Thursday, 13 June, 2024
HomeEthicsCall for guidelines and ethical boundaries in genetic editing therapies

Call for guidelines and ethical boundaries in genetic editing therapies

While progress in the field of genetics is being lauded as heralding endless possibilities for the future, reservations have been expressed by some experts who question how guidelines will be established and ethical boundaries set.

“A world where gene editing becomes commonplace will have a detrimental impact on family dynamics, society and the economy,” believes Professor Letlhokwa George Mpedi.

Writing in Daily Maverick, the vice-chancellor and principal of the University of Johannesburg says the challenges posed by these stunning breakthroughs in science, technology and medicine – like the gene-editing technique CRISPR, and advances in artificial intelligence and robotics – will deliver amazing, life-saving benefits, “but could create savage meritocracies that resemble apartheid, and massive unemployment, including to those in the current professional elites”.

The effectiveness of gene editing in treating the blood-clotting disorder sickle cell disease had already been seen, he said.

"But beyond the benefits and the socioeconomic impact, what are the ethical and indeed legal implications of reframing our creation of humans – are we bordering on eugenics with designer babies, with the implied risk of marginalising or even eradicating groups?”

How do we establish gene editing guidelines to ensure the process is ethical? Where do we draw the line?

His views are echoed by others, with some world leading experts believing that apart from the next generation of advanced genetic therapies raising profound medical and ethical risks, another danger “is that the people who would benefit most will not be able to access them”. These, they feel, must be thrashed out to ensure the game-changing technology benefits both patients and society.

Medicines based on these powerful tools will start transforming the treatment of blood disorders, conditions affecting the heart, eyes and muscles, and potentially even neurodegenerative diseases before the end of the decade, but the cost will put them out of the reach of many patients, they believe.

Trials of gene editing in embryos will probably follow, and while the procedure has limited clinical applications, some fear fertility clinics could embrace the technology and offer gene editing services that fuel “a new kind of techno-eugenics”.

Speaking ahead of the Third International Summit on Human Genome Editing, held recently at the Francis Crick Institute in London, Professor Jennifer Doudna, who shared the 2020 Nobel chemistry prize for gene editing, said: “We’ll definitely be seeing genomic therapies for heart disease, neurodegenerative diseases, eye conditions and more, and possibly some preventative therapies as well.”

The summit outlined how the technology has evolved in the five years since the last summit. For instance, prime editing was introduced, which calls for only a single-stranded cut in DNA instead of the double-stranded cut in current processes. This technique can potentially treat genetic conditions such as Huntington’s disease and Friedreich’s ataxia, and is more versatile and precise.

But she warned: “One of the biggest and most realistic risks is that the people who could benefit most won’t be able to access or afford them.”

Doudna and the other experts who talked to The Guardian were on the summit’s organising committee but shared their thoughts in a personal capacity.

Professor Françoise Baylis, a philosopher at Dalhousie University in Canada, said the new therapies would be prohibitively expensive for much of the global population, which could “seriously threaten” the aspiration for all humans to be born equal.

The experts, ranging from geneticists and public health researchers to bioethicists and philosophers, expect a wave of gene editing therapies to reach clinics in the next five years or so. These will correct disease-causing mutations in patients’ tissues and organs and become more sophisticated as researchers discover how to make multiple edits at once and reach difficult areas, like parts of the brain affected by neurodegenerative disease.

Regulators in the US, Europe and the UK could approve the first genome editing therapy later this year, as a treatment for sickle cell disease.

The same technology paves the way for therapies to enhance healthy humans, to make them faster, smarter, stronger, or more resistant to disease, though enhancement is trickier than mending single faulty genes, says Professor Ewan Birney, joint director of the European Bioinformatics Institute near Cambridge. “It is far harder to know the edits which will ‘improve’ rather than ‘fix’,” he added.

Regardless, some see it as inevitable. Professor Mayana Zatz at the University of São Paulo, Brazil and founder of the Brazilian Association of Muscular Dystrophy, said she was “absolutely against editing genes for enhancement”, but added: “There will always be people ready to pay for it in private clinics and it will be difficult to stop.”

Baylis believes genetic enhancement is “inevitable” because so many of us are “crass capitalists, eager to embrace biocapitalism”.

The previous summit, held in Hong Kong in 2018, was marred by controversy when Chinese scientist Jiankui He revealed he had edited DNA in three embryos which developed into babies, including twin sisters named Lulu and Nana. He intended to make the children immune to HIV, but was roundly denounced as reckless by the scientific community. He was jailed for three years for breaking Chinese laws but has since sought to relaunch his career.

Some experts told The Guardian gene editing might be safe enough to trial in human embryos in the next 10 to 20 years, though Professor Luigi Naldini, a gene therapist at Vita-Salute San Raffaele University in Milan, said niche therapies might “technically” be ready within five years.

Such treatments would aim to prevent devastating heritable diseases, including those that are fatal in the first years of life. But geneticists stress there are almost always alternatives, like genetic screening of IVF embryos, adoption, or donor eggs or sperm.

One reason for caution is that so-called germline editing in embryos alters the DNA in the sperm or eggs of the resulting adult, meaning any edits – including errors – are passed on to future generations. An alternative being worked on performs gene editing on foetuses in the womb.

At millions of dollars a shot, gene editing today is prohibitively expensive. But if costs fall substantially in coming decades, there is a risk IVF clinics could start offering services, whether the benefits are proven or not.

Birney speculates that clinics could offer combined IVF, embryo screening and gene editing packages, which would be “misguided”.

“It is important countries work out how to regulate this before it becomes a more credible prospect,” he said.

And Mpedi said: “Technology is undoubtedly evolving, and we need to evolve alongside it. But we must consider various ethical concerns, including humanity’s ugly history of eugenics, and the potential for harm.”

Human rights

In December 2018, the World Health Organisation (WHO) established a global, multi-disciplinary expert advisory committee to examine the scientific, ethical, social and legal challenges associated with human genome editing.

The committee made a set of nine recommendations, including:

• The WHO taking a distinct scientific and moral leadership stance by being open about the opportunities and challenges while acknowledging ethical concerns;
• An emphasis on international collaboration for effective governance and oversight;
• Human genome editing registries that include reviewal and approval by a research ethics committee;
• Human genome editing research should only take place in jurisdictions with domestic policy and oversight mechanisms;
• Encouraging reporting illegal, unregistered, unethical or unsafe research and other activities;
• Determine intellectual property rights;
• Create platforms for education, engagement, and empowerment;
• Create a set of officially endorsed and clearly defined ethical values and principles for use by WHO; and finally
• Periodical reviews of these recommendations should take place to keep up with the pace of technology.

Added Mpedi: “Although a step in the right direction, our legislation also needs to respond accordingly. How do we tap into the benefits of gene editing without violating human rights? As this technology becomes more entrenched and presents us with new possibilities, we must have the right ethical and legal frameworks in place.”


Daily Maverick article – Gene editing raises profound moral questions on ethics, eugenics and human rights (Open access)


The Guardian article – Forthcoming genetic therapies raise serious ethical questions, experts warn (Open access)


See more from MedicalBrief archives:


World’s first gene-edited babies’ creator relaunches career after prison release


Red flags after volunteer’s death in US gene editing study


Gene-editing researchers jailed over illegal medical practices


Gene-editing to eliminate HIV DNA a significant step towards human clinical trials





MedicalBrief — our free weekly e-newsletter

We'd appreciate as much information as possible, however only an email address is required.