The FDA has authorized the use of gene therapy against an aggressive form of leukemia.
This Wednesday, the Food and Drug Administration (FDA), the American equivalent of the ANSM, approved the first gene therapy against cancer. This is Kymriah, which was developed by Novartis laboratories to fight against acute lymphoblastic leukemia in children and young adults, up to 25 years old.
“There was an urgent need for innovative therapeutic options that could improve the chances of survival for patients suffering from relapses or resistance, and for whom the prognosis is poor, explains Novartis in a press release. Patients often undergo multiple treatments including chemotherapy, radiotherapy, targeted or stem cell therapies, but only 10% are still alive 5 years later. »
reprogrammed cells
What Kymriah offers is a one-time treatment, with an 83% remission rate within three months of treatment. It is individualized. The T lymphocytes, cells of the patient’s immune system, are removed, reprogrammed, cultured and frozen. They are modified to attack other failing, disease-causing, rapidly proliferating lymphocytes.
They spot a protein, CD19, which is expressed on the surface of these cells, and attack them. Once injected back into the patient’s body, the modified T cells replicate rapidly, and remission is rapid.
The era of gene immunotherapies
“We are entering a new era of medical innovation, in which we are now able to reprogram a patient’s cells to attack deadly cancer themselves,” said FDA Commissioner Scott. Gottlieb. New gene and cell therapies hold the potential to transform medicine and create an inflection point in our ability to treat, and even cure, many as yet incurable diseases. »
More than 550 experimental gene therapies are believed to be in development, he added. By the end of 2017, the FDA should validate a second, designed by Kite Pharma, intended to treat diffuse large B-cell non-Hodgkin’s lymphoma.
An expensive treatment
Two shadows on the board. Side effects can be severe. In addition to the classic effects – fever, nausea, vomiting, intestinal disorders, muscle and joint pain, etc. – Kymriah can cause serious infections, low blood pressure, kidney problems and a drop in blood oxygenation.
Then its price. The cost of the treatment should be around 400,000 euros. An argument that Novartis brushes aside. Conventional treatments for leukemia, which include a bone marrow transplant, cost more than 450,000 euros, and can climb up to 675,000 euros, the Swiss laboratory justifies itself.
Acute lymphoblastic leukemia is the most common type of leukemia in children. It is neither contagious, nor transmissible, nor hereditary, and the causes are still poorly understood. In France, about 300 cases are recorded each year.
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