bouncy 7-pound, 10ounce baby with downy dark hair and plump cheeks when 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, sud­denly stopped drink­ing milk and be­gan to cry con­stantly. Be­cause she had been a sunny, happy in­fant un­til then, her par­ents took her to see the doc­tor. He sus­pected a stom­ach bug, but just to be sure he took a blood test. The re­sults that came a few days later were a shock: Layla had an acute, deadly form of leukemia that she couldn’t sur­vive without im­me­di­ate treat­ment. She was just 14 weeks old.

When the di­ag­no­sis came in, an am­bu­lance rushed the fam­ily from their home to in­ten­sive care at the Great Or­mond Street Hos­pi­tal (GOS), the world-fa­mous pe­di­atric cen­ter 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 sort of ag­gres­sive ther­apy can of­ten be suc­cess­ful in ba­bies, but none of Layla’s treat­ments, 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 leukemia ward at Great Or­mond Street Hos­pi­tal is the of­fice of Dr. Waseem Qasim, a bearded, ge­nial im­mu­nol­o­gist who spe­cial­izes in dis­or­ders in chil­dren, in­clud­ing can­cers. For sev­eral months, Qasim had been work­ing on a new type of leukemia treat­ment in which an anony­mous donor’s white blood cells are en­gi­neered to rec­og­nize 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. He 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 be­fore been tested in hu­mans, there was the ob­vi­ous dan­ger of things 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 li­cense, 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, nearly two years on, she re­mains healthy and can­cer-free.


LIT­TLE Layla was a pioneer, the first per­son saved by gene edit­ing; and without the fa­vor­able 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 re­cent ad­vances in gene edit­ing and gov­ern­men­tal ap­provals, the U.K. 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 U.K. 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.

Op­po­nents of the tech­niques, in­clud­ing the U.S. Na­tional In­sti­tutes of Health (NIH) as well as bioethi­cists and re­li­gious lead­ers, be­lieve they her­ald a dystopian fu­ture of “de­signer ba­bies”—a world where par­ents will opt to edit 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, be­lieves 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 3 bil­lion molecule pairs known as DNA that de­fine who we are—from our phys­i­cal ap­pear­ance to bi­o­log­i­cal char­ac­ter­is­tics and even our per­son­al­ity. Mu­ta­tions or mis­takes in this ge­netic code can re­sult in dis­ease, such as di­a­betes or leukemia. Gene edit­ing means we can now find and cor­rect ge­netic er­rors in a lab. Once honed, the tools could be used to fix mal­adies like sickle cell ane­mia and cys­tic fi­bro­sis, and to fight can­cer.

The prom­ise of gene edit­ing goes beyond cur­ing adult dis­ease—it could even 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 could sig­nal the end of dev­as­tat­ing in­her­ited dis­abil­i­ties.

The Bri­tish gov­ern­ment’s re­cent en­dorse­ment of gene edit­ing re­search 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.


NEARLY four decades be­fore Layla Richards was born, an­other baby girl made his­tory in Bri­tain. In July 1978, Louise Brown was born by Cae­sarean sec­tion to very ea­ger par­ents. There was noth­ing par­tic­u­larly un­usual about the birth of this healthy, 5-pound, 12-ounce 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 through the process of in vitro fer­til­iza­tion (IVF). Back then, Louise was called the first “test-tube baby,” an in­di­ca­tion of how bizarre the now-stan­dard pro­ce­dure was con­sid­ered at the time. In 1981, The New York Times wrote that the pro­ce­dure was con­sid­ered “equiv­a­lent to abor­tion in the eyes of some op­po­nents.”

