“Nature already invented a cure for HIV”: how human genome editing could change the world

As the world discusses the ethics of growing human organs in pigs, scientists gathered at the Skolkovo Foundation last week to discuss an equally divisive topic: that of human genome modification.

While the idea of editing human embryos has sparked fierce debate across the world, the editing of cells in adults to cure genetic illnesses, cancer and some infectious diseases is the subject of active research, including by resident companies of the Skolkovo Foundation.

One of those working in this area is Marina Popova, a qualified doctor and the CEO of AGCT, a company that is working on a treatment for HIV-associated tumours and HIV itself using stem cell transplants and site-specific genome editing.

“The use of genome editing in HIV treatment was discovered by chance, because there are some people who have a natural immunity to HIV via a gene mutation: that is to say, nature already invented a cure for HIV,” Popova told a panel discussion held as part of Skolkovo’s annual Startup Village event on Thursday.

Timothy Brown, known as the “Berlin patient,” is the only person in the world believed to have been cured of HIV. After he received a bone-marrow transplant in Germany for leukemia in 2007, his HIV became undetectable, seemingly because the bone marrow donor was one of those people with natural immunity.

There are now two groups working on a treatment based on this theory, said Popova: Sangamo in California, and her company AGCT. On Friday, the day after the genome editing discussion, AGCT – a resident of the Skolkovo Foundation’s biomed cluster – won second prize and 2 million rubles ($30,000) in the competition section of the Startup Village event.

Genome editing could be used to treat many other diseases, including the x-linked severe combined immunodeficiency (X-SCID) disorder. Clinical research has already been carried out into the use of genome editing to treat X-SCID and is now ready for clinical implementation, while similar research into the treatment of beta-thalassemia, a group of inherited blood disorders, is at the preclinical stage, said Popova.

The technology could also be used to treat sickle cell disease (SCD), with which more than 5 million babies are born every year. Research into this field is at the late preclinical stage, Popova told the panel discussion, titled Human Genome Modification: A Way to Be Cured or Non-Natural Selection?

“What’s interesting here is that only 10 to 30 percent of cells need to be corrected for normal erythropoiesis [production of red blood cells],” she said.

From mosquitoes to pigs

Popova also mentioned research published last year by U.S. scientists who genetically engineered a mosquito incapable of transmitting malaria to humans. The malaria-free mosquito would pass on the disease-free trait to 99.5 percent of its offspring, raising the possibility of eradicating the disease, which kills up to a million people – mostly children – every year.

“It will be amazing if we can do that,” she said.

Scientists in the U.S. are currently making headlines for editing the DNA of a somewhat bigger animal, in an attempt to grow a human organ inside a pig.

Pavel Volchkov, head of the genome engineering laboratory at the Moscow Institute of Physics and Technology, presented the technology that the U.S. scientists are currently using to edit the DNA of a newly fertilised pig embryo. Using a system known as CRISPR gene editing, the DNA that would enable the future pig foetus to grow a pancreas is removed and replaced with human stem cells. The aim is for the foetus to go on to grow a human pancreas that could then be used for transplants, but currently the embryos are being destroyed after 28 days amid ethical concerns.

“We have been doing this [editing the genomes] in animal embryos for some time,” said Volchkov.

“Changing an animal, we can change it so much that its organs can be used for transplantation into humans,” he said, adding that he was neither advocating nor opposing the technology.

“It [this technology] could also be used to solve some ecological problems; the important thing is not to create any more problems in doing so,” he said.

If the experiments on pigs have elicited mixed reactions – some have expressed concern that some of the human cells might migrate to the pig’s brain during its development, making that organ more human as well – then reactions to experiments on human embryos have been even more divided.

Hands off human embryos

News that Chinese scientists had edited the genomes of human embryos elicited strong reactions last year, and at the beginning of this year, U.K. scientists studying miscarriages and infertility became the first to get permission from the national fertility regulator to alter human embryos. The scientists are obliged to destroy the embryos within seven days, and there are so far no plans for the clinical application of the technology, amid fears that the genetic changes to embryos will be inherited by future generations with unpredictable consequences.

In some circles, there is strong opposition to the idea of gene editing.

“Human genome editing elicits not only hope in people, but also fear,” said Vladimir Timakov, deputy head of the expert centre of the World Russian People’s Council, a religious and public organisation set up by Patriarch Kirill, head of the Russian Orthodox Church.

A biologist by education, Timakov says he can see the issue from both sides of the divide.

“We are used to associating innovators with science and conservatives with traditional religions, but that’s not always the case in real life,” he told the panel discussion. “There is a polemic in both scientific circles and religion about the effectiveness and ethics of human genome editing.”

To illustrate his point, Timakov cited one priest, Dmitry Pershin, as saying experiments to alter human embryos are “medical fascism,” and another, Alexander Pikalyov, as saying that uninformed reactions to human genome editing are simply the “panic of a horse upon seeing a steam engine.”

Moral maze

Timakov identified three main arguments among those opposed to human genome editing: that it could lead to future mutations that we know nothing about, that mankind should not interfere in nature, and that experimenting on embryos and then destroying them is unethical.

“We were all embryos once, and no one would want to end up as experimental material,” he said.

As for the consequences of interfering in nature, Timakov said the situation was not black and white.

“What is God’s plan: a healthy baby or one with Down’s?” he said.

While Russian scientists like Popova continue their work on editing the genome of adults suffering from a disease, the prospect of Russian experiments on human embryos does not look likely in the near future.

“It’s hard to say how prepared research groups in Russia would be to edit embryo genes if such technology was allowed,” Kristina Khodova, a project manager at Skolkovo’s biomed cluster, told Sk.ru in an interview earlier this year.

“I’m sure it would attract a huge amount of attention, both among scientists and the public.

“On the one hand, there are positive prospects of preventing the development of genetic disorders and other serious diseases, but on the other hand, it’s a dangerous path that could lead to parents starting to choose the colour of their baby’s eyes, and from there it’s a small step to eugenics,” said Khodova.

“So I’m sure society will return to the discussion of this issue again and again.”