Breakthrough as ‘designer cell’ therapy fights girl’s leukaemia

The head of research at the Irish Cancer Society has praised the designer of immune cell therapy which was used to reverse leukaemia in a one-year-old girl, and said it demonstrates how important such research is.

Breakthrough as ‘designer cell’ therapy fights girl’s leukaemia

“It really shows how important research is and the impact it can have and the hope that it can give people,” Dr Robert O’Connor said.

Dr O’Connor was speaking after doctors in London used a new gene-editing technique to manipulate cells and fight the cancer in one-year-old Layla Richards at Great Ormond Street Hospital (GOSH) in London.

The baby was diagnosed with Acute Lymphoblastic Leukaemia, the most common form of childhood leukaemia, when she was just 14 weeks old.

Despite being treated with chemotherapy and a bone marrow transplant, doctors told her parents to consider palliative care.

Her family was then offered an experimental treatment under development at GOSH, in which doctors modified white blood cells from a healthy donor so that they seek out and kill drug-resistant leukaemia.

Specialists at GOSH emphasised it could be more than a year before they know for definite whether the therapy has cured the disease, or delayed its progression.

Professor of cell and gene therapy at University College London and consultant immunologist at GOSH, Waseem Qasim said it was a “landmark” development and the effects for Layla have been staggering.

“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.”

Dr O’Connor described the case as a “very emotive example”. He said: “This is an important finding but it is a bit early to see just how important it will be.”

However, he said it was great news for the family, and for a new technology.

Speaking on Morning Ireland, Professor Owen Smith, consultant paediatric chemotologist at Our Lady’s Children’s Hospital, Crumlin, said he believes this is where the future lies.

“This technology will not be available to everybody overnight. It has to be scaled up and in time I do believe you will be getting this designer therapy off the shelf, as it were,” he said.

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