Gene editing human trials begin (CRISPR)

Bioethics news, from Nature.com

“A Chinese group has become the first to inject a person with cells that contain genes edited using the revolutionary CRISPR–Cas9 technique.

“On 28 October, a team led by oncologist Lu You at Sichuan University in Chengdu delivered the modified cells into a patient with aggressive lung cancer as part of a clinical trial at the West China Hospital, also in Chengdu.”

The focus of the article seems to be a possible competition between scientists in China and the US for locking down the therapies for lung, prostate, and other cancers.

Earlier reports describe the technique used in the Chinese experiments:

“Lu’s team will extract immune cells called T cells from the blood of the enrolled patients, and then use CRISPR–Cas9 technology — which pairs a molecular guide able to identify specific genetic sequences on a chromosome with an enzyme that can snip the chromosome at that spot — to knock out a gene in the cells. The gene encodes a protein called PD-1 that normally acts as a check on the cell’s capacity to launch an immune response, to prevent it from attacking healthy cells.

“The gene-edited cells will then be multiplied in the lab and re-introduced into the patient’s bloodstream. The engineered cells will circulate and, the team hopes, home in on the cancer, says Lu. The planned US trial similarly intends to knock out the gene for PD-1, and it will also knock out a second gene and insert a third before the cells are re-introduced into the patient.””

The CRISPR gene editing experiments have been on going for a while, in research on fighting bacteria and viruses, attempts to study specific gene function, and even experiments with pluripotent cell lines. And this is not the first time cells that have been manipulated with the CRISPR technique have been used. Here is a report about successful use in the United States to treat a little girl with leukemia.

As it’s being used, it appears to be ethical. Like any tool, CRISPR could be used unethically.  Scientists in the UK have gotten permission from their government regulators to conduct unethical experiments on human embryos using the techniques.

“Niakan’s team is interested in early development, and it plans to alter genes that are active in the first few days after fertilization. The researchers will stop the experiments after seven days, after which the embryos will be destroyed.

“The genetic modifications could help researchers to develop treatments for infertility, but will not themselves form the basis of a therapy.”

Besides the concern about killing human embryos in research, many scientists and ethicists are concerned about the consequences of the potential misuse of human embryos in experiments with gene therapy in general and CRISPR, specifically:

“Guoping Feng, a neuroscientist at the Massachusetts Institute of Technology in Cambridge, hopes that with improvement, the technique could eventually be used to prevent genetic disease. But he argues that it is much too soon to be trying it in the clinic. ‘Now is not the time to do human-embryo manipulation,’  he says. ‘If we do the wrong thing, we can send the wrong message to the public — and then the public will not support scientific research anymore.'”

Nature.com has produced a graphic outlining the laws that regulate human embryo experimentation across the world.

My immediate fear is we don’t know enough about the long term effects of the therapy. Will new disorders be created or unmasked accidentally? Will the genes in the gametes (sperm and egg) be changed, affecting the future children of recipients?  Even the discovery of penicillin had its side effects, after all.

One frequently mentioned concern is the possible use of gene modification to produce designer babies, including designing *down* for workers or slaves. These would be expensive and far in the future – since we still need mothers to gestate those babies.