Ruling Should See Gene Editing Trial Begin This Year

Updated on

The low-cost, “easy to use” CRISPR technology that allows for the editing of the human genome has received approval for its first trial by an advisory committee at the National Institutes of Health (NIH). While it will be a relatively small trial with a small scope, the University of Pennsylvania is poised to begin manufacturing cells and treating cancer patients at its facilities as well as in other institutes in Texas and California.

Gene editing with CRISPR will look to target cancer

CRISPR, or Clustered Regularly Interspaced Short Palindromic Repeats, allows scientists to turn genes on and off like a light switch as well as allowing them to edit specific genes and even change out components of DNA like a case for an iPhone. The ease of use surprised many but at the same time, raises no small measure of ethical questions as proved by the controversy, which surrounded a “secret” meeting of lawyers, regulators, doctors, researchers and others that met at Harvard Medical School last month to discuss the creation of a complete synthetic human genome.

But now, the Recombinant DNA Advisory Committee (RCA) which advises the NIH on oversight and ethics with regard to gene manipulation has followed the UK in permitting the first test on human patients set to begin in the last months of this year.

The trial will be paid for by former Facebook president Sean Parker through the Parker Institute for Cancer Immunotherapy, which he began with a $250 million investment in early 2016.

The trial will try to turn human T cells, a type of white blood cell, into hunter-killers of cancer cells. The idea is to take T cells from humans edit them and reinsert them into the patient’s bloodstream with the hope that the modified T cells will have a proper go at the offending cancer cells looking to spread its influence through the patient’s body.

The scientists hope to enhance the cancer fighting potential of T cells though editing and make them better detectives and more efficient assassins. Additionally, the researchers are hoping to remove a protein that seems to slow the T cells in their fight.

The ruling, specifically the approval of this trial as the first, is not without controversy as many experts had expected the company Editas to receive the first go-ahead in CRISPR use in living humans. Editas hopes to combat blindness using gene editing. For many, the decision was a bit off given the fact that in 1999, a teenager died after participating in a study led by the University of Pennsylvania. Jesse Glesinger passed away with days of receiving an injection of genes that were edited (though not with CRISPR).

News like this always begs ethical questions and often asks more questions than gene therapy and editing provides answers. That said, let’s skip that today and leave it to scientists and ethicists to sort that out as they are generally diametrically opposed to the other group. Researchers, given the opportunity would love to do what they want in the name of science, while ethicists are by definition kind of hung up on ethical concerns.

It’s happening whether a good idea or not, it’s now longer if but when

“Cell therapies [for cancer] are so promising but the majority of people who get these therapies have a disease that relapses,” says study leader Edward Stadtmauer, a physician at the University of Pennsylvania in Philadelphia while pointing out how edited genes could really improve the human immune system.

The small trial will involve 18 patients with varied cancers the manner described above: make the genes smarter, make them less burdened by that protein and finally make the killer T cells anonymous as they near cancer cells so the cancer cells can’t “diffuse” them.

“Last year’s excitement over CRISPR was in anticipation of this,” says Dean Anthony Lee, an immunologist at MD Anderson Cancer Center in Houston, Texas, who sits on the RAC committee.

While the University of Pennsylvania received the first go ahead to work with gene editing and reinsertion into humans, it won’t be the last and the aforementioned Editas Biotechnologies of Cambridge, Massachusetts is expected to submit its proposal to fight a rare form of blindness and hopes to receive approval in early 2017.

The potential problem with that trial is that the RAC could be approving a technique that would financially beneficial for a single company, raising yet another ethical question.

While CRISPR has been deemed “easy to use” and superior in its techniques to edit multiple genes simultaneously, the technology is not without its faults including a knack for “off-target” edits. And that’s a massive problem is the genome is accidentally altered accidentally that could see the edited cells go against their programming and simply attack the immune system rather than making it smarter more effective.

Leave a Comment