China has used CRISPR on at least 86 people since 2015 at Hangzhou Cancer Hospital under Dr Wu Shixiu. So far, it’s been used to treat all manner of patients with HIV, B-cell leukemia, and cancers of the kidney, lung, liver, throat, and stomach.
CRISPR, simply put, uses a modified virus to “cut and paste” DNA to edit genes with unbelievable precision and speed. It’s also surprisingly low cost. So far, non-human trials have shown it could be used to fight against HIV, cancer, blood disorders.
The CRISPR, abbreviation of Clustered Regularly Interspaced Short Palindromic Repeats, system is a prokaryoticimmune system that confers resistance to foreign genetic elements such as those present within plasmids and phages that provides a form of acquired immunity.
Doctors told journalists at the WSJ that some patients have improved. There have also been at least 15 deaths, although only about half of them were reportedly related to the gene therapy itself.
These therapies, which involved taking the immune cells from hospital patients, editing the cells, and transfusing them back into the body, are the first to use Crispr-Cas9 in living humans.
Meanwhile, in the US, there still haven’t been any CRISPR trials on humans. Part of China’s quick turnaround has been the lack of hurdles blocking Dr Wu. To carry out the trials he doesn’t need the national regulators’ approval, he simply has to ask his hospital’s review board.
In 2013 scientists first used (paywall) Crispr on human DNA, and in 2017, US scientists at Oregon Health & Science University reported using the technology to edit human embryos. It took two years for the Oregon team to receive ethical approval for their experiment.
Scientists at the Cambridge, Massachusetts-based Crispr Therapeutics also hope to start phase I clinical trials using Crispr to treat patients with a genetic disorder called beta-thalassemias.
The US is planning CRISPR trials on cancer patients that could “begin at any time” soon, according to MIT Technology Review. However, the process of receiving the official approval has been slow. This is namely because of the understandable apprehension surrounding the safety of CRISPR. For one, there could be all manner of unintentional and irreversible long-term effects on patients.
Crispr trials on humans have been relatively slow to develop in the US and UK in part due to concerns over how the risk of the procedure is communicated to patients.
Sources reveal, the Penn scientists first had to consult with an advisory board from the National Institutes of Health set up specifically to evaluate the potential risks and benefits of Crispr therapies, then get approval from the US Food and Drug Administration.
The Chinese ministry of health has to approve all gene-therapy clinical trials in China, but these regulations appear relatively relaxed.
At Hangzhou Cancer Hospital, a proposal to test a cancer treatment that modifies patients’ immune cells was approved in a single afternoon.
“It is hard to know what the ideal is between moving quickly and making sure patients are safe,” Dr. Carl June, the lead scientist for the CRISPR research team at the University of Pennsylvania, where the first human trials are slated to take place once researchers there can get through several regulatory hurdles, told.