New gene therapy apparently helps against blood cancer

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New treatment method could revolutionize blood cancer therapy

In the treatment of leukemia and other blood cancer diseases, gene therapy opens up new options, so that the preliminary conclusion of US medical professionals after the first years of using this treatment method. To date, some patients live without signs of the disease, reports Dr. Janis Abkowitz, President of the American Society of Hematology, active at the University of Washington in Seattle. In one study, all five adults and 19 out of 22 children who received gene therapy for acute lymphoblastic leukemia had complete remission. Cancer could no longer be detected in them. In many cases, this has continued to this day, even if a few have relapsed.

Gene therapy offers new hope for leukemia patients, reports the President of the American Society of Hematology. In several studies, the application of the new therapy has been tested on selectively selected patients for several years. To date, more than 120 patients with different types of blood and bone marrow cancer have received appropriate treatment. Mostly with considerable success, despite the fact that some of those affected have already had several unsuccessful bone marrow transplants and up to ten types of chemotherapy or other treatments. Dr. told the Associated Press. Abkowitz, the results of the previous studies are "really exciting", because "you can take a cell from a patient and reconstruct it so that it becomes an attack cell." These would then specifically attack and destroy the cancer cells.

Cancer often no longer detectable after gene therapy As one of the promising examples, the US physicians cite the case of eight-year-old Emily Whitehead from Philipsburg in Pennsylvania. Her cancer was so advanced that doctors assumed her main organs would fail within a few days. She was then the first child in the world to be treated with gene therapy and today - almost two years later - no longer shows any signs of cancer. As part of the novel therapy, the doctors filtered the patient's blood, removed millions of white blood cells and changed them in the laboratory or added a gene. The doctors then administered the modified T cells to the patients by infusion over three days. In principle, "a living medicine" is injected from permanently changed cells into the affected person, explained Dr. David Porter from the University of Pennsylvania. The cells can multiply in the body and fight cancer effectively, Porter continues. The results of the previous use of gene therapy will be presented at a four-day conference of the American Society of Hematology that has been running in New Orleans since Saturday.

Hope for a significant improvement in blood cancer therapy Although there is already the possibility to successfully treat patients with leukemia, non-Hodgkin's lymphoma and myeloma on the basis of chemotherapy, bone marrow or stem cell transplantation, the transplantation remains risky and a donor cannot always be found. In addition, chemotherapy is accompanied by massive side effects and success is not always guaranteed. The gene therapy, however, must be manufactured individually for each patient and causes laboratory costs of around $ 25,000, without profit margin, report the US doctors. However, it is significantly cheaper than many forms of drug treatment or a transplant. The University of Pennsylvania has patented its gene therapy process and transferred the license to the Swiss pharmaceutical company Novartis.

Gene therapy with promising successes So far, most treatment with gene therapy has been carried out at the University of Pennsylvania (59), with four of the first 14 patients with chronic lymphoblastic leukemia (CLL) having complete remissions, four partially and the rest not Treatment responded. At the National Cancer Institute, Dr. James Kochenderfer and colleagues treated eleven patients with lymphoma and four with CLL based on gene therapy and were able to achieve complete remissions in six of them, in some cases in six and in the others the course of the disease had stabilized so that it was too early to draw a final conclusion. According to the researchers, the side effects of the treatment were severe flu-like symptoms and other reversible or temporary impairments. This would make gene therapy even more gentle than chemotherapy, for example. The doctors conclude that the new method could be approved as the first form of gene therapy in the USA and open up completely new possibilities for blood cancer treatment. (fp)

Image: Andreas Dengs, /

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