Sharing the science of gene therapy
by Guest Author on 9 Jan 2019
This festive season, stem cell scientist Professor Bobby Gaspar, from the UCL Great Ormond Street Institute of Child Health, appeared as a special guest on the BBC Royal Institution Christmas Lectures. Here he shares the thrill of healing patients using gene therapy – and why it’s so important to communicate the science behind new medicines to the world.
To be a part of the Christmas Lectures alongside Rhys, the first patient to be successfully treated at Great Ormond Street Hospital with gene therapy back in 2001, was very special.
I first became fascinated by the body and how it works when I was a teenager. This led to me joining Great Ormond Street Hospital as a physician 25 years ago. I was immediately struck by the clear need to find a better treatment for children suffering from severe combined immunodeficiency diseases. These conditions leave children extremely vulnerable to infectious diseases from birth and can be fatal.
A futuristic concept
At the time, the idea of gene therapy sounded like science-fiction to me. But I was intrigued and motivated to investigate whether the use of a genetic therapy for these diseases could lead to an effective treatment.
We successfully treated our first patient in 2012 and we’ve treated another 29 patients since using their own blood stem cells. The cells are genetically corrected outside of the body by replacing the missing or faulty gene, then transplanted back into the body. Using the patient’s own stem cells from their bone marrow provides a perfect match and eliminates the need for a donor search, along with the associated risk of complications such as transplant rejection.
Getting to this point has been a massive team effort – it really does take a whole village. Expert input and fresh ideas from people specialising in a vast range of disciplines have been vital. It’s involved scientists doing bench-based lab work investigating the nature of cells and the genes within them, laboratory staff who engineer the patient cells, doctors and nurses who treat and look after our patients, as well as the patients themselves and their families.
Communicating our research
I think it’s important to devote time and effort to figure out how to best communicate research in an accessible way. Not everyone needs, or wants, to know the full intricacies of the research. But they might still be interested in the puzzle we’re trying to understand and why finding the answer could make a difference to people’s lives.
Gene therapy can be an emotive issue for many people. I’ve heard genes being referred to as the ‘stuff of our souls’. This means it’s an area of research which can so easily lead to controversy or contention if the aims and actions of scientists are misunderstood. This makes it all the more important to help people understand what we’re doing with the technology.
Medicine, doctors and scientists of the future
The way I describe gene therapy is as a potentially new form of medicine, made up of genetic material. We want to use it to eliminate a disease, not change a person or their soul. Added to this, the gene-corrected cells cannot be passed on to the next generation.
Engaging with young audiences like that of the 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.
Prof Adrian Thrasher has been Bobby’s co-investigator throughout his MRC-funded severe combined immunodeficiency diseases research.
Watch the lecture Who Am I? : What Makes Me, Me? on BBC iPlayer