BABY MAKERS: How British scientists are deciding the future of humanity

Pioneering gene editing research has put the UK at the forefront of the next scientific revolution. Madhumita Murgia wonders if the rest of the world is ready for it or not.

BABY MAKERS: How British scientists are deciding the future of humanity

LAYLA Richards was a bouncy, 7lb 10oz baby, with downy dark hair and plump cheeks. She was born in a London hospital in June, 2014.

But 12 weeks later, Layla, who had been settling in at home in North London, stopped drinking milk and began to fuss and cry constantly.

Because she had been a sunny, happy infant, her parents took her to the doctor. He suspected a stomach bug, but he took a blood test.

The results that came back a few days later were a shock: Layla had an acute, deadly form of leukaemia. She needed immediate treatment. She was 14-weeks-old.

An ambulance rushed the family from their home to intensive care at the Great Ormond Street (GOS) Hospital, the world-famous paediatric centre in Bloomsbury.

Her doctors described her cancer as “one of the most aggressive forms of the disease” they had ever seen. For the next few weeks, she endured several rounds of chemotherapy, followed by a full bone marrow transplant to replace her damaged blood cells.

This aggressive therapy can often be successful in babies, but none of Layla’s treatments, not even the experimental ones, worked. Medically, she was out of options. Only one choice remained — admitting her to an end-of-life care facility to make her final weeks more comfortable.

Just a few doors down from the leukaemia ward at Great Ormond Street Hospital is the office of Dr Waseem Qasim, a bearded, genial immunologist. He specialises in immune system disorders in children, including cancers.

For several months, Qasim had been working on a new type of leukaemia treatment: an anonymous donor’s white blood cells are engineered to recognise cancer cells, by tweaking their genes.

White blood cells are the body’s soldiers; they fight infectious disease and foreign invaders. The engineered cells form an arsenal of targeted cancer-killer cells that can be injected into anyone. There was one problem: The procedure had only been tested in mice.

Qasim’s lab is based in the University College London GOS Institute of Child Health, which is connected by a single corridor to Great Ormond Street.

“We move effortlessly between the two. There are no other physical or intellectual barriers, so it leads to serendipitous events,” Qasim says, as we stroll through a set of double doors from his lab into the hospital. Qasim heard about Layla’s case from her transplant surgeon.

“He asked, as a sort of joke, ‘I might be out of my mind, but could [your cells] be useful here’?” Qasim recalls.

Because the therapy had never been tested in humans, there was the danger of something going badly wrong, but Layla’s parents and doctors knew she would die without a miracle.

After the Medicines and Healthcare products Regulatory Agency granted an emergency licence, Layla became the first person in the world to receive a single vial of gene-edited cells from a stranger to attack her cancer.

What followed, after Qasim’s experimental gene treatment, a new technique using custom-designed molecular scissors to cut, edit, and delete DNA, was described by Layla’s doctors as “miraculous” and “staggering.”

She went into remission within four weeks and successfully survived a second bone marrow transplant. Now, two years on, she remains healthy and cancer-free.

Little Layla was a pioneer, the first person saved by gene-editing; and without the favourable environment created by British scientists and regulators over the past decade, Qasim’s experimental treatment, which gives special properties to cells, would never have been allowed.

With advances in gene-editing and governmental approvals, the UK is set to become the unlikely pioneer in one of the most controversial, yet astonishing, spheres of human knowledge: the manipulation of our genetic code.

While research labs around the world are working on genetic cures to childhood and adult diseases, most have been wary of interfering with the DNA of a human embryo, fearful of unintended consequences for future generations.

Yet, the UK achieved a double first in 2016: It became the first country to legally permit replacing part of an embryo with a third person’s genes, and the first to allow genetic modification in humans from the embryo stage.

The techniques’ opponents, including bioethicists and religious leaders, say they herald a dystopian future of ‘designer babies’ — a world where parents will ‘play God’ by editing their unborn child’s genes to make it stronger, taller, and healthier.

