CRISPR, the genome editing technology that has literally taken biomedical science by storm, is finally getting close to being tested in humans. On June 21, 2016, an advisory committee to the US National Institutes of Health (NIH) approved a proposal to use the CRISPR / Cas9 system to improve cancer treatments by involving patient T lymphocytes in research.
However, before clinical trials can begin, scientists must also obtain approval from the US Food and Drug Administration and the medical centers where the research will be conducted.
The essence of the "modified" treatment, proposed by scientists at the University of Pennsylvania, is that they extract the patient's cells and then genetically "edit" them using CRISPR technology. After that, the altered T-lymphocytes will be reintroduced into the human body, where the cells can attack the tumor. Thus, manipulation can help the body to overcome the disease on its own.
“Cellular cancer therapy is a promising treatment, but most people who have received this treatment have relapsed,” says Edward Stadtmauer, a physician at the University of Pennsylvania. "Gene editing can improve such therapy and eliminate some vulnerabilities in the body's immune system."
The first human trial will not be large-scale, it is only intended to test whether CRISPR technology is safe for use in humans. The researchers will receive US $ 250 million in funding from the Immunotherapy Foundation, created last April by Facebook President Sean Parker.
Staff at the University of Pennsylvania will fabricate modified (edited) cells and recruit volunteers for treatment (along with centers in California and Texas).
What will the experiment consist of? Researchers will extract T cells from patients with melanoma, sarcoma or myeloma and edit them using CRISPR technology.
One change will add a gene needed to make a protein engineered to detect cancer cells and train T cells to target them.
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The second edit will extract a natural T-lymphocyte protein that can interfere with this process.
The last change is defensive: Scientists will remove a gene responsible for the production of a protein that informs cancer cells that T-lymphocytes are immune cells. In this way, the researchers will prevent cancer cells from shutting down T-lymphocytes.
Then, after all the procedures, scientists will introduce the altered cells back into the patient's body.
"Last year's CRISPR hype foreshadowed just this," said Dean Anthony Lee, an immunologist at the Andreson Cancer Center in the United States (RAC) and a member of the NIH Recombinant DNA Advisory Committee, which reviewed the proposal.
CRISPR technology, he says, will simplify genetic engineering, allowing treatments based on it to move forward faster.
“This is an important new approach. We are going to learn a lot from this challenge. And I hope it will form the basis for new therapies,”says oncologist Michael Atkins of Georgetown University, one of three RAC members who also considered the proposal.
Note that other scientists are not left behind. For example, Editas Biotechnologies says it would like to use CRISPR in clinical trials to treat a rare form of blindness. This study is scheduled for 2017.