Layla (1) becomes first person to be cured of cancer using 'designer' DNA
Published 06/11/2015 | 02:30
A one-year-old girl has become the first person in the world to be free of leukaemia through a ground-breaking genetic editing technique which creates designer immune cells to fight cancer.
Doctors had given up all hope of saving Layla Richards, now 17 months, after she failed to respond to chemotherapy and a bone marrow transplant.
But when desperate parents Lisa and Ashleigh begged Great Ormond Street Hospital to try anything to cure their daughter, specialists agreed to attempt something entirely unique.
A team of scientists and doctors took donor immune cells and used 'molecular scissors' to edit out genes to create specialised killer cells which could hunt out and eradicate her leukaemia.
The treatment, which had only been tried in mice, was so experimental that specialists had to apply for emergency permission from health regulators and the hospital's ethics committee.
It took just 10 minutes to administer and now, just a few months on, Layla is free of cancer and back at home. Doctors are hopeful that she is cured but want to wait a year or two to make sure the disease does not return before giving her the all clear.
Professor Paul Veys, Director of bone marrow transplant at Great Ormond Street, said: "As this was the first time that the treatment had been used, we didn't know if or when it work so we were over the moon when it did.
"Her leukaemia was so aggressive that such a response is almost a miracle. She's a very tough little girl, almost indestructible."
Layla was born June 2014 but at just 14-weeks-old a blood test showed she had Acute Lymphoblastic Leukaemia (ALL). Doctors described it as 'one of the most aggressive cases we have ever seen.'
The baby underwent several rounds of chemotherapy and a bone marrow transplant but the cancer kept returning and eventually consultants informed her parents it was 'hopeless' as there were no treatments left Specialists said the only option was palliative care until Layla inevitably passed away.
"We didn't want to accept palliative care and give up on our daughter," said mother Miss Foley, of Enfield, north London. "We asked the doctors to try anything."
Unknown to the family, the consultants had been working with University College London on a cutting-edge new genetic treatment. It works by tweaking the DNA of donor immune cells so they would only target leukaemia cells and would not be rejected by the body.
It was hoped that a transfusion of the cells would clear Layla's body of any remaining leukaemia so that she could have a bone marrow transplant to reboot her immune system.
Layla was given an infusion of 1ml of the genetically engineered immune cells in a procedure that lasted around 10 minutes before being placed in isolation to protect her from infections.
Within a fortnight Layla had developed a rash which showed that an immune response was happening and her body was starting to fight off the leukaemia.
Two months later, Layla was given a transplant to replace her bone marrow which had been affected by the treatment, and one month later she was able to go home.
"I consider ourselves lucky that we were in the right place at the right time," said Ms Foley. "Hopefully Layla will stay well and lots more children can be helped."
The team at Great Ormond Street and UCL said the donor immune cells could be edited to help fight many other types of cancer and inherited diseases. They now plan to start clinical trials to show that the effects can be replicated.
Waseem Qasim, professor of cell and gene therapy ad UCL and consultant immunologist at Great Ormond Street Hospital said: "This is a landmark in the use of new gene engineering technology and the effects for this child have been staggering.
"If replicated it could represent a huge step forward I treating leukaemia and other cancers.
"It means there a whole bunch of other conditions that we can now try and create gene editing approaches for, where we are fixing genes in situ."
Dr Matt Kaiser, Head of Research at cancer research charity Bloodwise said the was a desperate need for new therapies for patients who could not be cured of blood cancers.