Louise’s im­mac­u­late-lab con­cep­tion is part of the U.K.’S long his­tory of 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 pi­o­neere d 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 con­cern­ing the def­i­ni­tion of hu­man life. When does a ball of cells be­come a fe­tus? Does an ar­ti­fi­cially cre­ated life form have rights? Should phys­i­cal im­pair­ments like deaf­ness be culled from our pop­u­la­tion? Af­ter Louise Brown’s birth the Bri­tish gov­ern­ment con­vened an ethics com­mit­tee, headed by philoso­pher Mary Warnock, to in­ves­ti­gate the im­pli­ca­tions of creat­ing and modi- fy­ing 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 Fer­til­iza­tion 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 chair­woman, Sally Cheshire, re­ports di­rectly to the U.K.’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 U.S. and Ger­many, where re­li­gion and pol­i­tics of­ten hold back re­search.

The HFEA re­cently granted two con­tro­ver­sial li­censes: 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.

Al­though the process has the U.K. gov­ern­ment’s stamp of ap­proval, it is not ap­proved as safe and ef­fec­tive by the U.S. or Chi­nese au­thor­i­ties. In a re­view of the tech­nol­ogy ear­lier this year, the U.S. 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 U.K.’S Fran­cis Crick In­sti­tute be­came the first sci­en­tist in the world to re­ceive a li­cense 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 re­design ba­bies. Even so, some law­mak­ers were de­ter­mined to

“In a coun­try ner­vous about ge­net­i­cally mod­i­fied crops, we are mak­ing the fool­hardy move to ge­net­i­cally mod­i­fied ba­bies.”

pre­vent this sort of re­search in Bri­tain. In a par­lia­men­tary de­bate about the li­cense, 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 U.S. Na­tional In­sti­tutes of Health have sim­i­lar con­cerns, and since 1995 the United States has banned the use of fed­eral fund­ing for any hu­man em­bryo re­search. “It’s very dif­fi­cult to have a real de­bate about any is­sues to do with ar­ti­fi­cial con­cep­tion tech­nol­ogy or gene ma­nip­u­la­tion there be­cause every­one is scared the ar­gu­ment will be­come about abor­tion, which they don’t want to raise up against,” says pro­fes­sor Robin Lovell-badge, a sci­en­tific ad­viser to HFEA and the U.S. Na­tional Academy of Sci­ences on is­sues of gene edit­ing. “It cre­ates a ner­vous­ness [in the U.S.] which in the U.K. we don’t have, be­cause we know ex­actly where the bar­ri­ers are; they are set out by law and de­tailed reg­u­la­tions.”

De­spite its qualms about em­bryo re­search, the U.S. fed­eral gov­ern­ment does per­mit IVF treat­ments, which rely on hu­man em­bryo re­search to keep them cur­rent. “So they are re­ly­ing on re­search done in other coun­tries but won’t sup­port it them­selves,” Ni­akan tells me. “It doesn’t make any sense what­so­ever.”

Ni­akan, along with Lovell-badge, be­came the first co­hort of sci­en­tists at the newly opened Fran­cis Crick In­sti­tute in Lon­don—named in honor of the renowned Bri­ton, and the very first re­search hub to be granted an HFEA li­cense to edit seven-day-old liv­ing hu­man em­bryos. Their work will fo­cus on help­ing im­prove the suc­cess rates of IVF, the tech­nique suc­cess­fully demon­strated just some 200 miles north, when Louise Brown was born in 1978.


HEADED by No­bel Prize–win­ning bi­ol­o­gist Sir Paul Nurse, Fran­cis Crick’s epony­mous re­search in­sti­tu­tion opened in Septem­ber. It’s an im­pos­ing ed­i­fice with glass atria and a dis­tinc­tive vaulted roof. With an in­vest­ment of about $805 mil­lion, 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 ever 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 greets me in May in her tem­po­rary lab in the Mill Hill neigh­bor­hood of north Lon­don, she could eas­ily be mis­taken for an un­der­grad­u­ate in her leg­gings and knit­ted jumper.

