Will the gene mutation defeat cancer?

Immune cells armed against tumors : Cancer treated for the first time with Crispr gene therapy

A pilot study gives hope for Advances in cancer immunotherapy: With the help of the CRISPR / Cas9 gene scissors, US researchers changed the genes of certain immune cells several times in three patients. The modified T cells in the patients were still able to kill cancer cells after up to nine months.

For the first time, researchers have used the gene scissors CRISPR / Cas9 (Crispr for short) Treatment of cancer patients used. A research group led by Carl June from the University of Pennsylvania in Philadelphia changed several genes in the genetic material of certain immune cells of three patients. The modified T cells were still able to kill cancer cells in the patients after up to nine months. There were no major side effects. In the journal “Science”, the researchers speak of a “proof of concept”.

Treated blood cancer and sarcoma patients

While it's not the first time Crispr has been used in humans. At the end of November 2019 it was announced that two patients in Germany and the USA were successfully cured of their genetic blood diseases were. However, this investigation is still ongoing and the results have not been checked by experts or published in a specialist journal. June's, however.

June's team first removed genes for two receptors that the immune cells usually carry on their surface from the T-cell genome. In addition, they cut the gene for the PD-1 receptor, which inhibits T cells under certain conditions, from the genetic material of the T cells. The procedure is one Further development an already used immunotherapy against cancer, also largely developed by Carl June, the so-called CAR-T-cell therapy. Blood is taken from a patient and some of the T cells it contains are genetically modified in such a way that they recognize and destroy cancer cells.

Two patients had multiple myeloma, a type of blood cancer, and the third had a sarcoma that had already metastasized. First of all, blood was taken from them, from which the researchers isolated the T cells, whose genetic material they then changed. The modified cells multiplied and injected them back into the patient's blood.

First confirmation of the potential of the Crispr method

It is true that the cancer was caused by the souped-up immune cells in only one patient slightly pushed back. Still, this is "the first confirmation of the ability of CRISPR / Cas9 technology to target multiple genes in humans at the same time," said June. In particular, this method avoids the sometimes life-threatening side effects of CAR-T-cell therapy. There were no serious side effects.

"This study doesn't answer the big question of whether Crispr-processed T cells are effective against advanced cancer," comment Jennifer Doudna, one of the developers of the CRISPR / Cas9 method, and Jennifer Hamilton, both from the University of California at Berkeley. the work in "Science". But the work is a "important progress”And highlight“ the potential to accelerate the development of cell-based therapies ”.

June shows that the method is applicable and safe, says Niels Halama from the German Cancer Research Center (DKFZ) in Heidelberg. However, the procedure is still "too expensive and complexin order to be able to find its way into everyday clinical practice in the foreseeable future, says Patrick Schmidt from the National Center for Tumor Diseases at the DKFZ.

Gerald Willimsky, Head of Experimental and Translational Tumor Immunology at the Berlin Charité, is confident: There have been many improvements in the CRISPR / Cas9 technology since the start of the study in 2016. (with dpa)

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