New gene therapy apparently helps against blood cancer

New treatment could revolutionize blood cancer therapy

09/12/2013

In the treatment of leukemia and other blood cancers, gene therapy opens up fundamentally new options, so preliminary conclusion of US physicians after the first years of use of this treatment method. To date, some patients live without signs of the disease, reports Dr. med. Janis Abkowitz, President of the American Society of Hematology, working at the University of Washington in Seattle. In one study, a complete remission occurred in all five adults and 19 out of 22 children who received gene therapy with acute lymphoblastic leukemia. With them, no more cancer could be detected. In many cases, this has continued until today, even if a few have experienced a relapse.

There is hope for new leukemia patients with gene therapy, says the president of the American Society of Hematology. In several studies, the application of the new therapy has been tested for several years on selected patients. To date, more than 120 patients with different types of blood and bone marrow cancer have received appropriate treatment. Mostly with considerable success, even though some of those affected had already had several unsuccessful bone marrow transplants and up to ten types of chemotherapy or other treatments. Opposite the US news agency „Associated Press“ explained Dr. Abkowitz, the results of previous studies are „really exciting ", because „You can take a cell from a patient and redesign it to become an attack cell.“ These would then specifically attack and destroy the cancer cells.

After gene therapy often no more cancer detectable
As one of the most promising examples, the US doctors call the case of eight-year-old Emily Whitehead from Philipsburg in Pennsylvania. Their cancer had progressed so far, doctors assumed that their major organs would fail within a few days. She was then treated as the first child in the world with gene therapy and shows today - almost two years later - no signs of cancer more. As part of the novel therapy, the doctors filtered the blood of the patient, removed millions of white blood cells and changed them in the laboratory or they supplemented by a gene. The doctors then infused the patients with the modified T cells for three days. In principle, the person affected „a living medicine“ injected from permanently altered cells, Dr. David Porter from the University of Pennsylvania. The cells can multiply in the body and fight the cancer effectively, Porter continues. Presented are the results of the recent use of gene therapy at a running since Saturday in New Orleans, four-day conference of the American Society of Hematology.

Hope for significant improvement in blood cancer therapy
Although it is already possible today to treat patients with leukemia, non-Hodgkin's lymphoma and myeloma successfully on the basis of chemotherapy, bone marrow or stem cell transplantation, transplantation remains risky and donors can not always be found. In addition, chemotherapy is associated with massive side effects and success is not always guaranteed. The gene therapy, however, must be made for each patient individually and cause laboratory costs of about $ 25,000, with no profit margin, report the US doctors. However, it is much cheaper than many forms of drug treatment or transplantation. The University of Pennsylvania has patented its gene therapy process and has transferred the license to the Swiss pharmaceutical company Novartis.

Gene therapy with promising results
The University of Pennsylvania has received the most gene therapy treatment to date (59), with four of the first 14 patients with chronic lymphocytic leukemia (CLL) having complete remissions, four partially responding and the remainder unresponsive to treatment. At the National Cancer Institute, Drs. James Kochenderfer and colleagues treated eleven patients with lymphoma and four with CLL on the basis of gene therapy and were able to achieve a complete remission in six of them, six of them in part and the remainder of the disease stabilized in the other, so it is too early to draw a final conclusion. According to the researchers, the side effects of treatment have been severe flu-like symptoms and other reversible or temporary impairments. Thus, gene therapy would still be much gentler than, for example, chemotherapy. The physicians conclude that the new procedure could be approved as the first form of gene therapy in the US, opening up entirely new options for blood cancer treatment. (Fp)