U.K. Scientists Are Deciding the Future of Humanity
Layla Richards was a bouncy 7 pound, 10 ounce 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 fuss and 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 (GOS) Hospital, 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 immune system 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, 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 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
