Zayed Centre at Great Ormond Street takes research from the lab to the bedside under one roof

The facility is geared towards treating rare conditions and has also been working this year on Covid-19 responses

Dr Maha Barakat, senior adviser to the Abu Dhabi Executive Office. Fatima Al Marzooqi / The National

A year after its launch, the Zayed Centre for Research at London’s Great Ormond Street Hospital has focused its work on how to transform specialised medical research into treatments for patients with a range of diseases and conditions.

Maha Barakat, senior adviser to the Abu Dhabi Executive Office, has revealed how the work of the centre was boosted by a £60 million ($79m) grant from Sheikha Fatima bint Mubarak, wife of Sheikh Zayed, that allowed it undertake an ambitious expansion. The funding enhanced its work in regenerative stem cell research, gene therapy and immunology technologies.

Targeting rare diseases for scientific breakthroughs lies at the heart of what the hub aims to do and those pioneering applications are more broadly for advances across medicine.

"The Zayed Centre is geared towards children with rare disease and they see patients with rare disease," Ms Barakat told The National.

“The research into a rare disease can be a catalyst into discovering new ways of diagnostics and treatments for much more prevalent adult diseases. I strongly support and advocate increased funding into the study of rare and undiagnosed diseases.

“If you take the smaller prevalence diseases and start targeting them, hopefully you can have a bigger impact.”

It is something she describes as a transformative approach to research.

“So often when scientists start researching something so rare that it's never been diagnosed before, you see an abnormality and you start going through the genetics and the changes of consequence of the genes, you can actually discover a pathway that can be apparent in a much more prevalent disease – for instance, cancer in adults or obesity or diabetes or heart disease, stroke. This is why research is always so important.”

The Zayed Centre possesses another unique facet, which Ms Barakat describes as a translational medicine model.

“You go from the lab to the bedside and it’s in the same building,” she explained. “It’s all geared towards researchers doing their work and then being able to implement their findings for patients for whom there is no other treatment or no other therapy options.”

For children born with an error in their genetic code, the benefit of ground-breaking gene therapy is that defect can be edited and be essentially cured. “That really is a lifesaver,” she said. “You target the genes and then the genes fix the body.”

Regenerative medicine is another frontier. “There are certain cells in our body that are not regenerative and if you have a problem in those cells because, for example, of a congenital disease, you will suffer,” she said. “The Zayed Centre is a regenerative medicine resource and within that area they look at regenerating cells – for instance, using stem cells. They look at engineering tissue. And then you actually regenerate organs. These are all critical elements of regenerative medicine.”

The Zayed Centre hosts the largest standalone academic manufacturing unit for gene and cell therapies in the UK and one of the largest in the world, and has been part of a new effort to tackle the Covid-19 outbreak in a UK government trial. Researchers are using small doses of the manufactured virus to find the smallest possible amount that can cause an infection of Covid-19. Volunteers will then be infected and closely monitored. The virus characterisation study could then be developed to prevent the infection.

The programme is a partnership between the UK government, Imperial College London, the Royal Free London NHS Foundation Trust and hVIVO, a company that develops human challenge models for viruses.

Great Ormond Street children's hospital. AFP

It is all very much in keeping with the overall philosophy behind the Zayed Centre, which is one of a range of health partnerships stretching from Washington DC to Morocco for the UAE.

“The centre opened a year ago and during 2020 it has faced the pandemic that the whole world has faced," Ms Barakat said. "Over and above all the activities it was founded on, the Zayed Centre has been able to pivot and innovate and help the UK government’s emergency response with Covid, and the labs are contributing to the initiative to fast-track a vaccine.”