Home Tech CRISPR therapy has been used to treat more than 200 patients

CRISPR therapy has been used to treat more than 200 patients

CRISPR therapy has been used to treat more than 200 patients

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This article is part of The Checkup, MIT Technology Review’s weekly biotech newsletter. Sign up here to receive it every Thursday in your inbox.

These last few days have been spent thinking about when, how, and if gene-editing tools should be used to alter the human genome. These are big questions and emotive questions–especially when it involves editing embryos.

This week, I was able to witness scientists, ethicists and patient advocacy groups grapple with these issues at the Third International Summit on Human Genome Editing in London.

Gene editing is a topic that has many exciting possibilities. Multiple clinical trials have been conducted in the decade since CRISPR was discovered by scientists to modify cell genomes. CRISPR has been used to save lives and transform others.

It hasn’t been easy. Some volunteers have died, and not all trials went according to plan. The cost of successful treatments is likely to be high, so they are only available to the very wealthy. These trials are based on changes in genes in adult cells. However, some people are looking to use CRISPR or other gene-editing tools in eggs and sperm. The field continues to be plagued by the possibility of designing babies.

He Jiankui, who was then based at Shenzhen’s Southern University of Science and Technology, made the announcement that he had used CRISPR to create human embryos at the 2018 summit. As you might imagine, the news of the “CRISPR babies” caused a huge commotion. Victor Dzau, president of US National Academy of Medicine, said, “We’ll never forget that shock.”

Protesters outside of the Third International Human Genome Editing Summit, London

He Jiankui was sentenced to prison and released last year. While heritable genome editing was already banned by China at the time, it has been outlawed in China since 2003. China has since passed a number of additional laws to prevent such an event from happening again. Yaojin Peng, from the Beijing Institute of Stem Cell and Regenerative Medicine, stated that heritable genome editing is now prohibited under criminal law.

This year’s summit was less dramatic. There was still plenty of emotion. Victoria Gray, a 37 year-old survivor of sickle-cell, spoke out about gene editing and how it might be used to treat the disease. She shared with the audience how her severe symptoms disrupted her childhood, adolescence, as well as scuppered her dream of becoming a doctor. She described painful episodes that kept her in hospital for several months. Her children were worried she might die.

Then, she had to undergo a treatment that involved editing her bone marrow’s genes. Her “super cells”, as she refers to them, have changed her life. She felt rejuvenated and cried of joy after receiving her transfusion of edited cell. She said that it took her seven to eight months to feel better. But, after that, she began to enjoy the “life that I once felt was just passing by.” I could see the normally stoic scientists around my shedding tears.

Victoria is one of over 200 patients who have received CRISPR-based therapies as part of clinical trialsDavid Liu, of Harvard and the Broad Institute of MIT, who led the development of CRISPR’s new and improved forms, said, Trials are underway for a variety of other diseases, such as cancers, amyloidosis, and genetic vision loss.

Liu spoke out about Alyssa, a young girl in the UK who was diagnosed as having leukemia. This is a type that affects white blood cells called T cells. The bone marrow transplant failed as well as chemotherapy. The Great Ormond Street Hospital in London tried a CRISPR-based method.

It involved taking healthy T cell from a donor and using CRISPR for modification. The cells were modified so that they wouldn’t be rejected by Alyssa’s immune system. However, they would be able track down and attack Alyssa’s cancerous T cells. These cells were then given as a treatment to Alyssa. It seems to have worked.

Liu stated that her cancer was still undetectable after 10 months of treatment.

It is amazing to hear about such success stories. There are some concerns.

At the summit, equity was brought up repeatedly. Gene-editing therapies will likely cost millions of dollars. Who will be able afford them? Multiple attendees were concerned that they would not be affordable by people who live in low- or middle-income countries.

