01 February 2018

Leukemia treatment: good news

An international multicenter study was organized and conducted to evaluate the efficacy and safety of CAR T-cell therapy using tisagenlecleucel.

The patient's own leukocytes are used for therapy with tisagenlekleisel, which are reprogrammed in the laboratory and injected back into the blood. The altered cells find and destroy the tumor.

In August 2017, the method was approved for the second phase of clinical trials for the treatment of acute lymphoblastic leukemia (ALL), the most common malignant tumor in children.

The study was conducted on the basis of several clinics in the USA, Canada, Europe, Japan and Australia. In total, 25 clinics from 11 countries of the world were involved. It was supported by the pharmaceutical company Novartis.

The study involved 75 patients aged 3 to 21 years with recurrent or refractory forms of B-cell ALL. 61% of them had a relapse after allogeneic hematopoietic stem cell transplantation (donor bone marrow transplantation), a therapy method that is currently the latest in the arsenal of oncologists.

According to the authors of the study, often when bone marrow transplantation is ineffective, patients are sent to a hospice, where they receive palliative treatment. Now there is another way out: to use CAR T-cell therapy, achieve remission and send the patient home.

Three-month therapy with tisagenlekleisel led to remission of ALL in 81% of patients. The overall survival rate for 6 months was 90%, for 12 months – 76%. The median duration of remission was not achieved, tisagenlekleysel remained in the blood for up to 20 months.

For comparison, the article presents the results of a study of another FDA-approved treatment method: 20% of patients with recurrent or refractory forms of ALL responded to therapy, the average survival time was 13 weeks.

Side effects of tisagenlekleisel therapy were noted on average in 73% of patients. Among the main ones were cytokine release syndrome in 77%, which often required hospitalization in the intensive care unit and the appointment of tocilizumab (48%), and moderate neurological disorders in 40% of patients.

CAR T-cell therapy is a hope for young patients suffering from ALL, especially in conditions of inefficiency of traditional methods of treatment.

Article by Shannon L. Maude et al. Tisagenlecleucel in Children and Young Adults with B-Cell Lymphoblastic Leukemia is published in the New England Journal of Medicine.

Aminat Adzhieva, portal "Eternal Youth" http://vechnayamolodost.ru based on EurekAlert: Landmark international study: CAR T-cell therapy safe and effective in youth with leukemia.


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