Children and adolescents with leukemia have access to CAR T treatment

Children and adolescents with leukemia have access to CAR T treatment


Children and adolescents with B cell acute lymphoblastic leukemia (ALL) who do not respond to the intensive first line treatment are now given access to CAR T treatment Kymriah (tisagenlecleucel).
This happens after the Medical Council on Wednesday decided to recommend Kymriah as a possible standard treatment for ALL children who are either refractory, relapsed after stem cell treatment or later relapse.

The treatment has the potential to cure up to two-thirds of the patients, which is why the Medical Council estimates the clinical added value to be great.

Until now, there has not been a standard treatment for the refractory ALL patients, which has meant that the treatment has very often been experimental, and the chairman of the Patient Association for Lymphoma, Leukemia and MDS, Rita O. Christensen, is pleased with the Medical Council’s recommendation. .

“It is a really good news for this group of patients with acute leukemia, because there has been a great deal of mortality, and we expect patients to have a longer life with this treatment. The mortality rate has been over 50 percent among refractory patients, and if you can increase the survival rate to 80 percent, this is a really good news, ”says Rita O. Christensen.

The Medical Council estimates the cost of the treatment to be very high, but when considering the severity principle, the cost-to-large clinical value-added ratio is reasonable, the Medical Council believes, which at the same time recommends systematic collection of relevant effects and effects. adverse reaction data for the ALL patients treated with Kymriah and after two years, the Medical Council will decide whether the recommendation should continue to apply.

Kymriah is indicated for the treatment of children and adolescents up to 25 years of age with B cell acute lymphocytic leukemia (ALL) who are refractory, in relapse following stem cell transplantation or in other or later relapses.

Treatment is given as a single intravenous infusion and the recommended dose for patients under 50 kg is between 0.2 × 106 and 5 x 106 viable CAR T cells / kg body weight and for patients over 50 kg between 0.1 × 108 and 2.5 x 10 8 viable CAR T cells / kg body weight.

Before treatment with Kymriah, the patient is treated with lymphocyte-depleting chemotherapy.

What is CAR T-cell therapy?

CAR (chimeric antigen receptor) T-cell therapy is a promising gene therapy that uses modified versions of a patient’s own blood cells to target and destroy cancer cells. It involves three types of innovative technologies:

Cellular therapy – using the patient’s own cells as therapy
Gene therapy – inserting genes into a patient’s cells, thereby causing these cells to produce a new therapeutic protein
Immunotherapy – harnessing the patient’s own immune system to treat his/her disease
How does it work?

A process called leukapheresis is used to collect the patient’s own T-cells intravenously. These cells are genetically engineered to target a protein called CD19. The new therapeutic cells are then reinfused to the patient via an intravenous line. Once returned to patient’s body, the CAR T-cells target and kill the leukemia cells that express CD19.

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