Appearing alongside the first patient ever to successfully receive gene therapy at Great Ormond Street Hospital, Bobby will explain how gene therapy, a potentially new form of medicine, can transform a disease by using a patient’s own stem cells.
Bobby’s interview will feature in the last of the three part series ‘Who am I?’ which will be broadcast at 20:00 on BBC Four on 26, 27 and 28 December 2018. This year’s series is supported by UK Research and Innovation (UKRI).
Bobby, Professor of Paediatrics and Immunology at UCL Great Ormond Street Institute of Child Health said: “I grew up watching the Christmas Lectures so to be asked to participate in them was a huge honour. Engaging with young audiences like that of the Christmas Lectures is so important because they are our society of the future, they are where the next generation of scientists and physicians will come from. If just one young person watching the programme is inspired to pursue medical research or become a physician as a result, then I’ll be happy.”
Fiona Watt, Executive Chair of the MRC said: “I am proud that the MRC has been able to support Bobby and his team every step of the way in their journey to find a treatment for children with rare bone marrow diseases.
“Our support for their work into severe combined immunodeficiency disease (SCID) started with funding their very early discovery and proof of concept work in 2007, through the trial process using cellular and animal models to test safety and efficacy, and finally into a clinical model that has made a huge difference to those diagnosed with this disease. The team treated their first patient successfully in 2012 and 30 patients have been treated so far. In 2015, their work led to the launch of Orchard Therapeutics, a UCL spin-out company. Orchard Therapeutics aims to develop commercialised forms of this therapy which we hope will benefit people around the world. This team effort is a truly brilliant example of what the MRC was set up to do – to support fantastic research from the laboratory bench right through to the patient’s bedside.
“Strong public support for, and participation, in medical research is crucial for the MRC’s mission and I’m delighted that Bobby will be able to explain his incredible work to an audience of millions across the UK.”
Gail Cardew, Director of Science and Education at the Royal Institution said: “This year we wanted to pick a topic that felt accessible and relevant to everyone's lives, and at the same time is tricky and complicated. ‘Who am I?' is a question that might simply be answered by giving your name and date of birth, but scratch a little deeper and you unravel the full complexity of what it means to be human and to be different from your friends and family. We're hoping that the lectures will unravel many secrets at the genetic level, and also spark a conversation about what identity means in today's world.”
Severe combined immunodeficiency (SCID) is the name given to several rare inherited diseases where children are born with defective immune systems; a lack of working white blood cells caused by a single gene defect in the bone marrow. It is also known as the ‘Bubble baby' disease because affected children are extremely vulnerable to infectious diseases and have to live in sterile environments. In the most severe forms, children are unable to fight off even very mild infections and, without treatment, will usually die within the first year of life.
The innovative gene therapy for SCID that Bobby Gaspar and his co-investigator Professor Adrian Thrasher (Professor of Paediatric Immunology at UCL and Great Ormond Street Hospital) and their multidisciplinary team have developed involves using the patient’s own blood stem cells, through a process known as an autologous blood stem cell gene therapy. Using the patient’s own stem cells provides a perfect match and eliminates the need for a donor search, along with the associated risk of complications such as transplant rejection. The blood stem cells are taken from the patient through a bone marrow extract and then genetically corrected outside of the body by replacing the missing or faulty gene. These gene corrected cells are then transplanted back into the body, through an intravenous infusion. Sometimes the patient is given mild chemotherapy before the transplant; this is done to eliminate any existing blood stem cells that carry the genetic defect so that the new engineered cells can repopulate the bone marrow more effectively after the transplant.
Image: Professor Aoife McLysaght, gene therapy patient Rhys & Professor Bobby Gaspar. Credit: Paul Wilkinson Photography