Molecular biologist and ethicist, David King, the founder of British watchdog group, Human Genetics Alert, says that embryo manipulation opens up, “for the first time in human history, the possibility of consciously designing human beings, in a myriad of different ways.”

A recent report from the Nuffield Council on Bioethics, in London, found that gene-editing — particularly in embryos — demanded further scrutiny. Ethical opposition has arisen especially, where, it said, the “scope for unforeseen consequences is considered to be great or editing is regarded as irreversible.”

All humans have a unique “genome” sequence, the more than three billion molecule pairs, known as DNA, that define who we are, from our physical appearance to biological characteristics and even our personality.

Our hair colour, our preference for certain kinds of food, even our ability to make deadlines — it’s all rooted in our DNA. Mutations, or mistakes, in this genetic code can result in disease, such as diabetes or leukaemia.

Because of gene-editing, we can now find and correct genetic errors in a lab. Once honed, the tools could be used to cure maladies like sickle cell anaemia and cystic fibrosis, and even cancer.

The promise of gene-editing goes beyond curing adult disease — it could be used to modify human embryos and delete egregious genetic defects before birth. That would prevent the transmission of debilitating illnesses from parent to child, and may end devastating inherited disabilities.

The British government’s endorsement of gene-editing research has thrust the country to the forefront of the next revolution in health and science, whether the rest of the world is ready for it or not.

Gene Genies

Decades before Layla Richards was born, another baby girl made history in Britain. In July, 1978, Louise Brown was born by Caesarean section.

There was nothing unusual about the birth of this healthy, 5lb 12oz baby — and yet, her arrival into the world helped two British scientists win a Nobel Prize.

The reason: Louise was conceived in a petri dish, the world’s first baby created by in vitro fertilisation (IVF). Louise was called the first ‘test-tube baby,’ an indication of how bizarre the now-standard procedure was at the time.

Louise’s immaculate-lab conception is part of the UK’s long history of developing groundbreaking biotech. That legacy began at the University of Cambridge in 1953, when doctoral students, Francis Crick and James Watson, cracked DNA’s double-helix structure, forever reshaping our understanding of human biology.

The simple, two-strand configuration — drawn by hand by Crick’s wife, Odile, in their original Nature paper — gave rise to the entire field of modern molecular biology, and it spawned cutting-edge techniques, from cloning to gene-editing.

British researchers have pioneered clinical techniques in reproductive biology, including IVF, the discovery of embryonic stem cells in mice (1981), and the first cloning of a mammal, Dolly the sheep (1996).

With each of these milestones, scientists around the world faced a moral dilemma about the definition of human life. When does a ball of cells become a foetus?

Does an artificially created life-form have rights? Should physical impairments, like deafness, be culled from our population?

After the birth of Louise Brown, the British government convened an ethical committee, headed by philosopher, Mary Warnock, to investigate the implications of creating and modifying human life in a lab. The resulting report, published in 1987, led to a nationwide consensus on the obvious social benefits of IVF.

The report also led to the establishment of the Human Fertilisation and Embryology Authority (HFEA), the first independent, legislative body in the world to regulate human embryo research and IVF treatment.

It is overseen by an independent board, rather than government ministers, but is sponsored by the British Department of Health, whose head appoints the board.

Members include geneticists, philosophers, former civil servants, and finance and business professionals. The chair, Sally Cheshire, reports directly to the UK’s minister for health.

The HFEA is a symbol of Britain’s commitment to innovation in medical science — unique in its progressive nature, compared to other advanced nations, like the US and Germany, where religion and politics often hold back research.

HFEA recently granted two controversial licences: In February, 2015, the British government approved a pioneering gene technology to prevent potentially fatal mitochondrial disease from passing from mother to child.

By placing a donor’s healthy genes in an IVF embryo, the researchers say the resulting baby could avoid severe symptoms, such as deafness,

muscle withering, liver or kidney failure, and brain damage. But critics worry that when these babies pass on the new genetic code to their children, grandchildren, and every subsequent generation, there will be as-yet-unknown consequences.

