A Powerful New Technology
The biotech world has been roiled this week by developments overseas.
A couple of years ago, I predicted we were on the cusp of this event: the world’s first humans born with artificially edited genomes.
He Jiankui, a researcher at China’s Southern University of Science and Technology, presented the results of a secretive clinical trial at the Second International Summit on Human Genome Editing in Hong Kong this week.
He claims he used CRISPR gene editing on 50 human embryos. He then implanted some of these embryos and two of them — twins — were successfully delivered to term. He also claims that there is another potential pregnancy ongoing.
These are possibly the first humans ever born who have had their genes altered from the very beginning of life.
Ostensibly, the researcher claims he was trying to find a way to prevent HIV transmission by editing these genes.
A small percentage of people worldwide have a variation in a gene called CCR5, although up to 10% of people of European or West Asian ancestry may carry it.
As best as we can tell, people with this variant can’t get HIV.
The gene modification made in these human embryos edited the gene in such a way as to duplicate the naturally occurring variation, potentially granting lifelong protection.
We first discovered this gene variant’s role in 2007 when an HIV-infected individual received a bone marrow stem cell transplant in order to treat leukemia, which he also had.
As it turned out, the donor’s cells used for his transplant contained the CCR5 gene variant. The new white blood cells circulating, produced by the donor marrow transplant, were resistant to HIV infection. As the old white blood cells died off, so did the HIV infection.
That turn of luck made Timothy Ray Brown the first person to be cured of HIV.
He Jiankui’s attempt to use this naturally occurring immunity to HIV has raised serious ethical and moral questions. Since he edited the genes at the embryonic stage of development, all resulting cells, including reproductive cells, carry the changes. That means these babies, if they go on to have children of their own, will pass on copies of these genes. This is a way of changing the human gene pool… maybe forever.
Adding to the controversy is the fact the CRISPR technology Dr. He used is far from proven. It’s known for off-target effects — meaning that other genes can be accidentally changed during the process, with potential harm, such as causing cancer. Off-target changes have happened with the embryos in question, and it’s still to be determined what the results will be.
According to Francis S. Collins, the director of the National Institutes of Health (NIH):
This work represents a deeply disturbing willingness by Dr. He and his team to flaunt international ethical norms. The project was largely carried out in secret, the medical necessity for inactivation of CCR5 in these infants is utterly unconvincing, the informed consent process appears highly questionable and the possibility of damaging off-target effects has not been satisfactorily explored. It is profoundly unfortunate that the first apparent application of this powerful technique to the human germline has been carried out so irresponsibly. The need for development of binding international consensus on setting limits for this kind of research, now being debated in Hong Kong, has never been more apparent. Without such limits, the world will face the serious risk of a deluge of similarly ill-considered and unethical projects. Should such epic scientific misadventures proceed, a technology with enormous promise for prevention and treatment of disease will be overshadowed by justifiable public outrage, fear and disgust.
And Dr. Collins is far from alone.
In a way, this controversy is an old one. Every powerful new technology we’ve developed has come with serious ethical and moral issues regarding its use.
But technology is just a tool: We can use it for good, or we can use it for evil. It’s up to us to decide.
For Technology Profits Daily,
Chief Technology Expert, Technology Profits Daily