2018 may be the year the gene editing technology known as CRISPR starts becoming reality in human trials, and while there are some very ambitious plans out there for the science, I'm thinking this is another case of eyes being too big for the task at hand, and there's going to be some sort of political and ethical movement to clamp down on CRISPR if it's as powerful as the proponents say.
Ever since 2012, when researchers first discovered that bacterial immune systems could be hijacked to edit DNA in living creatures, CRISPR has been hailed as a maker of revolutions. This was the year that prediction felt like it was starting to come true. U.S. scientists used the CRISPR gene editing technique to treat a common genetic heart disease in a human embryo. Many more diseases were successfully treated in mice using CRISPR. Hell, a particularly enthusiastic biohacker even spontaneously injected himself with muscle-growth genes while giving a talk at a conference.
But if 2017 was the year that the potential of CRISPR began to come into focus, 2018 may be the year that potential begins to be realized.
Next year, the first human trials of CRISPR-based treatments in the U.S. and Europe are slated to begin.
This month, biotech firm CRISPR Therapeutics became the first to submit a clinical trial application to European regulators. Tests are set to begin next year for its therapy that combines CRISPR gene editing and stem cell therapy to treat the blood disorder beta thalassemia. CEO Samarth Kulkarni told Gizmodo that the company also plans to file an application to conduct a clinical trial using a similar therapy to treat sickle cell disease in the first half of 2018. “In 2018, the first human is going to get dosed with CRISPR in the clinic,” Kulkarni told Gizmodo. “And we’re going to be the first ones to do it.”
Both disorders are genetic, caused by mutations to the genes that produce hemoglobin, a protein essential to ensuring that red blood cells ferry oxygen throughout the body. Without that oxygen, people can suffer from severe anemia, developmental delays, damage to organs, and pulmonary hypertension. The idea is extract stem cells from patients’ bone marrow and correct the faulty genes with CRISPR, a gene-editing technique that allows scientists to cut and paste tiny snippets of genetic code. Then those edited cells would be infused back into the body, where they would multiply, eventually outnumbering the diseased cells. Sickle cell disease and beta thalassemia are good candidates for CRISPR because in many cases, they are caused by a mutation to one single DNA letter.
At Stanford, a different spin on using CRISPR to treat sickle cell disease is also moving toward clinical trials. Matthew Porteus, who heads the research, said that his group expects to file a clinical trial application with the FDA by the end of 2018 and begin trials in 2019. “Our New Year’s resolution for 2018 is to gather the data so we can file a [trial application] by the end of the year, so we can start a clinical trial in 2019,” Porteus told Gizmodo. “We just need to check off all the boxes.”
Chinese scientists, meanwhile, used CRISPR for the first time on a human in 2016, and conducted a second human trial this year, setting off a biomedical duel between the U.S. and China and sparking concerns that the trials were irresponsibly premature. The first U.S. human CRISPR trial was slated to begin this summer at the University of Pennsylvania, after receiving a regulatory stamp of approval to proceed last year. It is unclear what has caused that trial’s delay.
Porteus said that he expects 2018 will bring many more preclinical studies demonstrating how CRISPR might be used to treat different diseases. In 2017, there were several such studies, addressing devastating diseases and conditions such as Huntington’s disease, Lou Gherig’s disease, and an inherited form of hearing loss in mice.
“There is going to be a lot of behind-the-scenes work of turning those into a real clinical protocol,” Porteus said. He also predicted 2018 will see applications for more clinical trials, though most likely ones the involve simply deleting a problematic gene rather than correcting it.
If anything, I expect ridiculous legal limitations on the science just to protect the profit margins on the treatment. Gene therapy clinics may be a near future sci-fi staple plot point these days in both novels and games, but this is what we're looking at if the CRISPR people are right, and man that's a huge, huge "if" we're talking about.
Rarely does that kind of technology end well in the stories. Either it's way too dangerous, or made impossible to afford for everyone except the ultra rich, and you'll excuse me if I don't believe the current administration would be capable of recognizing the potential social and ethical dangers.
But then again, the future of CRISPR may very well be not up to America at all.