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Gene therapy will be used to treat blood cancers
Last reviewed: 02.07.2025
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The U.S. Food and Drug Administration has issued a recommendation regarding the use of gene therapy in the treatment of leukemia.
The therapy consists of making changes to the patient's genetic cellular code.
Genetically modified lymphocyte cells present in the body are used as a “living medicine” because they direct the immune system to fight leukemia, most often causing a long and stable period of remission.
Specialists can only wait for a positive response from experts. It is assumed that, if events develop as planned, gene therapy will be used this year.
The new method will be presented by the pharmaceutical corporation Novartis, and it was developed by scientists from the University of Pennsylvania.
Gene therapy will be used to treat patients with B-cell acute lymphoblastic leukemia. Scientists are confident that the treatment will be relevant for patients with multiple myeloma, certain aggressive types of cancerous brain tumors and other neoplasms.
The essence of the therapy is that the patient's T-lymphocytes are genetically modified, setting them against malignant cells. A neutralized virus based on HIV is used to interfere with lymphocyte DNA. The virus moves the desired gene into cellular structures without causing the development of the disease.
The modified T-lymphocytes are then sent to find and destroy cancer cells. They are able to detect them thanks to the CD19 marker.
Of course, everything described above is the theoretical side. In practice, everything looks simpler. A small amount of blood is taken from the patient's vein, frozen and sent for transformation. Then the cells are changed and the blood is returned to the patient's vein. Experiments have shown that such a procedure led to a stable long-term remission even after a single injection. Some patients experienced complete recovery.
Patients suffering from leukemia took part in the clinical testing of the new method. Their ages varied from three to 25 years. Before the experiment, their bodies either did not respond to chemotherapy or had repeated exacerbations.
One of the first positive results was recorded in a six-year-old girl. Back in 2012, she underwent gene therapy, and as a result, the girl was completely cured. Years have passed, and her tests are still good.
According to the authors, they have managed to improve the method over the past few years. This has allowed them to minimize the risk of side effects: previously, symptoms such as fever, pulmonary congestion, and hypotension could be observed after an injection.
The new trial, which was conducted two years ago, involved 63 patients. As a result, 52 of them completely disappeared from the disease. Eleven people, unfortunately, could not be saved. Scientists plan to observe the surviving and cured patients for another fifteen years.
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