Gene therapy will be used to treat blood cancer
Last reviewed: 23.04.2024
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The US Food and Drug Administration has recommended the use of gene therapy in the treatment of leukemia.
Therapy consists in making changes in the genetic cell code of the patient.
Genetically modified lymphocyte cells existing in the body are used as a "living medicine", since they direct immunity to fight leukemia, most often provoking a prolonged and stable period of remission.
Experts need to wait for a positive response from experts. It is assumed that with the planned development of events, gene therapy can be applied already this year.
Represent the new method will be the pharmaceutical corporation Novartis, and developed its scientists from the University of Pennsylvania.
Gene therapy will be used to treat patients with B-cell acute lymphoblastic leukemia. Scientists are sure that the treatment will be relevant for patients with multiple myeloma, separate aggressive varieties of cancer brain tumors and other tumors.
The essence of therapy is that the T-lymphocytes of the patient are modified genetically, tuning them against malignant cells. To interfere with lymphocyte DNA, a neutralized virus based on HIV is used. The virus moves the desired gene into the cell structures, without provoking the development of the disease.
Further, the modified T-lymphocytes are sent to search and destroy cancer cells. They manage to detect them, thanks to the CD19 marker.
Of course, everything described above is a theoretical side. In practice, everything looks easier. Take a small amount of blood from the vein of the patient, subject it to freezing and sent to a transformation. Then the cells are changed and the blood is taken back to the patient's vein. Experiments have shown that this procedure resulted in sustained long-term remission even after a single administration. Individual patients had complete cure.
In clinical testing of the new method, patients suffering from leukemia participated. Their age was different - from three to 25 years. Before the experiment, their body either did not react to chemotherapy, or there were repeated exacerbations.
One of the first positive results was recorded in a six-year-old girl. In 2012, she was given gene therapy, and as a result, the girl was completely cured. Years have passed, and her analyzes are still good.
According to the authors, over the past few years they have managed to improve the method. Thanks to this, it was possible to minimize the risk of side effects: earlier after the injection, symptoms such as fever, pulmonary congestion, hypotension could have been observed.
In the new test, which was conducted two years ago, 63 patients participated. As a result, 52 of them completely disappeared manifestations of the disease. Eleven people, unfortunately, could not be saved. For surviving and cured patients, scientists plan to observe for another fifteen years.
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