Q and A with Professor Bobby Gaspar, Director Designate of the Zayed Centre for Research into Rare Disease in Children

Professor Bobby Gaspar outlines his aims and vision for the Zayed Centre for Research into Rare Disease in Children that has been made possible thanks to a transformative £60 million gift from Her Highness Sheikha Fatima bint Mubarak, the wife of the late Sheikh Zayed bin Sultan Al Nahyan.

What are the main aims of the Zayed Centre for Research?

We want to understand the genetic basis of rare diseases and develop new treatments using the research taking place in the centre. The researchers will be focusing on the areas of genomics, gene and cell therapy, and regenerative medicine. 

What is your vision for the future of the Zayed Centre for Research?

I want it to be a vibrant and exciting centre that has the most pioneering researchers working together. Part of our ethos will be to try and bring together researchers who complement each other’s work in order to drive forward new diagnoses and treatments. Therefore, the first thing we want to do is to create a very dynamic working environment with these individuals and the facilities that will support their work.

I envisage that the Zayed Centre for Research will also act as a national and international centre for certain rare conditions. It will mean that patients either come to Great Ormond Street Hospital (GOSH) to access therapies unavailable anywhere else in the world, or we will be able to generate new therapies that can be given to patients in different centres worldwide.

How does the Zayed Centre for Research complement the rare diseases strategies of GOSH, University College London (UCL), the UK and Europe?

The vision for the Zayed Centre for Research is very much in line with these rare diseases strategies, nationally and internationally. For example, one aim of the European rare disease strategy is to create 200 new medicines for rare diseases by 2020, which the Zayed Centre for Research will undoubtedly contribute towards.

Rare diseases represent a considerable health burden when grouped together. They were designated a national priority in November 2013 with the publication of ‘The UK Strategy for Rare Diseases’ by the Department of Health. Part of this strategy is about using research to try to develop new therapies for rare diseases. GOSH and the UCL Great Ormond Street Institute of Child Health are right at the heart of that because we will be one of the few centres that will be developing new therapies for these conditions.

What have been the main research highlights in the past year?

There has been so much activity. We are curing genetic diseases with stem cell gene therapy and, we have become world-leaders in that field, which has attracted commercial interest.

We are in the process of treating and curing acute leukaemia using novel techniques, which is extraordinary. We have clinical trials that are looking at treating solid tumours using gene therapy, which has never been done before – that is extremely exciting. We are also looking at developing gene therapy for metabolic diseases for which there are currently no effective treatments, and this is going to go into the clinic very shortly. In addition, we are excelling at engineering new tissue organs using stem cells.

The Zayed Centre for Research is due to open in November 2018.