CRISPR gene-editing has been called the scientific breakthrough of the century … and sometimes, it feels like we’ve been hearing about it for just as long. Bacterial palindromic repeats—which are key to this technology—were first discovered in the 1980s. But it took years for scientists to uncover their function … and even longer to use that information to develop CRISPR-Cas 9 gene-editing. Then we had to wait for CRISPR to get into clinical trials!
At the close of 2016, the moment finally arrived: cells modified with CRISPR-Cas 9 were injected into a human subject. With more trials set to launch in 2017, it’s worth examining these studies in closer detail. Sure, the waiting game is far from over, but at least we’ll have some preliminary results soon.
In October 2016, China launched the world’s first gene-editing trial in humans. For the first time ever, genetically-modified cells were injected into a patient—marking the start of a new era of research. This phase I study, based out of Sichuan University, is primarily focused on safety, but will also measure survival and response rates in patients with non-small cell lung cancer.
Ten patients are involved in the study. Each will receive up to four injections of the immune cells, which have had a select gene edited out—namely, one suspected of helping to spread the cancer. In this case, it’s the gene that codes for protein PD-1, which restricts a cell’s immune response and inadvertently facilitates cancerous growths.
Sound familiar? That’s because there are PD-1 inhibitors already on the market, including Keytruda, developed by Merck (NYSE:MRK), and Opdivo, made by Bristol-Myers Squibb (NYSE:BMY). Indeed, some question the long term merits of this CRISPR study for just that reason: editing out the PD-1 gene may not be very scalable, particularly when compared to the pharmaceutical drugs that perform a similar function and can be manufactured in mass quantities.
Still, the study is proceeding—though not on the timeline originally set out. Initially scheduled to launch in August, the trial was delayed by several months because developing the genetically modified cells took longer than anticipated. Nevertheless, China did beat the United States in getting to the clinic first.
“I think this is going to trigger ‘Sputnik 2.0,’ a biomedical duel on progress between China and the United States,” Dr. Carl June, a researcher at the University of Pennsylvania, told Nature. But he hastened to note that this duel was a boon for the scientific and medical communities alike, “since competition usually improves the end product.”
University of Pennsylvania
Based out of the University of Pennsylvania and funded by Sean Parker, the American trial only recently received approval from the US National Institutes of Health. The FDA still needs to give it the green light—but if they do, the study will commence early this year.
The trial will involve removing T cells from 18 patients with several types of cancer, including myeloma, sarcoma and melanoma. Those T cells will be edited outside of the body, with genetic material both added and removed, then injected back into the patient.
As in the above trial, those edits will remove the gene that codes for the PD-1 protein. But they will also give the T cells a receptor for NY-ESO-1, a protein found on some tumors. When those T cells are injected back into the body, they should be be able to locate and attack tumors that display the protein. In theory, at least.
The study builds on earlier work by June’s team of researchers and should take two years to complete.
More on the way
More trials are expected to launch in March 2017. Three are being planned by researchers at Beijing’s Peking University, for example, investigating CRISPR’s efficacy against bladder, prostate and renal-cell cancers. They do not yet have final approval. Editas (NASDAQ:EDIT) has likewise suggested that they could launch a clinical trial as early as this year.
Bottom line? 2017 should be exciting for CRISPR-Cas 9 research, with preliminary results coming available. But the wait is far from over. Each of these studies is in very early stages … and that means there’s a long way to go before any therapy is commercialized.
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Securities Disclosure: I, Chelsea Pratt, hold no direct investment interest in any company mentioned in this article.