By Robyn Bell and Aarash Saleh
Early morning alarms were set, science tattoos and BSGCT paraphernalia packed, it was time for BSGCT Public Engagement Day 2019. Travelling from all over the UK, BSGCT board members, volunteers, and local secondary schools piled into planes, trains and coaches bound for Oxford. Returning to last year’s fantastic venue, Oxford University Museum of Natural History, the day began with registration and catch-ups between familiar faces as the lecture theatre filled with a buzz of over 300 student and members of the public keen to get to grips with this ground-breaking science.
To kick off, Simon Waddington from UCL introduced classical gene therapy techniques; harnessing viruses to deliver healthy copies of genes for treatment of genetic conditions. Simon’s run-through of ‘Gene Therapy in Hollywood’ highlighted the rather interesting portrayal of gene therapy in the media, including the film ‘I am Legend’ where a genetically engineered cancer cure transforms humans into blood-thirsty mutants. (Who knows, maybe gene therapy scientists just hate Will Smith!). Jo Mountford from the Scottish National Blood Transfusion Service gave a masterclass in stem cell biology, introducing important advances in the field such as ‘induced pluripotency’ whereby adult cells can be reprogrammed to become other cell types, with exciting applications for tissue regeneration.
After a short break, Emma Morris from UCL brought great enthusiasm to her description of CRISPR Cas9. This relatively cheap and simple technology allows scientists to target and change DNA, rapidly changing medicine (despite frequently being depicted as a “weird blobby rubber duck”).
Armed with the basics, it was time to ask the audience their opinion on key questions in the field of gene and cell therapy. Students participated in a live poll on some widely debated issues: Was the human gene editing experiment in China ethical? Is it right to invest in technologies that may be prohibitively expensive and if so how can we address these issues? The poll was revisited later in the afternoon to gauge changes in opinion throughout the day. The results make interesting reading!
Lunchtime was filled with engaging gene and cell therapy activities with several new ideas launched this year. An excited crowd gathered around the #TattooMyScience stall where attendees emerged ‘inked’ with images of pluripotent stem cells, CRISPR gene editing machinery and assortment of gene therapy viruses among other designs.
The ever-popular ‘build a virus’ stand saw students creating novel vector constructs which Prof Alan Parker was later seen quietly slipping into his bag to inspire his next grant application. Attendees took every opportunity to speak with our junior scientists who came from as far and wide as Glasgow, Cardiff and Manchester to get an idea of science careers as an academic or in industry. Interesting conversations discussing the latest scientific developments and ethical questions raised in the talks and live polling sessions could be overheard.
After lunch we moved onto some real-world examples. Ralph Hector from the university of Edinburgh took us through some success stories in diseases like Spinraza gene therapy for the devastating disease Spinal Muscular Atrophy which is hugely extending survival and muscle function for the young children affected. Qasim Majid, on behalf of Professor Sian Harding at Imperial College, gave an excellent talk outlining clinical progress of stem cell therapies in heart failure where these cells are being used to regenerate damaged cardiac muscle. In a much-awaited talk, David Cole from Immunocore transformed audience members into T-cells, tasked with identifying ever more subtle abnormalities in his ‘spot the difference’ presentation. How can we modify or ‘train’ T-cells to be more effective at spotting these well disguised cell abnormalities which may indicate cancer? Amid all the excitement was a palpable sense of disappointment that David did not manage to knock anyone out this year by hurling his laser pointers into the crowd.
Professor John Harris from Manchester University gave us a lot to think about with his bioethics talk. He addressed societal anxieties around gene editing of embryos – a situation where consent obviously cannot be given by the (unborn) patient – “I don’t remember being asked for consent when my parents created me!” – he mused. Finally, we had a very moving perspective from Carol-Anne Partridge of the charity Cure CDKL5 UK, She described her family’s journey with the rare neurological disease, CDKL5 disorder, which affects her daughter and causes severe seizures and neurological impairment. The audience were clearly affected by Carol-Anne’s emotional account of how hard it often is to reach a diagnosis in rare diseases and hopes for a cure which may one day come through gene therapy.
As well as our wonderful speakers and volunteers, we were grateful to our sponsors Psioxus, Oxford Biomedica, Theolytics and Immunocore and benefited from a sponsorship grant from the Biochemical Society which enabled us to extend the reach of this fantastic public engagement event. Until next year!