Despite vocal opposition from a smattering of members of parliament, as well as challenges from the Church of England and the Catholic clergy, the British House of Commons voted by an overwhelming majority to allow this mitochondrial donation.

And although the process has the UK government’s stamp of approval, it is not approved as safe and effective by the US or Chinese authorities. In a review of the technology earlier this year, the US Food and Drug Administration warned that the evidence does not yet support the safe use of mitochondrial transfer in humans.

In February, 2016, geneticist Kathy Niakan, of the UK’s Francis Crick Institute, became the first scientist in the world to receive a licence to edit healthy human embryos for research. (The embryos cannot be implanted into a human.)

Her goal is to better understand the process of early human development, not to redesign babies. Even so, some lawmakers were determined to prevent this sort of research in Britain.

In a parliamentary debate about the licence, Conservative Party parliamentarian, Jacob Rees-Mogg, said: “In a country nervous about genetically modified crops, we are making the foolhardy move to genetically modified babies.”

Key figures at the US National Institutes of Health (NIH) have similar concerns. At a gathering of scientific experts in July, to discuss the implications of gene-editing, NIH director, Francis Collins, underlined his agency’s reluctance to fund research into modification of human embryos. This is because of ethical concerns.

Collins has acknowledged that his religious beliefs prevent him from backing gene-modification in human embryos. “I do believe that humans are, in a special way, individuals and a species with a special relationship to God, and that requires a great deal of humility about whether we are possessed of enough love and intelligence and wisdom to start manipulating our own species,” he said in a recent interview with Buzzfeed News.

Niakan, and Robin Lovell-Badge, a scientific adviser to HFEA and the US National Academy of Sciences on issues of gene-editing, became the first scientists at the newly opened Francis Crick Institute in London (named in honour of the renowned Briton). It is the very first research hub to be granted an HFEA licence to edit seven-day-old, living human embryos).

Their work will focus on improving the success rates of IVF, the technique successfully demonstrated just 200 miles north, when Louise Brown was born in 1978.

The team that pioneered in-vitro fertilisation, on July 25, 1978: physiologist Dr Robert Edwards holding the world’s first test tube baby, Louise Joy Brown, with, right, gynaecologist Patrick Steptoe, at Oldham General Hospital. Picture: Keystone/Getty Images
The team that pioneered in-vitro fertilisation, on July 25, 1978: physiologist Dr Robert Edwards holding the world’s first test tube baby, Louise Joy Brown, with, right, gynaecologist Patrick Steptoe, at Oldham General Hospital. Picture: Keystone/Getty Images

Every Conception a Miracle

Headed by Nobel Prize–winning biologist, Paul Nurse, Francis Crick’s eponymous research institution opened in September. It’s an imposing edifice, with glass atria and a distinctive, vaulted roof, mirroring the nearby St Pancras International train station.

With an investment of €800m and known to insiders as Sir Paul’s Cathedral, the building will house 1,250 scientists in four interconnected blocks, making it the largest biomedical research institute in Europe.

Within its corridors, British scientists will be the first people to glimpse the molecular mysteries that result in the conception of human life.

The woman at the vanguard of this effort is 38-year-old Niakan, petite and dark-haired, with a birdlike face. When she greeted me in May, in her temporary lab in the Mill Hill neighbourhood of north London, she could easily have been mistaken for an undergraduate, in her leggings and knitted jumper.

As she described what an early human embryo — or blastocyst — looks like on day five of its existence, she grabbed a scrap of paper and began to sketch. Every so often, she punctuated the illustration with circled numbers to show the low survival rate of IVF embryos: Only 40% of fertilised embryos become blastocysts, of which only 50% will implant in a woman’s uterus. Another 50%, she said, fail to make it past three months of development.

