
The Expert Council of the American Directorate for Control of food and medicine (FDA) unanimously approved the access to the genetic therapy of acute lymphobiasa leukemia in children and young adults. This does not mean that such treatment becomes available to everyone, but the final resolution of the FDA company Novartis will most likely receive on October 3, 2017. If this happens, then such therapy will become the first of its kind approved FDA.
The essence of the therapy, called the CTL019, is that the patient takes his own T cells (which make up part of the immune system), freeze, they are taken to the pharmaceutical company factory, with the help of a specially modified human immunodeficiency virus, a gene is placed in them, helping to kill malignant cells (this may take several weeks), they are again frozen, freezing again, freezing, freezing, freezing, freezing again, freezing They are taken to the clinic and introduced back to the patient. It is assumed that initially such treatment will become available only in 30–35 American hospitals. Specially trained personnel will work there, including employees of the company. Over time, according to representatives of Novartis, CTL019 will be introduced in other countries.
In studies, genetic therapy showed promising results: 29 patients since 2015 or 2016 have no signs of the disease, 11 people have a relapse, 11 underwent additional treatment.
Therapy cannot be called completely safe: in some patients it causes flu -like symptoms (sometimes serious enough) and convulsive attacks. Therefore, such therapy is planned to be used when standard treatment does not work, and therefore such side effects are considered justified.
The company does not disclose the future cost of therapy, but it is assumed that it will be about 300 thousand dollars. For the treatment of leukemia in the United States, such amounts are quite common.
In addition to novartis, several pharmaceutical companies are developing such therapy at once, which also plan to get the approval of the FDA soon. This treatment method is intended to use for other oncological diseases.