The Future of Cancer Treatment: FDA Approved
Cancer immunotherapy, a process that involves genetically modifying a patient’s own immune cells to fight cancer, has received its most rewarding breakthrough to date. This past summer, Novartis’ CDL019 CAR T-cell therapy, coined KymriahTM, was unanimously approved by the FDA to treat B-cell Acute Lymphoblastic Leukemia (ALL) in children and adolescents ages three to 25.
This treatment is life-changing for many in the patient population it targets — those with refractory or relapsed ALL. After exhausting front-line options, including chemotherapy, radiation, and stem cell transplants, these refractory or relapsed patients have a five-year disease-free survival of less than 10 to 30 percent. Optimistically, Novartis’ KymriahTM therapy demonstrated a promising 83 percent overall remission rate in the clinic, saving and extending the lives of patients with such limited options.
However, the use of CAR-T cells hasn’t come without risks. Once the patient’s T cells are collected and engineered to express a cancer-specific chimeric antigen receptor (CAR) on their surfaces, the T cells are reintroduced into the patient’s body with the ability to recognize and kill cancer cells with specificity. This reintroduction stimulates the immune system and can cause it to become overactive, toxically increasing secretion of proteins called cytokines that can cause high fevers, organ damage, and potentially death. Of course, this is a potential risk with all immunotherapy approaches, and symptom management has become a priority within this field.
Perhaps the most significant decision in this FDA approval is that this therapy involves genetically modifying immune cells using a viral vector whose long-term effects are unknown. There is cause for concern from researchers that the integration of vector DNA into the body may eventually become mutagenic and thus potentially create harmful and cancerous T cells. However, the unanimous FDA approval set a precedent that paves the way for more gene therapies that are set back by this potential risk to gain approval.
Despite the risks and challenges of cancer immunotherapy, Novartis has demonstrated what effective treatments can look like for patients without many options. All eyes will be on this therapy as it becomes an FDA-approved drug, and hopefully more approvals will follow in the life-saving field of research that is cancer immunology.
DOI: 10.1097