Right now, we have little idea why embryos fail so often. When I said that, statistically, it seemed miraculous that humans had been reproducing successfully for centuries, she exclaimed, “I know, right?”

The daughter of Iranian immigrants, Niakan grew up in the small town of Silverdale, Washington, where her father was a neurologist. She became interested in genetics as a first-year student at the University of Washington, in Seattle, where she begged to be allowed to wash dishes in a lab that studied congenital diseases in large families; the lab allowed her to assist researchers studying human genetics, and she eventually discovered the gene responsible for a type of thalassemia, a genetic blood disorder.

“I remember being in a genetics class and there was the textbook view of all the different DNA bands, and then I was in the lab, physically. I was literally reading out, ‘That’s an A. That’s a T. That’s a G, C.’ I just loved it,” Niakan says, recalling her earliest experiences of DNA sequencing.

“I was hooked, and since then I haven’t stopped.”

Niakan has studied developmental biology at the University of California, and Harvard University, in the US, and at the University of Cambridge, in the UK, where she moved in 2009 as a postdoctoral fellow.

“I really love trying to understand how you go from a single-celled organism to this really complicated set of three, distinct cell types that are very set in their ways. That’s, in fact, the same question I’m still asking, many years later,” she says.

“The UK has very proactive ways of approaching reproductive health and medicine — it’s brilliant and it’s the reason why I’ve stayed for so long.”

Niakan’s goal is to understand the earliest stages of a human life, when we are nothing but a ball of 200 cells. She knows her work could help women to conceive and could eradicate genetic diseases, but these are not what drives her. Her motivation is cracking the scientific mystery of human reproduction.

“It has the potential to really revolutionise our understanding of human biology, in a petri dish,” she says. “That’s fascinating to me.”

Using a gene-editing tool called CRISPR-Cas 9 (pronounced “crisper”), which can cut and edit DNA precisely, she wants to isolate genes thought to be important for foetal development; only then can we figure out what role each plays.

“This basic biological question — which genes are critically required? — is important, because it can help us understand which blastocysts will go on to develop, implant, and thrive.”

Today, when a woman comes into a clinic for IVF treatment, experts score her embryo quality based on physical shape, size, and other visible features, rather than genetic features. Although embryos can be screened for chromosomal abnormalities, little is known about human developmental genetics.

“There are very few molecular tools used to identify those embryos. We know there’s a 50% drop-off rate, so I think there’s room to determine the key signatures that embryos need to successfully implant,” Niakan says.

“It could increase the chances [of pregnancy], or it could help to choose those embryos that will likely go on to develop successfully into a healthy baby.”

Eventually, the knowledge could help us fathom causes of reproductive defects or even infertility.

The HFEA spent three years investigating Niakan’s request to use the CRISPR-Cas9 scissors, conducting a series of detailed inspections of her lab work, including whether embryos were handled respectfully and carefully in the lab, and if donors were counselled and updated appropriately.

Niakan was notified of their decision in late January. The overwhelming feeling wasn’t excitement or even elation, she says; she had just been afraid that irrationality and fear of the unknown would win out over science.

The decision was celebrated by scientists, patient groups with genetic diseases, and mothers who had struggled to conceive.

Emma Benjamin, a 34-year-old woman who miscarried four times, spoke to the press of her support.

“I found it frustrating I never had answers as to why I kept miscarrying,” she said. “If this research had come earlier and could have helped me provide answers, then I guess, you know, it could have, maybe, saved a lot of heartache.”

Baby Steps

The new, £650m Francis Crick Institute in London, the first research hub licensed to edit seven-day-old, living human embryos. Picture: Daniel Leal-Olivas/AFP/Getty Images
The new, £650m Francis Crick Institute in London, the first research hub licensed to edit seven-day-old, living human embryos. Picture: Daniel Leal-Olivas/AFP/Getty Images

Despite Niakan’s momentous victory, it remains illegal in the UK to implant genetically modified embryos into a womb for the purpose of giving birth. That ensures that modified genes are not passed onto future generations; Niakan’s lab must destroy every embryo after the seven-day mark.

