The FDA approved a new gene therapy to fight leukemia

During the past week, the American Food and Drug Administration's (FDA) unanimously approved the treatment of the patient to genetically alter its own cells to fight leukemia. If approved by the FDA, such treatment for the first time will be the culmination of the age of "living medication". Thus, we will pursue technology to strengthen our natural immune system and improve its ability to suppress previously invasive diseases.

This leukemia treatment, known as CTL019, is the first gene therapy to reach the pazara, but many are waiting. Treatments for an aggressive type of brain tumor, myeloma and other types of leukemia are being developed.

This technique is a real example of a personalized medicine: a unique type of treatment for each patient created from their own cells. After the cells are taken by the medical staff, they are sent to Novartis, which is freezing and practicing the therapy. After being treated, it is frozen again and sent back to the medical center.

If treatment is approved, it will be a first for the FDA. In light of the surprising results of the experiment, the approval of the treatment is, frankly, not surprising. The withdrawal observed after a single dose of gene therapy for patients with death, as all other treatments have been tested, is proving that treatment will cure over time. Indeed, the FDA Panel has approved the approval of treatment for recurrent or treatment-resistant B-cell lymphoblastic leukemia in children and young adults aged 3-25 years.

Access to treatment

One of the participants of the Mission Meeting was Emily Whitehead, 12 years old, from the first patients in the experiments. The leukemia, thought to be deadly, was being treated at the age of six while he was about to lose his life. She has survived cancer since then. His father, Tom Whitehead, told The New York Times that when this treatment is approved, it will save thousands of children across the globe. I hope someday you guys in the advisory committee will have generations of families; You can tell that you are part of a process that ends toxic treatments such as standardized treatment chemotherapy and radiation and transforms blood cancers into a treatable disease that people survive, but they are still alive, "he

Now researchers at the University of Pennsylvania have developed Novartis-licensed treatment. However, the disease for which treatment is sought is rare and affects about 5,000 individuals per year. 60% of them are young adults and children. Although standard treatments cure most children, 15% of patients like Emily do not respond to treatment or the disease is recurring.

Novartis will initially limit the use of CTL019 because of the complexity of the treatment regimen and the need for special care for side effects. For this reason, approximately 30-35 treatment centers can be reached after they have been applied to the market. In addition, according to the New York Times, staff in these centers will receive special training and approval to implement treatment. Although the amount of such unprecedented treatment is predicted to exceed $ 300,000, the Novartis Authority refuses to respond with a definite amount to the question of the New York Times.