A new UAE-backed research centre will help doctors develop cures for children battling rare diseases "quicker than ever before", said a senior professor at one of the world's leading hospitals.
Bobby Gaspar, professor of paediatrics and immunology at Great Ormond Street Hospital in London, said the Zayed Centre for Research into Rare Diseases in Children will support hundreds of clinicians and researchers to make vital medical breakthroughs to improve the lives of countless children when it opens in July.
The facility, which is to be based in London, benefitted from a £60 million (Dh278m) donation from Sheikha Fatima bint Mubarak, the wife of the UAE's Founding Father Sheikh Zayed, and chairwoman of the General Women’s Union.
“The work that will take place in the Zayed Centre for Research will enable us to make breakthroughs and develop cures for rare diseases quicker than ever before, giving children everywhere the chance of a longer and fuller life,” said Prof Gaspar.
The centre, to be run in partnership with Great Ormond Street Hospital and the Great Ormond Street Hospital Children’s Charity, will drive forward research into rare disease such as childhood cancers, cystic fibrosis and muscular dystrophy.
The professor told an audience including Sheikh Mohamed bin Zayed, Crown Prince of Abu Dhabi and Deputy Supreme Commander of the Armed Forces and King Abdullah II of Jordan, that the human body itself holds the key to combating a whole host of diseases.
During the Mohamed bin Zayed Majlis lecture, entitled 'Changing the global face of children's medicine' held at Al Bateen Palace, Prof Gaspar spoke of the remarkable results achieved through gene therapy, in which functioning genes are placed into cells in the body to fight disease.
Prof Gaspar said children’s medicine is moving away from the traditional idea of medications, pills and a “one size fits all” approach to treatments.
Instead, rare diseases found in children can be treated by treating the gene that is causing the disease.
“Inside every cell we have a nucleus, and inside it there are 23 pairs of chromosomes, and inside it there are 25,000 genes that control everything that we are: from how tall we are to the colour of our hair," he said.
“Just one single gene mistake can cause a devastating disease. And if we can understand what that gene is we can understand [how to treat it].”
Professor Gaspar cited an inspiring examples of a gene therapy success story, while showing a picture of a young child from Poland.
“This is a little girl back in May 2013. A single gene in her bone marrow meant that she couldn’t produce any white cells, which are there to protect us from bacteria and viruses.
As the girl lacked any white cells, even a cold could be life threatening.
The only treatment that was available was to carry out a cell bone marrow transplant from another individual in order to rebuild her white cells.
“But that meant a 30 per cent chance she would die, and her father did not want his child to take that risk. So he asked if he could enrol her in the gene therapy that we wanted to do.”
After treating the gene in her own bone marrow cells, she grew into a healthy child and was cured of her illness.
“This is all we had to give her - she did not have any other treatment," he said.
He showed a recent picture of her, looking happy and healthy with her father.
“Her father often sends me pictures of her skiing and wearing ballet costumes to show how she is leading a normal life," he said.
“This [gene therapy] will change the word of medicine because it will allow us to treat those severe genetic disorders.
“That is why it is so important to have a centre that is uniquely dedicated to looking after these children with such a high burden disease and very few opportunities."