Although Niakan insists this research has no bearing on actual babies (for now), many in the scientific community are considering the possibility that a modified embryo could result in a living child.

In December, 2015, several hundred scientists from around the world gathered in Washington, DC, for the first ever international summit on gene-editing. At its close, the event chair and Nobel Prize–winning biologist, David Baltimore, of the California Institute of Technology, issued its conclusions, saying: “As scientific knowledge advances and societal views evolve, the clinical use of germline [embryo] editing should be revisited on a regular basis.”

The scientists have reason to be anxious: Some of their brethren have raced ahead already. In April, 2015, researchers in Guangzhou, China, announced they had conducted a CRISPR gene-modification experiment on defective human embryos, to edit the gene responsible for beta-thalassaemia, a potentially fatal blood disorder. It was a resounding failure, because the CRISPR method accidentally edited the wrong genes, which ended up irreversibly scrambling the embryo’s DNA.

That research sparked a hot global debate in the academic fraternity about whether to declare a moratorium on embryo modification, until ethical laws and regulations could catch up with science.

In response, scientists from the United States, Britain, and China, at the Washington summit, called for a temporary freeze on altering human embryos destined for birth, calling it “irresponsible” and potentially dangerous. The quick decision to cooperate internationally speaks to the transnational nature of this research; this is a strand of science that could change what it means to be human.

Even gene-editing’s strongest proponents acknowledge that there could be catastrophic mistakes. For instance, CRISPR could edit genes inaccurately, causing unintended mutations and disfigurations.

There’s also the very real risk of rogue editing by malicious parties — wealthy people paying for genetic enhancements. This could become a form of social discrimination and could introduce novel genetic sequences into the species — a sort of genetic cosmetic surgery. Until these safety and ethical issues have been resolved, the scientific community proposed holding back, and constantly reassessing, current research.

Rumours in academic circles of several, other Chinese experiments on human embryos sparked worries of an unregulated black market of clinical research. In a parallel case, the US has no laws governing private research on embryos, despite federal funding sanctions, meaning embryos end up being traded like contraband.

“There’s a billion-dollar IVF enterprise in private clinics, many of whom are using techniques that are dubious, to say the least,” Lovell-Badge says. “There are strict rules against implanting more than two embryos in a woman. Yet there are famous cases in the US, like the ‘Octomom’ who had eight babies, where they clearly couldn’t have followed any regulations at all.”

While most scientists acknowledge that editing embryos will probably be a clinical option one day, some remain staunchly opposed. King, of Human Genetics Alert, refers to gene-editing as the “new techno-eugenics.”

Lovell-Badge believes frank discussion and public trust in the HFEA are the key to a safe, clinical transition. “It is illegal in the UK to transfer any gene-edited embryo into a woman,” he says.

“Given the experience with the way the HFEA regulates [this research], and if the law were to be changed, I expect the public could also be reassured that any applications would be restricted to important clinical uses.”

Niakan agrees, pointing to UK regulators’ ability to separate church and state in the matter of controversial scientific research, such as hers.

“The UK’s pioneering role in advancing reproductive medicine and health, especially IVF, has a lot to do with the regulatory framework, where people are willing to engage in frank discussions about these complex issues,” Niakan says. “In other countries, the message gets muddled up with politics and religion.”

Although embryo-editing remains firmly confined to laboratories, scientists at Newcastle University, in the north of England, are taking the next step into the future by genetically modifying IVF embryos to create healthy babies.

In September, the world’s first baby with three people’s genes was born in Mexico, to Jordanian parents who had lost two children and had four miscarriages due to mitochondrial disease. The genetic illness is caused by dysfunctional mitochondria, the cellular units that are responsible for generating energy.

