UAE-backed breakthrough offers hope for leukaemia and epilepsy sufferers

London's Zayed Centre for Research into Rare Disease in Children shares latest gene-editing advances

The Zayed Centre for Research is a partnership between Great Ormond Street Hospital, University College London and Great Ormond Street Hospital Children’s Charity. Photo: Executive Affairs Authority

Immune cells can now be engineered to carry out increasingly targeted attacks on leukaemia, scientists have revealed.

Researchers based at the UAE-backed Zayed Centre for Research into Rare Disease in Children in London have shared how advances in gene-editing are offering hope to leukaemia patients. Pioneering work at the facility also includes advances in early genetic analysis that is making a "big difference" in outcomes for epilepsy sufferers.

Waseem Qasim, professor of cell and gene therapy at the Institute of Child Health and Great Ormond Street Hospital, said: "We can now engineer immune cells to attack leukaemia more specifically.

"We can collect cells from the patient or a healthy donor, take them to a laboratory, pick out the immune cells and as various bits of technology have merged — or converged — it is now possible to reprogramme those cells and introduce new genes into them to give them new properties.

“We are trying to rearm these cells and give them receptors on their surface, which can pick out — very specifically — target cells that they recognise as being dangerous or abnormal.”

Leukaemia is not the only rare pathology where a focus on genetics is paying dividends. Research indicates those with early onset epilepsy will have their outcomes improved by shifting away from the treatment of the disease and its symptoms towards a focus on its underlying genetic abnormalities.

Dr Amy McTague, clinical consultant academic and principal research fellow at UCL GOS Institute of Child Health, said her team is focused on rapid testing for babies and their parents, which has had “real impacts for families”.

“Doing [the research project] internationally has been very exciting — and quite challenging as well," she said.

"We’re hoping to show that, even across different healthcare systems, having the diagnosis earlier in the patient journey can make a big difference to the patient and parents’ experience as well as the management of the epilepsy."

Sheikh Mohamed at the Zayed Centre in 2021 with the former UK health minister Sajid Javid, right, and former UK education minister Nadhim Zahawi, left. All photos: Ministry of Presidential Affairs

Sheikh Mohamed and Sheikh Hamdan bin Mohamed bin Zayed during the visit to the Zayed Centre for Research into Rare Disease in Children.

Sheikh Mohamed speaks with Mohamed Al Ameri, researcher and PhD candidate.

Sheikh Mohamed visits the Zayed Centre for Research into Rare Disease in Children, located in London.

Sheikh Mohamed speaks with a family member of a patient at the centre.

Sheikh Mohamed and Sheikh Hamdan hold a meeting with Mr Javid, the former UK health secretary, at the centre.

Sheikh Hamdan attends a meeting with the former UK health secretary at the centre.

Sheikh Mohamed toured the centre, meeting researchers, professors and doctors working on pioneering research and life-saving treatments.

Sheikh Mohamed visiting the centre in 2021, which will celebrate its fourth anniversary in October.

Sheikh Mohamed was briefed on the centre’s specialist facilities that have enabled it to play a vital role in the global response to Covid-19.

Sheikh Mohamed tours the centre.

Sheikh Mohamed speaks with family members of a patient at the centre.

Dr McTague shares the excitement around tailored treatments that address genetic abnormalities rather than treating the seizures or movement disorder.

Early diagnosis means patients can be moved into the appropriate pathway for a tailored treatment more quickly, which can maximise the outcomes and life chances of children.