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Breakthroughs in gene therapy offer hope for young patients with rare diseases

Khalifa at GOSH
Khalifa at GOSH

Meet Khalifa

Four-year-old Khalifa Al Qemzi from the UAE became one of the first children in the region to be treated using gene therapy. Khalifa was admitted to hospital in Abu Dhabi aged six months where he was diagnosed with severe combined immunodeficiency (SCID), and then admitted to GOSH where he was treated by Professor Gaspar and his team using gene therapy.

“Three months after birth, the symptoms of Khalifa‘s genetic immune deficiency started to appear. We then prepared to send Khalifa to the United Kingdom after the appropriate hospital for treatment was identified,” Saeed, Khalifa’s father.

Once at the hospital, Professor Gaspar and his team recommended the use of gene therapy as Khalifa’s family were not bone marrow compatible. Over the course of a week, bone marrow cells were taken out of Khalifa, the faulty gene was corrected, and then given back. Seven months on, Khalifa’s immune cells came back and his immune system is now functioning properly. 

“When we arrived at GOSH, Professor Bobby Gaspar examined Khalifa and suggested gene therapy as the best treatment option for him. We approved the recommendation and Khalifa received the best care from consultants and nurses at GOSH. The hospital provided Khalifa with everything he needed in order to reach his current medical condition,” added Saeed. “We thank God and all the staff involved for the treatment they provided Khalifa and the breath-taking results. Khalifa now visits GOSH for regular check-ups, and is living a normal life like other children.”

Commenting on Khalifa’s treatment, Professor Gaspar said “I have led a number of successful trials just like Khalifa’s that have seen gene therapy transform the outcomes of children with SCID. My hope is that continued efforts in gene therapy research will allow us to go even further and develop gene therapy as well as cell and stem cell therapies as a standard treatment for many more rare conditions.”

“We aim to pave the way for a new era of treatment for children and young people around the world,” he added. “I believe gene therapy will be able to improve the life and health of many children with life-threatening diseases, where other treatment methods are either ineffective or non-existent.”

All about GOSH

GOSH and its research partner, the UCL Institute of Child Health, are an internationally recognised centre for gene therapy research and genetic medicine. In 2001, paediatric immunologists Professor Bobby Gaspar and Professor Adrian Thrasher carried out the first successful gene therapy treatment to cure children with X-linked severe combined immunodeficiency (X-SCID). They have since developed this expertise to build a large, world-renowned programme of clinical trials for primary immunodeficiencies, successfully treating many patients.

GOSH and the ICH have expanded their genetics expertise across the hospital to include other first-in-child gene and cell therapies, including metabolic diseases, inherited skin conditions, HIV and T cell therapies for cancer. In 2015, GOSH became the first hospital to treat a patient with ‘incurable’ leukaemia using an experimental form of gene therapy from genetically engineered donor immune cells. Within a month, these cells – engineered by Professor Waaseem Qasim – had killed off all the cancerous cells in the child’s bone marrow.

UCL and GOSH are established globally as one of the top centres in the world for paediatric gene therapy. GOSH also plays a lead role in co-ordinating the North Thames network of hospitals involved in Genomic England’s 100,000 Genomes Project, helping to better understand and ultimately treat rare diseases and common cancers.

Khalifa and father in hospital
Khalifa, father and Professor Gaspar
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