In the case of this baby, the malfunction was caused by mutations, or errors, in the mitochondrial DNA. The procedure was performed by a team of doctors from New York City, although details on how it was done are scant. The only country with any legal or regulatory framework for the technology is the UK, where — as of December, 2016 — an embryo can legally be modified, and implanted into a woman’s uterus.

Until Rachel Steel turned 20, she knew nothing about IVF regulations and cared even less. She competed as a gymnast as a child, studied pediatric nursing at Northumbria University, in Newcastle, and taught gym to children in her neighbourhood. She wanted to have children before she was 30. The only health problems she’d ever had were ear infections, which caused a slight difficulty in hearing. She compensated by lipreading.

Doctors could never quite pinpoint the cause of her ear problems. But five years ago, Steel learned she had a genetic mutation in her mitochondrial DNA.

Doctors at the Royal Victoria Infirmary, in Newcastle, where Steel, 26, is a nurse, first suspected something odd when her mother was brought in for a pancreatic transplant, following a kidney transplant some years before.

“They realised that all of her five siblings had diabetes and some mild deafness and found it strange,” she says. When they did a genetic test, they found Steel’s mother had mitochondrial disease. Since mitochondria are passed on exclusively from mother to child, her daughters had inherited her mutation.

The vast majority of your 20,000 genes are found in the nucleus of each of your cells, which contains DNA from both your parents, but mitochondria have their own genome, which carries only about 37 genes and is inherited from your mother alone.

The severity of mitochondrial disease depends on the fraction of mutations in the 37 genes inherited; in Steel’s case, the news was not good. She had inherited 80% of her mother’s mutations. Steel remains mostly healthy, but her disease could progress to anything from diabetes to full-blown hearing loss, or extreme muscle deterioration.

If Steel has children, they could be even more severely afflicted. “When I was younger, I thought, ‘Oh, it affects babies, that’s bad’. But I didn’t think it actually applied to me,” Steel says.

About one in 200 babies in the UK are born with mitochondrial disease. In the US, the percentage is lower, at roughly one in 1,000 babies; many only live a few hours, while others begin to rapidly sicken after a few years, suffering from brain, heart, or kidney disease. There is no cure for mitochondrial disease.

For women who have the condition and want to have children, the only options are to get pregnant and then screen out affected embryos — a heartbreaking process for would-be parents — or have an IVF baby using a donor egg.

The man fighting hardest for Steel’s future is her 64-year-old doctor, Sir Douglass Turnbull, who has been specialising in mitochondrial disease for 35 years.

“Several of my patients I’ve known for 20 or 30 years, along with their entire families,” he says in his lab at Newcastle University.

“There can be three generations in a family that are affected, many of whom lose three or four children due to the disease. For me, that’s the biggest motivation.”

Since 2001, Turnbull, along with Newcastle embryologist, Mary Herbert, has been working on a new IVF technique, known as mitochondrial donation, which offers women like Steel — 2,500 of whom have been identified in the UK alone — a way to have biological children who do not have their mother’s mutations.

The technique is like swapping the yolk of an egg: It involves removing a healthy nucleus, or yolk, of the mother’s fertilised egg, which contains about 99.8% of genetic material that the child will inherit. This is transferred into the egg of a donor that has had its nucleus removed. The donor, who does not have mitochondrial disease, will pass on her healthy mitochondria. This way, the baby will inherit the vast majority of its biological characteristics from its parents, via its nuclear DNA, but will have the healthy mitochondrial genes of the donor.

In a paper published in Nature this past summer, Turnbull and Herbert found that their technique could reduce to under 5% the risk of passing on defective mitochondrial DNA, which is far better than the 60% to 90% risk otherwise.

“For people who just watch their child fall apart before their eyes, this is a hugely positive outcome,” says Herbert.

The Newcastle-based scientists started lobbying the HFEA, to approve their technique, in 2012, and came up against intense opposition. Because the mitochondrial transfer method passes on genetic change from one generation to another, British MPs, and even some scientists, worried that it could give rise to unexpected problems.

