Professor Sir Munir Pirmohamed – NHS Chair of Pharmacogenetics at University of Liverpool
Doctor Pradeep Harish – Research Associate University of Liverpool
Blog reviewed by the ARDAT PPIE group
Since September 2023 we have been recruiting people who have had gene therapy to the ARDAT project, to help us evaluate immune responses (side-effects) that can be triggered by gene therapy. Immune responses to the therapies themselves can be problematic – they can make people unwell (often with ‘flu-like’ symptoms) and can stop gene therapies working. One of the primary aims of the ARDAT consortium is to figure out why such immune responses occur, and what we can do to prevent such immune responses from occurring. We are proud to report that we have collected a series of blood samples from 9 people over time which have been used for identifying proteins and other ‘markers’ of immune responses. We have also recently recruited 4 more patients for a second immune study (the T-cell study), marking a significant milestone in expanding the research. All infusion centres across NHS England – clinics that can deliver gene therapies to patients – are supporting this study. The study is truly a collaborative approach which should lead to impactful results.
Examining patient samples is a critical part of this research, as it provides invaluable insights into how we can best translate the scientific progress into new therapies. By studying samples and data from patients who have had gene therapy, we can ensure that our approach takes into account the complexities of human biology as closely as possible. This not only strengthens our preclinical research (work that takes place before reaching clinical trials) but may also lead to the development of new blood tests that can predict and prevent these immune reactions before they occur. This information will ensure the patient has more benefit from the gene therapy, and with less side-effects. The recruitment of people who have had gene therapy into our studies is therefore not just a milestone to be celebrated but a vital step in ensuring effective therapies for the future. We would like to thank all the patients and their parents and families for supporting this research – we know how much time and effort this takes, but without their involvement, none of this would be possible.