As she de­scribes what an early hu­man em­bryo—or blas­to­cyst—looks like on Day Five of its ex­is­tence, she grabs a scrap of pa­per and be­gins to sketch. Ev­ery so of­ten, she punc­tu­ates the il­lus­tra­tion with cir­cled num­bers to show the low sur­vival rate of IVF em­bryos: Only 40 per­cent of fer­til­ized em­bryos be­come blas­to­cysts, of which only 50 per­cent will im­plant in a woman’s uterus. An­other 50 per­cent, she says, fail to make it past three months of de­vel­op­ment. Right now, we have lit­tle idea why em­bryos fail so of­ten. When I point out that, sta­tis­ti­cally, it seems mirac­u­lous that hu­mans have been re­pro­duc­ing suc­cess­fully for cen­turies, she ex­claims, “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...i just loved it,” Ni­akan says. “I was hooked, and since then I haven’t stopped.”

Ni­akan has stud­ied de­vel­op­men­tal bi­ol­ogy at the Univer­sity of Cal­i­for­nia, Har­vard Univer­sity and the Univer­sity of Cam­bridge in the U.K., where she moved in 2009 as a post­doc­toral fel­low. “The U.K. has very proac­tive ways of ap­proach­ing re­pro­duc­tive health and medicine,” she says. “It’s bril­liant and it’s the rea­son why I’ve stayed for so long.”

Her 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 ul­ti­mately help women to con­ceive and ge­netic dis­eases to be de­feated, but that is not what drives her. Her real mo­ti­va­tion is crack­ing the sci­en­tific mys­tery of hu­man re­pro­duc­tion. “It has the po­ten­tial to re­ally rev­o­lu­tion­ize 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”) that can cut and edit DNA very pre­cisely, she wants to iso­late genes thought to be im­por­tant for fe­tal de­vel­op­ment; only then can we fig­ure out ex­actly what role each plays.

To­day, when a woman comes in to 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 at this early stage. “There are very few molec­u­lar tools used to iden­tify those em­bryos. We know there’s a 50 per­cent dropoff 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-CAS 9 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 coun­seled 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 widely 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.”

“It’s very dif­fi­cult to have a real de­bate about gene ma­nip­u­la­tion be­cause every­one is scared the ar­gu­ment will be­come about abor­tion.”


DE­SPITE Ni­akan’s mo­men­tous vic­tory, it re­mains il­le­gal in the U.K. 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, D.C., for the first ever in­ter­na­tional sum­mit on gene edit­ing. At its close, the event chair­man, 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 ethics 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 in­ter­na­tion-

ally 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, which 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 re-as­sess­ing cur­rent re­search on a con­stant ba­sis.

Ru­mors of sev­eral other Chi­nese ex­per­i­ments on hu­man em­bryos in aca­demic cir­cles have sparked wor­ries of an un­reg­u­lated black mar­ket of clin­i­cal re­search. In a par­al­lel case, the U.S. 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 U.S., 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 is the key to a safe clin­i­cal tran­si­tion. “It is il­le­gal in the U.K. 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 U.K. reg­u­la­tors’ abil­ity to sep­a­rate church and state in the mat­ter of con­tro­ver­sial sci­en­tific re­search such as hers. “The U.K.’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 Bri­tain, 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 al­most 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 kids in her neigh­bor­hood. She wanted to have chil­dren be­fore she was 30, but was in no hurry. The only health prob­lems she’d ever en­coun­tered were ear in­fec­tions—which caused a slight dif­fi­culty in hear­ing. She of­ten had them grow­ing up and tended to com­pen­sate 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, works as a nurse, first sus­pected some­thing odd when her mother was brought in for a pan­cre­atic trans­plant, fol­low­ing a kid­ney trans­plant some years be­fore. “They re­al­ized 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 per­cent of her mother’s mu­ta­tions. Steel re­mains mostly healthy so far, 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 U.K. are born with mi­to­chon­drial dis­ease. In the U.S., the per­cent­age is lower at roughly one in 1,000 af­flicted ba­bies born ev­ery year; 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 spe­cial­iz­ing in mi­to­chon­drial dis­ease for 35 years. “Sev­eral of my pa­tients I’ve known for 20 or 30 years, along with their en­tire fam­i­lies,” he tells Newsweek 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, that of­fers women like Steel—2,500 of whom have been iden­ti­fied in the U.K. alone—a way to have bi­o­log­i­cal chil­dren who do not have the mother’s mu­ta­tions. The tech­nique is a bit like swap­ping the yolk of an egg: It in­volves re­mov­ing a healthy nu­cleus, or yolk, of the mother’s fer­til­ized egg, which con­tains about 99.8 per­cent 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 the risk of pass­ing on de­fec­tive mi­to­chon­drial DNA to un­der 5 per­cent, far bet­ter than the 60 to 90 per­cent 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 the idea of delet­ing dis­abil­ity out of the pop­u­la­tion com­pletely, be­liev­ing it would af­fect 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 feels par­tic­u­larly un­com­fort­able about ge­netic cures as a so­lu­tion for all dis­abil­i­ties, al­though she con­cedes 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 crit­i­cized 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 U.S., fer­til­ity doc­tors in New Jersey per-