CRISPR therapies remain experimental at the moment. None have been approved. Therefore, clinical trials are the only way to access them. Most of these are being conducted in the wealthy world. Natacha Salome Lima is a bioethicist and psychologist at the University of Buenos Aires. She pointed out that, while 70% of all cancer cases worldwide are found in low- or middle-income countries (Argentina), two-thirds of gene therapy cancer trials are being conducted in rich countries.

I could see that the summit organizers had tried to bring in speakers from all parts of the world and to include people with the genetic disorders being addressed by gene editing. Several attendees felt that there were still some voices missing from the discussion. Marc Dusseiller, ETH Zurich in Switzerland, asked me, “What about the LGBTQ community?” He describes himself as a “workshopologist” who is interested in biohacking, bio art, and biohacking.

It is also important to note that not all CRISPR treatments are successful. Multiple researchers pointed out that we still don’t understand the process. We know that DNA can be cut and that DNA can be swapped between DNA bases or parts of the genetic code. Unintended consequences elsewhere in the genome are not something we can be certain of. It is possible to accidentally cause a genetic change in another part of the genome, which could have potentially harmful consequences.

Terry Horgan, 27, died last year while participating in a clinical study of a CRISPR therapy to treat his Duchenne muscular disorder, a fatal condition that causes muscle degeneration. It is not known if the treatment was involved in his death.

There’s always the risk that rogue scientists might set up companies offering unapproved procedures for desperate people who are willing and able to pay. Robin Lovell-Badge is a stem-cell biologist at Crick Institute, where this summit was held. They may even sell unapproved procedures that are intended to enhance rather than treat people.

A couple of protesters stood at entrance to the summit with a banner that read “Stop designing babies.” This sentiment was shared by many scientists. They are especially concerned about future attempts at editing the genes of eggs, embryos, and sperm.

The theory is that you could alter the DNA of an embryo to stop a baby developing a heritable condition. However, early embryos can be affected by gene editing in unintended ways. Scientists are allowed to study them for 14 days before being required to destroy them. These changes would also be passed on to the next generation.

While most attendees are focused on ethical and technical concerns, Dusseiller has a different concern. He said that the summit was too dry. However, serious issues around gene editing can be addressed with humor. He said, “We need more weirdness.” “We need more jokes.”

Tech Review archive: Read more

More than 50 studies are currently underway that use gene editing to treat blood diseases, HIV, cancer, and other conditions. Many of them involve CRISPR as my colleague Antonio Regalado reported earlier in the week.

Last year, a volunteer from New Zealand was the first to receive a CRISPR treatment to lower cholesterol. One scientist behind the work believes that the approach could be beneficial to almost everyone.

CRISPR is also being investigated for an inherited form blindness. In 2020, the first volunteer was subject to the experimental treatment.

He Jiankui’s work wasn’t published. It was rejected by all the major medical journals to which it was submitted. Antonio managed to get hold of the manuscript and showed it off to four experts. Their verdicts were shocking. His claims were not supported, and the parents of the babies may have been pressured to participate in the experiment. The researchers continued without fully understanding what was happening.

While the summit was primarily focused on human genome editing and CRISPR, it is also being explored for making farmed animals stronger and more powerful. In an effort to make catfish more resistant to diseases, a team of scientists have introduced an alligator gene to them.

From all over the web

A microbiologist discovered that a forgotten beef soup had turned bright blue in her fridge. So she embarked on a scientific search to discover why. (Twitter)

All over the globe, governments use algorithms to restrict access to certain services. An investigation found that a system that flags Rotterdam residents suspected of benefit fraud appears to discriminate against those who are ethnically or genderally different. (Wired)

Retro Biosciences, a biotech company, announced its launch last year with $180 million of funding. All of this is actually from Sam Altman (CEO of OpenAI). (MIT Technology Review)

The company that makes Makena, a drug that prevents preterm births, has pulled it from the market. Numerous studies have shown that the drug does not work. The US Food and Drug Administration recommended that the drug be withdrawn in 2020. (The New York Times).

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