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UK: 13-year-old receives 'world-first' leukemia treatment

Kieran Burke
December 11, 2022

The patient with "incurable" T-cell leukemia received the groundbreaking treatment and went into remission, the hospital treating her said.

Exterior of Great Ormond Street Hospital for Children
Great Ormond Street Hospital for Children said Alyssa had exhausted all other forms of treatment before enrolling on the clinical trial which saw her go into remission 28 days laterImage: John Stillwell/empics/picture alliance

Pioneering cancer treatment given to a 13-year-old British patient has seen her go into remission, London's Great Ormond Street Hospital for Children (GOSH) said on Sunday.

The hospital released a statement telling the story of a teenager, identified as Alyssa, and her battle with T-cell acute lymphoblastic leukemia — an aggressive form of blood cancer. 

Alyssa's battle against 'incurable' cancer

According to the hospital, Alyssa was diagnosed with the illness in 2021 and received all current conventional therapies, including chemotherapy and a bone marrow transplant.

None of the treatments were found to work and there were no further treatments available under conventional care.

Alyssa then became the first patient to be enrolled in a new clinical trial in May of this year to receive "genetically modified" immune cells from a healthy donor.

After 28 days, Alyssa was in remission and received a second bone marrow transplant to help restore her immune system, the hospital said.

Without this experimental treatment, Alyssa's only option was palliative care, according to the statement.

Robert Chiesa, a GOSH consultant, said that while her turnaround had been "quite remarkable," the results still needed to be monitored and confirmed in the coming months.

What does the treatment involve?

Medical teams at the hospital have been using a genome editing technique called base-editing — a method of chemically converting DNA code to change the T-cells, which are white blood cells that are a critical part of the immune system.

The edited T-cells are given to the patient, which then attack and destroy cancerous T-cells in the body without destroying one another.

"It's our most sophisticated cell engineering so far and paves the way for other new treatments and ultimately better futures for sick children," said Waseem Qasim, a professor of cell and gene therapy at GOSH.

The hospital emphasized that Alyssa was the first patient in the world to receive the base-edited cell therapy and was at home recovering from her treatment.

"Once I do it, people will know what they need to do, one way or another, so doing this will help people," Alyssa said.

Doctors are now looking to get another ten patients who have also exhausted other available treatment options, and are hoping that the treatment can be offered earlier to children and also possibly be seen as another option to treat other types of Leukaemia.

The hospital said that the trial would only accept patients eligible for National Health Service (NHS) care.

AFP material contributed to this report.

Edited by: Farah Bahgat