Catholic Church ethicists were also opposed to the introduction into an embryo of a third person’s genes, arguing that this “dilutes parenthood.”

The Newcastle team argues that since the donor remains anonymous and has no rights over the child, she shouldn’t be considered a third parent. Other critics are uncomfortable with deleting disability out of the population, believing it would impact on the rights of the handicapped.

Bioethicist Tom Shakespeare, who has dwarfism and uses a wheelchair, doesn’t believe ‘fixing’ genetic mutations is necessarily what the disabled community wants, although he doesn’t oppose mitochondrial donation, in principle.

“Contrary to the prevailing assumption, most people with disabilities report a quality of life that is equivalent to that of non-disabled people. Their priority is to combat discrimination and prejudice,” he writes in a paper in Nature.

Fellow bioethicist and deaf researcher, Jackie Leach Scully, is uncomfortable about genetic cures as a solution for all disabilities, although she says it would be hard to find anyone opposed to correcting mitochondrial mutations, which are “generally very nasty diseases.”

The Newcastle-based scientists strongly object to this reasoning — they believe every mother with genetic disease should have a choice between hoping for the best or using science to screen for a healthy baby.

“We are often criticised, because we don’t value disability. I don’t think that at all. I spend my whole life looking after disabled people, but people should have the right to decide whether or not they want to have disabled children,” says Turnbull.

In the US, fertility doctors in New Jersey performed a crude version of this technique in the 1990s, which led to the births of at least 50 babies, in the US, Israel, Taiwan, and Italy.

Many are healthy today, but the US federal Food and Drug Administration banned the technique in 2001, because of concerns about unexpected genetic defects and reduced fertility in the women born this way.

During the five-year debate in the British parliament over this technique, patients, including Steel and even those beyond their reproductive years, went to the House of Commons to add their perspective to the discussion, explaining what they wanted.

“I 100% want a family,” Steel tells me. “It’s not to say I can’t go naturally ahead, having children, but there’s a huge risk and that’s a risk I wouldn’t take.”

Curing the Incurable

While scientists are still fighting to get approval to test their cutting-edge biomedical techniques before using them on humans, Qasim, the immunologist at UCL, is saving more lives — and saving parents from the ultimate tragedy.

Around Christmas, 2015, months after Layla Richards was sent home in remission, Qasim’s team obtained a second emergency licence to treat another baby girl with the identical type of leukaemia, which had been diagnosed when she was just four weeks old.

When she was 16 months old, the child (whose parents did not want to make her name public) was given the same dose of gene-edited killer cells Layla received. Weeks later, she was declared cancer-free; now, at two years and four months old, she is doing well.

Qasim’s emergency treatment, which has now saved two children, is part of a larger trial that opened to the public in June. It will treat up to 10 children with the same type of leukaemia as the two toddlers who are in remission. If the treatment works for the 10 new patients, the introduction of modified genes could become the primary treatment for cancers like this, supplanting even chemotherapy.

With a slight tweak, Qasim says, this gene therapy could be applied to other cancers, and even genetic diseases like thalassemia. Gene therapies are already being tested for those conditions, so the timeline for fixing a wide range of genetic defects could be as short as five years, he says. The therapy could even be used for diseases considered incurable, like HIV. American pharmaceutical firm, Sangamo, is running a trial that uses gene-editing to engineer the immunity of HIV patients to the disease.

Meanwhile, nearly two years on, Layla remains cancer-free and healthy. At a charity fundraiser for the Great Ormond Street Hospital in December, 2015, Layla’s mother encouraged other parents with sick children to be unafraid of “guinea pig” treatments, and to “try new things.”

If Qasim’s therapy is approved for general use, it could be the first of thousands of similar treatments. “Layla has a purpose — to help other people. She was nearly at death’s door. You don’t normally hear a happy story with cancer,” her father said during the appeal.

“One day, there will be a cure for cancer. Who knows? Maybe, in 40 years’ time, Layla may have helped to make the first step towards that.”

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