Leukemia : experts call for access to a gene therapy

The U. S. Food and Drug Administration/Flickr

Published the 13.07.2017 at 11h53

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Keywords :

leucémiethérapie géniqueEtats united States

The ESSENTIAL

Acute lymphoblastic leukemia is a cancer of the blood and bone marrow that affects blood stem cells.
In 2012, 810 new cases of ALL were diagnosed in France.
44 % of diagnoses were in children under 15 years of age.
In 70 to 90 % of cases, a complete remission is obtained.
But 15% of the cases of LAL are resistant to treatment.
Gene therapy CTL019 teaches the immune system to recognize cells that fail and destroy them.

A first in the world prepares for the United States. Gene therapy may soon be authorized by the Agency to the u.s. food and drug administration (FDA). Designed to treat the acute lymphoblastic leukemia (ALL), it modifies the action of the t lymphocytes.

An advisory committee of experts concluded in favour of the placing on the market of this innovative approach. Any lifting of the reservations on the long-term side effects of such support.

If this opinion is followed by the health authorities, this would be the first time that such a strategy would be permitted in the country. The gene therapy in question, CTL019, which was developed by the university of Pennsylvania (Usa) and purchased by the swiss laboratory Novartis, which now owns the patent.

29 patients in remission

The treatment of acute lymphoblastic leukemia is based on the technique of CAR-T cells : the T lymphocytes of the patient are extracted before being re-programmed genetically in the laboratory, using a HIV inactivated.

Once the material of the cells changed, they are fed back to patients. They then multiply and gradually replace the cell not treated. At the same time, these white blood cells improved in attack the B cells faulty which are at the origin of this cancer of the blood and bone marrow.

In practice, the results delivered by the laboratory are rather conclusive. Of the 63 patients treated between 2015 and 2016, 52 have reached the stage of remission. The others are deceased. At the end of November, 29 people were still in remission. 11 have experienced a relapse.