Zayed Centre for Research powers ahead with seven experts at the helm

Most complex surgery involves operations on a single patient but there are procedures that involve two.

That is work in vitro such as that carried out by Prof Paolo De Coppi, who works at London's Zayed Centre for Research into Rare Disease in Children, the dedicated facility that pursues genetic and regenerative breakthroughs for conditions affecting children.

Precision implants

Prof De Coppi works to develop mini-organs inside the mother's body to implant in a child who might be suffering from medical conditions. Personalised organ grafts could prevent life‑threatening complications before or soon after birth.

Developing mini-organs for implantation is work that draws on “the whole village of ZCR” to complete the research.

“We have in ZCR the expertise that goes from the stem-cell biologist to the ability to sequence genes,” he told The National. “The lab is really a mixture between surgeons that come with a lot of ideas and then biologists that know how to culture cells, and the bioengineers.

“Engineers help us create modelling and create the special flasks to grow the cells in a special way. One example that was particularly innovative is the oesophagus. We can do something very innovative, which is called spatial sequencing or spatial transcriptomics, which look at the expression of every single cell in 3-D.

“The technology is now such that you look very deep at the gene expression in the cell and put this in the context of the tissue. Cells do certain things in context and understanding that context gives us functionality and a degree of safety.”

Prof De Coppi is one of a cluster of seven leading researchers at ZCR engaged in work that delivers breakthroughs in gene therapy, regenerative medicine, rare disease diagnosis and innovative treatments.

Show caption: Prof Paolo De Coppi at work. Photo: GOSH

ZCR opened to patients in October 2019 to bring together pioneering research and clinical care under one roof.

Partnered with the world-renowned children's hospital Great Ormond Street, ZCR is dedicated to manufacturing gene and cell therapies to provide new treatments or cures for seriously ill children from across the UK and international patients.

Opened in late October 2019, ZCR sits next to UCL Great Ormond Street Institute of Child Health.

The purpose-built medical complexes enable hundreds of researchers and clinicians to work side by side. Prof De Coppi relates how the entrance way to treatment gives families a glimpse of the scientists at work. “We invite the family and the patient themselves … to join us with the researchers so they see what's going on and they can raise their concerns at any stage,” he said.

The professor was named an honorary fellow by the prestigious American College of Surgeons earlier this year. “It is really the recognition of the whole group and I feel very humble and privileged.”

Show caption: ZCR opened in late October 2019. Victoria Pertusa / The Nati…

AI‑driven analysis

With AI driving the analysis of the latest developments in the laboratory, insiders say there is an acceleration of progress in making new diagnoses, treatments and cures for rare and complex diseases.

• ZCR's Prof Manju Kurian, a consultant paediatric neurologist, researche identifying the genetic causes of childhood neurological disorders, including movement disorders and neurodegeneration. Prof Kurian's team investigates genetic changes on the developing brain, using advanced models to develop precision therapies, including novel gene therapy approaches.
• Dr Amy McTague, a consultant paediatric neurologist, focuses on genetic causes of early onset epilepsy. She using genomics to improve diagnosis, treatment and long-term outcomes for children.
• Prof Francesco Muntoni, a neuromuscular disease specialist, shapes international understanding of conditions including Duchenne muscular dystrophy and spinal muscular atrophy. His work has bridged the gap between genetic discovery and treatment, helping to lay the foundations for therapies that are transforming outcomes for children with previously untreatable conditions.
• Prof Waseem Qasim, a cell and gene therapy research leader, focuses on harnessing and engineering immune cells to treat children with cancer and severe immune disorders. Prof Qasim led pioneering work in genome-edited CAR-T cell therapies, including world-first treatments for children with otherwise untreatable leukaemia.
• Sara Ghorashian, consultant paediatric haematologist, conducts research on CAR-T cell therapy and cellular immunotherapy, with a focus on improving outcomes for children with high-risk or relapsed leukaemia. This has led to new trials for children with aggressive blood cancer.
• Prof Claire Booth, a paediatric immunologist leads multiple gene therapy clinical trials. Developing treatments for children with rare immune deficiencies and other inherited disorders. Her research covers everything from laboratory development to first-in-human clinical studies.

Lifelines for children

In the seven years since opening, more than 13,300 children have attended appointments in its Falcon Outpatients suite, where they received expert care for rare and complex conditions.

It was made possible in 2014, thanks to a transformative £60 million ($80.7 million) gift from Sheikha Fatima bint Mubarak, Mother of the Nation.

Show caption: Sheikh Theyab bin Mohamed inaugurates the Zayed Centre for R…

Major contributions were also made by Research England, The Wolfson Foundation, John Connolly & Odile Griffith and the Mead Family Foundation. The centre is a partnership between Great Ormond Street Hospital, UCL and Great Ormond Street Hospital Children’s Charity.