01 February 2017

Genetically modified lymphocytes against blood cancer

Doctors have managed to defeat cancer for the first time with the help of genetically modified donor immune cells

DailyTechInfo based on Nextbigfuture: Medical first, children had cancer cured with genetically engineered T-cells from another person

A group of doctors from Great Ormond Street Hospital, London, for the first time in the history of medicine successfully used genetically modified donor immune cells to fight leukemia in two children, aged 11 and 16 months. The technology used by physicians is an extension of standard cell therapy and it uses inexpensive donor universal cells that can be obtained and used at any time.

"For the first time" we will leave on the conscience of the most enthusiastic authors of notes on this topic. In many other retellings of the new article of the same Waseem Qasim and colleagues, who recently used this method of treating leukemia for the first time, have nothing like "the world's first" - VM.

The technology developed by London doctors is significantly ahead of cell therapy technologies developed and used by a number of companies, including Juno Therapeutics and Novartis. The technologies of these companies use cells taken from the patient's body. And their use requires a lot of time for their selection, genetic modification and their introduction back into the patient's body.

In their work, British doctors used CAR-T type T cells, "ever-hungry" predator cells, which are "combat units" of the immune system, which, after appropriate gene modifications, attack only cells affected by leukemia or other oncological diseases. The donor cells were subjected to a total of four genetic modifications, two of which are carried out using TALENs genome editing technology. One of these modifications deprives the donor cell of the ability to attack normal cells of another person's body, and the second directs their "aggression" strictly to cancer cells.

Despite such a great success, there are skeptics in the medical world who question everything done by the British. "Both cured children had previously undergone a standard course of chemotherapy," says Stephan Grupp, director of the immunotherapy department at the Children's Hospital of Philadelphia, "Therefore, in this case there is no convincing evidence of the high effectiveness of the new method. Of course, it would be great if this method turned out to be working, but we have yet to see this in the near future."

The rights to the new cell therapy technology were transferred to the biotech company Cellectis, and the pharmaceutical companies Servier and Pfizer are engaged in the production of drugs. "Any patient can receive immediate treatment using our new method. In other methods, there is inevitably a delay necessary for taking, genetic processing and re–administration of the patient's own cellular material," says Julianne Smith, vice president of Cellectis, who oversees the development of technologies based on CAR-T cells, "In our case, blood taken from a donor can be turned into hundreds of doses, immediately ready for use. We estimate that the cost of producing one dose will be 4 thousand dollars, which is much less than the amount of 50 thousand required to receive a dose from the patient's own cells."

Portal "Eternal youth" http://vechnayamolodost.ru  01.02.2017


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