Bristol family hopes US drug could save Elaiya's life

Elaiya is currently being treated at the Sheffield Children's Hospital

The family of a toddler with an extremely rare form of leukaemia have said they want to take her to the US for potentially life-saving treatment.

Elaiya, from Bristol, was diagnosed with acute myeloid leukaemia (AML) with a GLIS2 genetic mutation, which puts her at high risk.

Despite finding a stem cell match this was "sadly unsuccessful", her parents said.

They now want to raise more than £600,000 to pay for new treatment.

"This is the last chance to save our daughter's life," Elaiya's dad Muzahir said.

They have raised more than £87,000 in just over seven days - including a £10,000 donation from the Azaylia Foundation - set up after the death of the daughter of former Coventry City footballer Ashley Cain.

About 100 children and adults under the age of 19 are diagnosed with AML in the UK every year, according to the charity Blood Cancer.

But Elaiya's rare gene means that the standard course of treatment - chemotherapy - would not cure her.

Elaiya will be two years old this month

Muzahir said: "If you have a specific mutation, it just makes things a hundred times more complicated.

"As far as we are aware, Elaiya is the only child in the UK to have this genetic rearrangement, or mutation."

The family had hoped to attempt a bone marrow transplant, but while a compatible donor was found, the transplant was not possible after chemotherapy failed to bring Elaiya into remission pre-transplant.

It was decided to take the riskier option of using a mismatched umbilical cord instead, and despite that transplant going well, it was also failed to achieve remission.

Her parents now hope to fly her to Houston, Texas, where she would be admitted at MD Anderson Cancer Centre for several months to receive stage one of a two-stage treatment plan.

A drug called STRO-002 has been developed which, her parents said, specifically targets Elaiya's gene mutation. If this is successful, it will bring Elaiya into remission.

Stage two of her treatment would explore CAR-T therapy or a bone marrow transplant.

Elaiya's family say they must act as quickly as possible

"She will hopefully be the first international patient to test this new drug over in the States, called STRO, so she could be making history, and we are just praying we can get her out there on time," Muzahir said.

He added that Elaiya's leukaemia was progressing tenfold every two to three weeks and they were "really pressed against time".

"So far this drug has only been trialled on 20 children in the world... of those 20 children about nine of them have successfully gone into remission so at the moment it's 50-50, but 50-50 is better than nothing," Muzahir said.

"Elaiya is a fighter, she has been fighting and is not going to stop fighting, so we owe it to her to do everything in our capability to get her out there."