Designer Cells Cure Cancer in One-year Old in World First

London’s Great Ormond Street Hospital has been the first to use designer cells to treat leukaemia in a baby girl that was thought to be incurable.

The treatment, which involves using tiny molecular “scissors” to edit genes and make immune cells especially capable of hunting out and destroying cancer, represents a promising new front in the fight against cancer.

Layla Richards, whose aggressive form of leukaemia was treated with genetically engineered immune cells.

The parents had been told there were no options left to save their baby as her leukemia was extremely aggressive and didn’t respond to chemotherapy or bone-marrow transplants.

Layla’s father Ashleigh Richards said she may have to take drugs for the rest of her life, but after spending months in isolation to protect against infection she is free of cancer.

So, doctors at Great Ormond Street got permission to use a cell treatment they were developing with a French biotech company called Cellectis.

“We thought that the little bit of liquid in the syringe was nothing”, Ashleigh Richards recalled in the press release.

“We didn’t want to accept palliative care and so we asked the doctors to try anything for our daughter, even if it hadn’t been tried before,”she said”.

The treatment works by adding new genes to healthy donated immune cells known as T-cells, which work against leukemia.

Professor Waseem Qasim, professor of Cell and Gene Therapy at UCL ICH and Consultant Immunologist at the hospital, explained: “We have only used this treatment on one very strong little girl, and we have to be cautious about claiming this will be a suitable treatment option for all children”.

“But this is a landmark in the use of new gene engineering technology and the effects for this child have been staggering”, he said.

Dr Andre Choulika, chairman of Cellectis, said Layla’s story could herald the start of a revolution in cancer treatment.

Dr Paul Veys, from Great Ormond Street, said the transformation was one of the most remarkable things he’d seen in 20 years: “We’re in a wonderful place compared to where we were five months ago, but that doesn’t mean cure”. She underwent several rounds of intense chemotherapy, a bone marrow transplant, and took part in an experimental trial all before her first birthday, but the cancer came back.

“Importantly, unlike recent controversial reports of gene editing in human embryos with another technology modifications made to the T cells should not be passed on to future generations”.

“Doctors don’t want to say that “there’s nothing we can do” and offer palliative care, but sometimes that’s the only option”, Layla’s mother, Lisa Foley, said in a hospital press release.

MERLIN CROSSLEY: Any cancer which is characterised by one particular surface molecule that you could generate these killer t-cells against would be a good candidate for treatment but not every cancer is of that type. Before they are infused, the cells are given an extra gene to make them target leukaemia cells. “Hopefully Layla will stay well and lots more children can be helped with this treatment”. And, once placed back in the body, they attack cancerous cells.

Two months on, Layla remained clear of Leukaemia.

Firstly, the cells can become “invisible” to a powerful leukaemia drug that would otherwise kill them during the course of treatment. This experiment was only ever done on lab mice so a meeting was called to get the treatment approved, Independent reports.

TALENs is one a wave of new “gene editing” techniques that have caused huge excitement because they allow DNA to be accurately altered.