA) announced the approval of a new form of immunotherapy, a form of therapy that utilizes the body’s immune system to fight cancer, for the indication of ALL in patients aged 25 or below who have failed to respond to treatments or have relapsed. Known as Kymriah, the treatment involves taking T-cells from the patient’s own immune system, genetically modifying and programming them to fight cancer cells, and then re-introducing them into the patient. Developed by the University of Pennsylvania in collaboration with Novartis, a global pharmaceutical company, the treatment is the “first therapy based on gene transfer approved by the FDA.” A clinical trial studying the effectiveness of the Kymriah therapy showed impressive results; 52 of the 63 (83%) patients evaluated for efficacy achieved remission within three months.
While Kymriah seems very promising, the trial also shed light on possible adverse reactions, or side effects, such as cytokine release syndrome, a potentially deadly immune reaction. Other adverse reactions that patients experienced during the trial included, but were not limited to, fatigue, headaches, hypotension, nausea, and vomiting. Additionally, the one-time treatment has a rather outrageous price tag of $475,000.
Nevertheless, the development of Kymriah is an exciting medical achievement, and its approval is an equally significant step towards adopting new treatments and technologies. It also brings up interesting ethical questions regarding genetic modification and its use in medical treatments or beyond.
Acute Lymphoblastic Leukemia (ALL). St. Jude Children’s Research Hospital. Retrieved fromhttps://www.stjude.org/disease/acute-lymphoblastic-leukemia-all.html.
Novartis receives first ever FDA approval for a CAR-T cell therapy, Kymriah™ (CTL019), for children and young adults with B-cell ALL that is refractory or has relapsed at least twice. (2017, Aug 30). Novartis. Retrieved from https://www.novartis.com/news/media-releases/novartis-receives-first-ever-fda-approval-car-t-cell-therapy-kymriahtm-ctl019.
Stein, R. (2017, Aug 30). FDA Approves First Gene Therapy for Leukemia. NPR. Retrieved fromhttp://www.npr.org/sections/health-shots/2017/08/30/547293551/fda-approves-first-gene-therapy-treatment-for-cancer.