China's Gene-Editing Scientists Still Aiming for Crispr Supremacy
(Bloomberg) -- Chinese scientists are pressing ahead with their attempts to perfect human gene-editing technology, even after one of their compatriots drew a global backlash for editing the genes of twin girls.
In recent weeks, the work by Shenzhen-based researcher He Jiankui -- controversial because he claimed to have used the gene-editing tool Crispr on the twins when they were embryos -- was met with criticism globally and at home.
But in the less-controversial use of Crispr in adults, Chinese researchers appear determined to keep pushing to get the lead. While the technique was invented in 2012 in the U.S., the Chinese have been first at many junctures -- from using it successfully in monkeys, testing it on lab embryos not intended for live births, and using it in adults with cancer.
In an interview last week, oncologist Lu You at China’s Sichuan University said a trial he’s leading, which used Crispr on 10 lung cancer patients, is done and the data will be ready for submission to a scientific journal next month. Meanwhile, at the Chinese People’s Liberation Army General Hospital in Beijing, the head of the biotherapeutic department said it’s proceeding with five Crispr-related trials in adult cancer patients.
The continued push by Chinese researchers gives the country an edge in harnessing a potent technique with the potential to transform businesses from pharmaceuticals to agriculture. For decades, China has trailed far behind the U.S. on scientific innovation, but the speed at which the Chinese are moving on genetic editing finally sets them up to go head-to-head with the American biotechnology industry.
China isn’t close to eclipsing the U.S. as yet, but has "done a remarkably good job at taking what is one of the world’s most complex industries that the U.S. has dominated and gone straight to the forefront,” said Evercore ISI analyst Ross Muken. “It ran past Europe remarkably quickly.”
The Crispr technique, in providing a relatively simple way to alter the DNA of living things, has unlocked new possibilities for improving everything from medications to the food we eat. Scientists are experimenting with creating low-fat pigs, malaria-resistant mosquitoes and higher-yielding rice crop.
In the U.S., biotechnology companies like Editas Medicine Inc., Intellia Therapeutics Inc. and Crispr Therapeutics AG have raised millions of dollars in the hopes of monetizing the technology. One of the biggest potential windfalls is in cancer drugs and treatments, a $133 billion industry worldwide, and Editas in recent weeks received FDA approval for its first Crispr trial on humans.
At Sichuan University, Lu’s team was the first to test Crispr in a human in 2016, according to science publication Nature. Lu has been public about his efforts and his work has been allowed because it doesn’t involve changes to reproductive cells that can affect future generations.
“Our trial went through a deep and serious ethical approval process, and is nothing like He’s,” said Lu, whose team activated immune cells using Crispr and infused them back into patients to fight lung cancer. He declined to say what the trial data showed.
Even though the Chinese have been moving quickly on Crispr, so far their research hasn’t revealed conclusive results to show that their trials have worked, said Ja Lee, senior intelligence analyst at CB Insights.
"It’s hard to say if the Chinese would be the first to produce a proper Crispr medical treatment," Lee said.
China’s ascent in the space has also been bolstered by looser regulation than the U.S. and Europe. As part of its push to dominate the technologies of the 21st century, Beijing has since 2016 unleashed dramatic regulatory reforms in the pharmaceutical industry that are now reshaping the local and global landscape.
That opening up is aimed at giving the Chinese middle class access to the best health care, and its researchers an edge in the global biotech race. Yet in some cases, it’s also granted an intoxicating freedom to a research community that is in its infancy, and news of He’s trials raised concerns that there may not be sufficient checks on the industry. Crispr is still a nascent technology and all its side effects aren’t fully known.
“There’s an environment, and social pressure, to really move fast with this technology, and it may have pushed some people too far,” said David Bikard, a microbiology researcher at Institut Pasteur.
China’s government has said He’s work flouted 2003 regulations governing in-vitro fertilization, which bans the use of gene-manipulated embryos for reproduction. But Chinese law is relatively silent on gene-editing in adult humans where changes won’t affect future generations, urging only “rigorous supervision” by medical institutions.
The U.S. doesn’t allow the use of Crispr for editing the germline. And although U.S. regulators haven’t banned gene-editing of adult humans, the FDA has paid special attention to these proposals, placing months-long holds on the trials for regulatory scrutiny.
Besides the Sichuan University and Beijing army hospital trials, there are at least six other Crispr-related studies ongoing in China at present, according to the global database clinicaltrials.gov.
“We are doing everything according to law, so why shouldn’t we continue?” said Han Weidong, head of the biotherapeutics department at Chinese PLA General Hospital. “We are not afraid as we have all the relevant approvals and laws on our side. He’s case doesn’t affect us.” The hospital is using the technique to activate patients’ immune cells to fight their cancers, focusing on leukemia and lymphoma.
Still, Chinese researchers expect that the global outcry from He’s experiment will lead to tighter regulations at home or even put brakes on a further opening up. Lu said that he expects new rules on the use of gene-editing in China.
“We are waiting for a message from Beijing to come,” he said. “In the meantime, we continue our work.”
©2018 Bloomberg L.P.