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Addressing the challenge of informing the wider population on advances in gene and cell therapy

Author: Gerry McLachlan, 3rd March 2021

The role for the BSGCT in engaging with the public is becoming ever more important as an increasing number of gene and cell therapies are either licensed for clinical use or are currently in, or are reaching the stage of recruiting patients for, clinical trials.

One of the aims on our Mission & Vision page of the website is the following;
•. Improving public awareness and providing an informed and responsible voice for science in this field.

Several recent developments bring the need for this into sharp focus. 1) The volume of misinformation around the development and use of the COVID vaccines 2) The recent reporting of Suspected Unexpected Serious Adverse Reactions (SUSARS) in gene therapy clinical trials.

The COVID vaccines produced by Pfizer/BioNTech, Oxford/AstraZeneca and Moderna are all vaccines that rely on the delivery of genetic material (DNA or RNA) that produce the SARS-COV-2 Spike protein in the cells that they are delivered to. The fact that these vaccines are delivering genetic material has led to social media networks being swamped by stories suggesting that the delivered material can somehow mix with the recipient’s DNA and irreversibly alter it. Unfortunately a large proportion of the population lack the biological expertise that would allow them to dismiss this type of profoundly inaccurate claims. The fact that the genetic material being given in the vaccine will not incorporate into the DNA of the cells and is only present for a short time to allow the cells to make the spike protein that then trains the immune response to respond to an actual infection with the virus, may not be getting across effectively enough to combat the deluge of misinformation.

In the case of the SUSARS in gene therapy clinical trials (1, 2, 3), thorough investigations are ongoing to determine whether the gene therapy vectors were responsible. One case from 2018 (1) involving a patient participating in a trial of gene therapy for Sickle Cell Disease concluded that the Myelodysplastic Syndrome developed by the patient could not be attributed to insertional oncogenesis by the lentiviral vector. One challenge lies in explaining the technicalities of such investigations in a transparent manner that allows patients to make informed decisions on the risk: benefit ratio of participating in trials of gene therapy or agreeing to receive licensed products.

This raises the prospect that similar campaigns of misinformation may be propagated in response to the proposed clinical use of future gene therapy applications, particularly those using integrating vector systems or genome editing strategies where host cell DNA is in fact being “altered”. The implications of such therapeutic strategies are open to misinterpretation.

In 2020, there was a call from the EU Commission H2020 that is aimed at helping to address this significant need.

SC1-HCO-19-2020: Reliable and accessible information on cell and gene-based therapies
Scope: Proposals should offer well-structured and detailed strategies to convey accurate and up-to-date information on cell and gene-based therapies using multiple contemporary modalities, including a website…..
…..For broader audiences proposals should create a reliable, transparent, accessible resource for patients to make informed decisions and for citizens to have access to scientifically viable information on cell and gene-based therapies, including sex and gender aspects when relevant.
…..Proposals should provide state-of-the-art strategies to engage the public and foresee regular evaluation of whether they reach the targeted audiences. In addition, a series of communication events should be organised, also open to the public, where innovative technologies could be presented and discussed

A successful proposal was put together to form the European Consortium for Communicating Gene and Cell Therapy Research (EuroGCT). EuroGCT brings together a new partnership, including several European learned societies for cell and gene therapy, national or regional Stem Cell Networks, the UK Cell and Gene Therapy Catapult, patient representative organisations and many other partners spanning 18 European countries. The new project builds on the experience of the highly successful EuroStemCell initiative https://www.eurostemcell.org/ that aims “to help European citizens make sense of stem cells”.

EuroGCT will develop a Network of Expertise (NoE) with working groups around specific conditions/disease groupings or legal and societal issues, who will advise on priority areas, ensure that all information produced represents the rigorous consensus view of specialists in the field, and support development of rapid responses to topical issues. A hub team staffed by professional digital communications experts and science communicators will work with the NoE members to develop specific information and engagement resources including website content tailored for researchers, patients and people affected by conditions and the wider public. The outputs will be published in seven major European Languages.

This initiative will provide opportunities for those working in the field to contribute to this resource which, amongst other objectives, aims to provide a point of contact for the media and a focal point for patient and public enquiries.

(1)   Hsieh et al, Blood Advances Vol 4 Number 912 2058-2063 May 2020
(2)   https://www.sciencemag.org/news/2020/12/liver-tumor-gene-therapy-recipient-raises-concerns-about-virus-widely-used-treatment
(3)   https://www.biospace.com/article/-bluebird-bio-shares-plunge-34-percent-after-halting-sickle-cell-trials/
(4)   The EuroGCT webpage will go live shortly at www.eurogct.org

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Addressing the challenge of informing the wider population on advances in gene and cell therapy

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