Analysing Gene Therapies: A note on how to find the future

Gene therapies are undoubtedly the pharmaceutical hot topic of the moment. As the first genuinely revolutionary treatments such as Zolgensma and Libmeldy are slowly being integrated into regular care in several countries, the industry seems to have at last arrived at the often-discussed underlying step-change in the nature of medical treatment, moving to personalised modifications on the molecular level. Gene therapies are cutting-edge techniques that modify “a person’s gene to treat or cure disease” by replacing certain genes with their healthy copies, by inactivating them or by introducing “a new or modified gene into the body to help treat a disease” (FDA 2018). For decades dubbed as the treatments of the future, gene therapies are becoming, as some argue, the treatments of the present, transforming the ways in which diseases, especially rare diseases could be managed and even cured (Finkel 2019). Media reports have turned this new development into “miracle cures” (Fay Cortez 2019) promising to treat incurable and rare genetic disorders, giving hope to patients and their families. As more gene therapies are being approved by regulators – in the US for example, the Food and Drug Administration (2023) has so far approved about 30 such novel treatments – the stakes for such types of speculation are increasing, as is the price for each gene treatment (Zolgensma currently has a list price of 1.795 million pounds per dose).

In this context, future visions clearly operate as more than just potentially hyperbolic statements. Instead these (often officially generated) expectations are collectively produced and circulated between actors and within innovation and policy spaces, and they sustain specific agendas and shape policy making (Van Lente 2012). Rather than being mere descriptions of events, expectations have a mobilising and performative function that seek to bring about specific changes to the current situation by emphasising hopes and opportunities rather than challenges and obstacles. In the context of pharmaceutical development, these expectations can also motivate future investment and garner attention for research that is still underway – these types of expectations have in the past been highly pivotal in the development of the modern biotech sector (Cooper 2008). At first glance, the case of gene therapy seems to bring together both the best that research can deliver, and the worst in terms of excess expectation raised on the back of these new developments. We argue that the ways in which futures are at work here can bring about a better understanding of both the way in which pharmaceutical development operates in the post-genomic era, and also how different actors may be positioning themselves at the emergence of a new paradigm of treatment.

A collaborative research project explored these (and other) questions over the past year by beginning to map the promises made and expectations raised during this transition to the future of medical treatment. A team of researchers including Eva Hilberg, Aleksandra Stelmach, Tineke Kleinhout-Vliek, and Paul Martin at the University of Sheffield and the University of Utrecht worked together on developing an approach to analysing this emerging vision of the future of medical treatment, and also held an inaugural stakeholder workshop that engaged with the meaning of future narratives for the regulation of new pharmaceuticals. The event mapped different regulatory futures in practical detail, and also began to open the debate to explore a potential for different types of participation, such as NGOs and also government representatives. Overall, it became clear that how the future is envisaged depends largely on who imagines it, and what images and words are used to describe future developments.

These images and words will be studied more closely in the next stages of the project, thus continuing the study of a new field of medical intervention at the moment in which imagination becomes reality. There are of course still numerous challenges to the development of gene therapies, including extremely high prices of treatments that risk undermining the ‘gene therapy revolution’ (Editorial 2023) and making it unavailable to patients, especially those from disadvantaged sections of society and from the Global South. The counter-imaginaries already paint the visions of gene therapies as the preserve of the wealthy rather than a cure for the masses (Editorial 2021). The futures of gene therapies as envisioned by patients and their families could also differ significantly from those imagined by companies (see previous blog posts by Jin Ding  and Eva Hilberg and Tineke Kleinhout-Vliek). Other key issues remain, such as if gene therapies will work and for how long, and what type of legislation would be needed to make them accessible (Reardon 2023). But the emergence of gene therapy still marks a unique moment of (potential) paradigmatic revolution that could re-set our imagination of future medical treatment for decades to come.
 

References

Cooper M (2008) Life as surplus: biotechnology and capitalism in the neoliberal era (Seattle: University of Washington Press)

Editorial (2021) Gene therapies should be for all. Nature Medicine, 27 131, https://doi.org/10.1038/s41591-021-01481-9

Editorial (2023). The gene-therapy revolution risks stalling if we don’t talk about drug pricing. Nature 616, 629-630. Doi: https://doi.org/10.1038/d41586-023-01389-z

Fay Cortez M (2019) Welcome to the Age of One-Shot Miracle Cures That Can Cost Millions’, Bloomberg UK,  June 1 available at: https://www.bloomberg.com/news/features/2019-06-05/gene-therapy-appears-to-be-beating-once-incurable-diseases?in_source=embedded-checkout-banner

Finkel E (2019). The gene therapy revolution is here. Medicine is scrambling to keep pace. The Conversation  June 5. Available at:  https://theconversation.com/the-gene-therapy-revolution-is-here-medicine-is-scrambling-to-keep-pace-118329

Food and Drug Administration [FDA} (2018) What is Gene Therapy?. July 25. Available at: https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/what-gene-therapy

Food and Drug Administration [FDA] (2023) Approved Cellular and Gene Therapy Products, June 30. Available at: https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/approved-cellular-and-gene-therapy-products

Reardon S (2023). ‘It’s a vote for hope’: first gene therapy for muscular dystrophy nears approval, but will it work?, Nature 618, 451-453. doi: https://doi.org/10.1038/d41586-023-01799-z
Van Lente, H. (2012). Navigating foresight in a sea of expectations: lessons from the sociology of expectations. Technology analysis & strategic management, 24(8), 769-782.