A British baby is recovering after a world-first treatment for cancer.
Layla Richards had one of the worst cases of leukaemia her doctors had seen and, when all other treatments failed, her parents were told to expect the worst.
But they refused to give up and doctors at London’s Great Ormond Street Hospital got permission to give Layla a treatment so experimental that it had been tested only on mice.
Now, thanks to an infusion of 50million cells genetically engineered to hunt and kill the cancer, the disease has vanished and she is a happy, energetic and bubbly toddler.
Her ‘near miracle’ recovery paves the way for a revolution in the treatment of cancer.
Professor Waseem Qasim, one of Layla’s doctors, said: ‘We have only used this treatment on one very strong little girl and we have to be cautious about claiming this will be suitable for all children.
‘But this is a landmark in the use of new gene engineering technology and the effects on this child have been staggering. If replicated, it could represent a huge step forward in treating leukaemia and other cancers.’
It could help adults with cancer and lead to groundbreaking new therapies for cystic fibrosis and other genetic conditions.
Layla, who lives in north London with her parents Ashleigh Richards and Lisa Foley, was a happy and healthy baby when born in June last year. But she became ill at just three months old. Doctors suspected a tummy bug but a blood test revealed that she had acute lymphoblastic leukaemia, a cancer of the disease-fighting white blood cells particularly hard to treat in babies.
When chemotherapy and a bone marrow transplant failed, Layla’s condition was judged hopeless. She was expected to die within months. Miss Foley, 27, a dental receptionist, said: ‘We didn’t want to accept palliative care and so we asked doctors to try anything for our daughter, even if it hadn’t been tried before.’
ACUTE LYMPHOBLASTIC LEUKAEMIA: THE MOST COMMON CANCER IN CHILDREN
Acute lymphoblastic leukaemia (ALL) is the most common type of cancer affecting children. Around one in every 2,000 children will develop ALL, with most cases occurring between the ages of two and five. Symptoms of ALL usually begin slowly before rapidly getting severe. They include pale skin, tiredness, breathlessness, having repeated infections over a short space of time and unusual and frequent bleeding. Usually, the bone marrow in the body produces stem cells which are allowed to fully develop into other cells before being released into the blood. But in ALL, the bone marrow starts releasing large numbers of immature white blood cells. These are known as blast cells. As they increase, there is a corresponding drop in the number of red blood cells and platelet cells, causing symptoms of anaemia. Around 8,600 people - children and adults - are diagnosed with leukaemia each year in the UK.
Great Ormond Street was given permission to use a cell treatment being developed with Cellectis, a French biotech company. It involves arming blood cells taken from a healthy donor with cancer-fighting genes. It was so new that it had been tested only on mice and just one vial was available.
Layla was given 1ml of the cell treatment, an amount that would fit on the tip of a teaspoon, in a ten-minute infusion in June, when she was a year old.
Mr Richards, 30, a driver, said: ‘Layla didn’t notice anything was going on and was bouncing in her cot all the way through. It was scary to think that the treatment had never been used in a human before but, even with the risks, there was no doubt we wanted to try. She was sick and in lots of pain, so we had to do something.’
Initially, it was unclear if the treatment had worked but within weeks Layla started to show signs of recovery. A bone marrow transplant followed, to ensure the disease had been eradicated, and tests show her to be cancer-free. She is now home with her parents and eight-year-old sister Reya.
Professor Paul Veys, the lead doctor on Layla’s case, said: ‘As this was the first time the treatment had been used, we didn’t know if or when it would work, so we were over the moon when it did. Her leukaemia was so aggressive that such a response is almost a miracle.’
Layla is having regular checks to ensure that the cancer has not come back and it will be about a year before it will be known if she has been cured.
But her doctors are so excited about her recovery that they have released details now, although a full presentation of her treatment will be made at the American Society of Haematology’s annual conference in Florida next month.
Dr Andre Choulika, the chairman of Cellectis, said Layla’s story could herald the start of a revolution in cancer treatment.
Dr Matt Kaiser, of the leukaemia charity Bloodwise, cautioned that much more research was needed, but added: ‘The concept of training immune cells to specifically recognise and hunt out leukaemia cells is very exciting and in theory could provide a lifetime cure for these children.’
HOW SCIENTISTS MODIFIED DONOR CELLS TO MAKE THEM FIGHT CANCER
Layla Richards’ life was saved by blood cells that had been genetically modified to make them fight cancer. Scientists made three changes to the cells, which came from a ‘random’ donor in the US. First, they added genes that fight leukaemia, a cancer of white blood cells. These genes made a protein that zeroed in on the cancer and killed the diseased cells. Then, they used a cutting-edge technique called TALENs to switch off two genes. Switching off the first gene ensured the donor’s cells wouldn’t be rejected by Layla’s body – or by anyone else given the treatment in future. Turning off the second gene meant they weren’t wiped out by powerful drugs that were essential to her recovery. In June, Layla was given an infusion of 50 million of the genetically-engineered cells. Once in her body, they spent three months seeking out and killing the leukaemia. In September, when cells had killed as much of the cancer as they could, Layla was given a bone marrow transplant. The bone marrow is the body’s blood cell ‘factory’ and replacing faulty marrow with healthy stuff should mean only healthy cells are made from now on. The GM cancer-fighting cells were killed during the transplant and so don’t live on in Layla’s body. The treatment is so complex that it is likely to be used as a last resort, when conventional therapies have failed. However, five to ten children with acute lymphoblastic leukaemia, the form of the disease Layla had, could benefit a year in the UK. Dozens of children and adults with other leukaemias could also be helped. And tweaking the donor cells in a different way could allow other cancers, including lung tumour to be targeted. TALENs is one a wave of new ‘gene editing’ techniques that have caused huge excitement because they allow DNA to be accurately altered. This is the first time that cells edited using TALENs have been given to a person and only the second time in the world any gene-edited cells have been used. It is hoped the techniques can also be used to correct the genetic faults that cause diseases like cystic fibrosis and haemophilia. However, there is concern that the technology could be misused to create designer babies, made to order by hair or eye colour. Much more research is needed to prove that the leukaemia treatment is safe and effective. If it is, the cells given to Layla could be an ‘off-the-shelf’ treatment for adults and children. Initially, they will be reserved for those for whom nothing else works but biotech company Cellectis wants them to eventually available to almost any patient and inexpensive enough to be paid for by the NHS.