Even gene edit­ing’s strong­est pro­po­nents agree that cat­a­strophic mis­takes are pos­si­ble.

formed a crude ver­sion of this tech­nique in the 1990s that led to the births of at least 50 ba­bies in the U.S., Is­rael, Tai­wan and Italy. Many are healthy to­day, but the 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. Since then, sev­eral Amer­i­can labs have ap­plied for clin­i­cal li­censes, just like Turn­bull and Her­bert, but U.S. reg­u­la­tors have shot them down. “Hu­man-sub­ject re­search uti­liz­ing ge­netic mod­i­fi­ca­tion of em­bryos for the pre­ven­tion of trans­mis­sion of mi­to­chon­drial dis­ease can­not be per­formed in the United States in FY 2016,” an FDA spokesper­son said in a state­ment.

Dur­ing the five-year de­bate in Bri­tish Par­lia­ment over this tech­nique, pa­tients in­clud­ing Steel—even those beyond their re­pro­duc­tive years—went to the House of Com­mons to add their per­spec­tive to the dis­cus­sion, ex­plain­ing what they wanted. “I one-hun­dred per­cent 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.”

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 li­cense to treat an­other baby girl with the iden­ti­cal type of leukemia, which had been di­ag­nosed when the girl was just 4 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 2 years and 4 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 leukemia as the two tod­dlers who are in re­mis­sion. If the treat­ment works, 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 treat­ment could even be used for dis­eases con­sid­ered in­cur­able, like HIV. Amer­i­can phar­ma­ceu­ti­cal firm Sang­amo is cur­rently 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.”


MIR­A­CLE BABY: Layla Richards be­came the first per­son to re­ceive a sin­gle vial of gene-edited cells from a stranger to at­tack her can­cer. She is now healthy.

GENE-IUS: Dr. Waseem Qasim’s ex­per­i­men­tal treat­ment uses cus­tom-de­signed molec­u­lar scis­sors to cut, edit and delete DNA. It has had stag­ger­ing re­sults.

FROM TEST TUBE TO TOD­DLER: Louise Brown was the world’s first baby cre­ated through in vitro fer­til­iza­tion.

MOLEC­U­LAR MAVENS: Sci­en­tists work at the Fran­cis Crick In­sti­tute in Lon­don, the first re­search hub to be granted a HFEA li­cense to edit seven-day­old liv­ing hu­man em­bryos.

GATE­WAY TO GATTACA: Some are un­com­fort­able with the idea of delet­ing dis­abil­ity out of the pop­u­la­tion, be­liev­ing it could hurt the rights of the hand­i­capped.

Newspapers in English

Newspapers from USA

© PressReader. All rights reserved.