Zayed Centre for Research offers vital lifeline to children battling rare diseases

UAE-funded centre opened in London in 2019 to undertake life-saving research

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.

The Zayed Centre for Research, which opened in 2019, focuses on understanding and treating rare diseases in children – and in doing so may offer insights into adult illnesses too.

Linked to the world-renowned Great Ormond Street Hospital for Children (Gosh), it was named in honour of Sheikh Zayed after his wife Sheikha Fatima bint Mubarak donated £60m ($82.6m) to the hospital’s charity in 2014.

As Sheikh Mohamed bin Zayed, Crown Prince of Abu Dhabi and Deputy Supreme Commander of the Armed Forces, visits the centre, we look at its crucial role in combating disease.

Why is the centre needed?

Much work at the Zayed Centre for Research into Rare Disease in Children, to use the full name, relates to conditions with a genetic basis.

Speaking in 2019 to The Lancet Child and Adolescent Health, Prof David Goldblatt, Gosh director of clinical research and development, said most rare diseases with a genetic cause develop in childhood.

There is “a strong imperative to study and treat these diseases” because three in 10 children with a rare condition do not live beyond the age of 5.

While the focus is on rare illnesses, insights in molecular biology and genetics can have wider applications, including for common diseases affecting adults.

The centre is also important in clinical care, having 21 child-friendly consultation rooms and eight clinical investigation rooms.

What does the research cover?

One area is gene therapy, under which healthy forms of genes are inserted into cells to make up for the patient’s “faulty” copies.

It may also involve adding a new gene to produce a protein to combat disease, or disabling a faulty gene.

While still experimental, gene therapy sometimes offers the potential for a permanent cure.

Research also covers cell therapy, where healthy cells are introduced to compensate for diseased ones in the patient.

Another cutting-edge field is regenerative medicine, in which diseased cells, tissues or organs can be repaired, regrown or replaced.

Central to this are therapeutic stem cells, which are “master cells” that have not undergone specialisation into particular types.

The centre has played a part in the first human challenge trial for Covid-19, in which healthy people aged 18 to 30 are being intentionally infected with the coronavirus to help vaccine and treatment development.

What are the research facilities?

The centre, which was purpose built adjacent to Gosh and the University College London Great Ormond Street Institute of Child Health, has world-class facilities.

These include seven rooms where gene and cell therapies can be produced, described as the largest such unit in the UK.

There are 140 laboratory benches “for early development work to test the safety and efficacy of potential new treatments”, nine tissue-culture rooms where new treatments can be tested on lab-grown cells and a flow cytometry suite for the sorting and identification of cells.

At a cardiac research suite, 3-D models of the heart can be produced, which allows treatments and devices to be tailored to the patient.

There are offices for 400 researchers and clinicians, along with meeting areas where teams can collaborate.