GOSH’s groundbreaking gene therapy research to feature in Royal Institution Christmas Lectures

28 Dec 2018, 10:25 a.m.

Photographer: Paul Wilson photography

Professor Bobby Gaspar, who has pioneered gene therapy research at Great Ormond Street Hospital (GOSH) for the last 20 years, was a special guest at the Royal Institution Christmas lectures this year.

Bobby, who is Professor of Paediatrics and Immunology at UCL Great Ormond Street Institute of Child Health (ICH), appears on stage in the third lecture in the series explaining how gene therapy can be used to fix genetic errors for children with rare immune system disorders. Joining Bobby on stage was Rhys Evans, the first patient to be successfully treated with cutting edge gene therapy at GOSH almost 17 years ago.

The three part series addresses the question ‘Who am I?’ and will be broadcast at 8pm on BBC Four on 26, 27 and 28 December 2018. Episode three focusses on emerging genetic technologies including gene therapy and personalised medicine, both key areas of focus for research at GOSH.

“I grew up watching the Royal Institution Christmas Lectures so to be asked to participate in them was a huge honour,” said Bobby. “To be part of it alongside Rhys, the first patient to be successfully treated with gene therapy at GOSH back in 2001, was very special" he added.

Rhys’ story 

Aged just nine months, Rhys was admitted to GOSH where he was diagnosed with a serious immune system disorder called Severe Combined Immunodeficiency (SCID), more commonly known as ‘bubble baby’ syndrome. Rhys was lacking a copy of a gene called SCID-X1, which meant his immune system did not develop properly and he was prone to infections. Even a cold could be life threatening.

Back in 2001, the only treatment available was a bone marrow transplant, an incredibly difficult procedure. At that time Professor Bobby Gaspar and his colleague Adrian Thrasher were working on a revolutionary new type of therapy where a working copy of the gene could be inserted into the patients’ own cells using a modified, harmless virus.

Rhys became the first to receive this groundbreaking gene therapy and after a few weeks his immune system started to develop.

How gene therapy works

Groundbreaking research

Since Rhys was treated back in 2001, GOSH researchers have been continuing to break new ground and develop new types of gene therapy for children with serious immune system conditions.

Having first developed this treatment for children with SCID, gene therapy is now available at GOSH for several other immune system disorders – Wiskott–Aldrich syndrome, Chronic Granulomatous Disease and another rare form of SCID called ADA-SCID. Together these conditions affect thousands of children across the world; without treatment, life expectancy is just a few years.

Gene therapy at GOSH since 2001

Professor Gaspar, along with colleagues at GOSH and ICH continue to make use of the latest technological advances to further increase the effectiveness of gene therapy. Their work is supported by a range of funders, including the National Institute for Health Research GOSH Biomedical Research Centre, GOSH Charity, and a current trial, supported by the Medical Research Council is looking at a new more effective method of delivering a working copy of the missing gene into a patient’s cells.

Photography credit: Paul Wilson photography

Gene therapy offers hope to children with rare and fatal brain diseases

Scientists and doctors at UCL GOS ICH and GOSH have given hope of a gene therapy cure to children with a rare degenerative brain disorder called Dopamine Transporter Deficiency Syndrome (DTDS)

Origins of kidney cancers confirmed

The origins of seven types of kidney cancer, including several rare subtypes, have been identified.

Learning from patients and families during a pandemic

Researchers have worked with a patient support group to better understand the wide range of parenting experiences during the pandemic.