Genetic modification at service of cancer

CRISPR is a kind of genetic manipulation that has also been at the service of oncology. From the Novartis Boston Research Institut (NBIR), an old candy factory, not far from the Massachusetts Institute of Technology, Rob McDonald, one of the researchers, explain how they use the famous genetic scissors discovered by the scientific Doudna and Charpentire to try to develop new drugs against different types of cancer.

McDonald explains that cancer is a complex genetic disease, and although the tumor initially responds to treatment, we know that is able to develop resistance because has secondary mutations, usually in tumor suppressor genes (that are the responses to the control of tumor cells). "If those guards are mutated or missing, how can we attack them, if they are not already?"

Thanks, CRISPR technology scientific from NIBR are putting one by one million of genes to checking how it affects to the tumor cell and  tries to accelerate the discovery of new anticancer molecules. In this old candy factory, they are working with 400 cell lines, 19000 genes and about 76000 million cells that could cover an entire baseball field. This new kind of technology suppose a titanic effort, says McDonald, and it would be impossible without the new genetic technology that is not only apparently simple (its use has spread quickly to all fields of science), but also very cheap (about $ 20-30 per gene, compared to a previous edition genetic technology, TALEN, which could cost up to around $ 4,000 per gene).Technology allows doing precise changes in the DNA cells to try to answer a question: Is it important that gene for the survival of tumor cells? And consequently, is not is a good candidate to try to attack with a drug potential?

Although this application now seems to have their main potential in the field of drug development it is not the only one. Laboratories around the world are also exploring the therapeutic potential of this genetic technology in the treatment of cancer patients. In 2015, researchers at the Eliza Hall Institute (Australia) demonstrated  that this technique was able to remove Burkitt lymphoma cells by deleting the gene (MCL1) responsible for keeping alive the tumor cells. And from the same NIBR, in collaboration with the University of Pennsylvania, the use of CRISPR has been investigated to improve the effectiveness of treatment with modified cells. This hematologist showed few years ago that modify genetically the patient own cells in the laboratory and then transfering this cells to the patient allowed to control certain hematological tumors, mainly leukemia. This cells from the laboratory have already proved effective in a group of 36 children with lymphoblastic leukemia who had not responded to other treatments.

Glen Dranoff is the responsible for Immuno-Oncology at NIBR. He explains that CRISPR could improve the control of the defensive cells and make them more powerful.  Although he is very cautious about the therapeutic potential of genetics in cancer cells edition: "Of course there is much interest, but we are not there yet. At the moment, CRISPR is a very potent drug development tool." And Glen Dranoff is right. In the clinic, there is some risk you don't have in the laboratory like the security. Because at the end you are making changes in the genome and you have to be very sure of what you do.