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Health, Scientific progress

13 - 11 - 2015

Leukemia: the success of cell therapy

Thanks to an injection of genetically modified cells, the small one-year-old Layla Richards, is in remission from her leukemia. This is a world first. However, to reach this stage, animal tests were carried out preliminarily. 


Layla Richards




Cell therapy involves transplanting human cells to treat disease or repair damaged tissue. “The goal is to sustainably treat the patients with a single injection of therapeutic cells,” explain the INSERM researchers. This treatment was developed in animals, before it was given to humans.


Thus, the small Layla, suffering from an aggressive form of leukemia, was given genetically modified T lymphocytes (white blood cells). Derived from a healthy donor, these immune cells had been “reprogrammed” to detect and destroy cancerous cells. 


Hope against hemophilia?


This reprogramming technique, dubbed “gene editing”, alters the DNA within cells and has many applications in research.


The scientific community is also interested in other blood diseases like hemophilia, an inherited disorder characterized by the absence of blood clotting. Scientists have managed to “reprogram” monkey liver cells so they develop the capacity to produce a coagulation factor.


For more information on gene and cell therapy: hope is in the cells - c’est pas sorcier