This page provides an overview of the field of gene and cell therapy. You will find a directory of educational resources towards the end of the page.
Gene and cell-based therapies hold great potential in creating effective therapies for hard-to-treat conditions. This field has developed rapidly in recent decades. There are already approved therapies in Europe which can cure congenital fatal conditions. Education professionals may find themselves heading discussions on this topic more frequently, in the context of biology and healthcare, or in terms of the social and ethical implications.
Between popular depictions and misconceptions of gene and cell therapies and the rapid rate of advancements and change within the field, there is ample scope for them to be misunderstood.
Unfortunately, there are also individuals and clinics who will exploit this misunderstanding, and take advantage of patients and carers whose treatment options are limited.
There is a risk that these misunderstandings and misinformation may affect people’s healthcare choices, and cause them to make a decision which is not ultimately the correct one for them. It may also affect how the wider public – including funders and policy-makers – view the field, and how people who choose to receive gene and cell therapies are viewed.
If you are in a position to provide information about gene and cell therapies – either to a classroom, a lecture theatre, or an audience of readers, listeners, or viewers – you have the potential to shape how people think and feel about these therapies.
We have collated resources which will provide a thorough introduction to the field of gene and cell therapy. We hope these will be helpful to you in providing accurate and balanced information to the learners you work with.
Gene and cell biology: the basics
The gap between the technical meanings of terms related to gene and cell therapy and their common use can lead to confusion. You should also be mindful of the language you use.
Many terms in gene and cell therapy have specific technical meanings, distinct from their colloquial use. The increased coverage of the topic in the media may mean that your group have a partial but uncertain understanding of some terms. Check in to confirm that everyone in the group is using the same terms to mean the same thing.
You can find resources explaining fundamental biological concepts in gene and cell therapy in our 'Cell and Gene Fundamentals' section.
You can find resources explaining the techniques involved in research and therapy in our 'Methods and Tools' section.
Some media depictions present gene and cell therapies as a ‘magic bullet’ which will eventually be used to cure all diseases and injuries. Your learners may believe, consciously or unconsciously, that gene and cell therapies are a ‘better’ way to treat disease and injury.
It may be helpful to discuss why some conditions are better suited to gene and cell therapies than others, and why gene and cell therapies may not be the best solution even when they are scientifically plausible.
If your learners are confident in their understanding of the basics, they will better be able to understand and critically appraise other material you present.
How do gene and cell therapies reach the clinic?
In order to reach the clinic, new therapies need to:
- prove that they are effective and safe for use through clinical trials
- receive market authorisation at an EU level
- receive market authorisation at a regional level
All new therapies must go through the clinical trials process to confirm their safety and efficacy.
After a therapy has been shown to be clinically effective and safe, it must receive regulatory approval, also referred to as market approval, or simply authorisation or approval.
In the EU, the regulatory body responsible for granting market authorisation is the European Medicines Agency (EMA).
When a therapy has been approved, it may be some time before it is available through regional health services. This is determined at a regional and national level, rather than at an EU level.
If a therapy has been approved by the EMA, but is not available in all locations, patients in EU countries may be entitled to travel for care. This is facilitated through a European Reference Network (ERN). These are international networks which allow for the sharing of specialist healthcare knowledge. The decision travel for care or not can be advised by the ERN, but ultimately must be made by the patient in consultation with their trusted team.
Accessing unauthorised or unproven therapies
There are several different channels through which someone might receive a therapy which has not yet been approved for market use.
Media coverage about new therapies may use the terms ‘unproven’, ‘experimental’, ‘investigational’, and others to mean the same thing. This confusion can help unscrupulous clinics offer therapies which are not grounded in scientific evidence. We recommend reading more about unproven therapies, so that you can make your learners aware of the differences between these terms.
Ethical and social issues around gene and cell therapies
The subject of gene and cell therapy can be a contentious one. Some people object on religious, ideological or personal grounds to the techniques used in research, to gene editing at an embryonic level, or to the idea of the therapies themselves.
You can find resources discussing some of the ethical questions raised by gene and cell therapies in our 'Ethics and Society' section.
It is difficult to discuss the topic of gene and cell therapy without addressing some of the ethical questions, and the subject may be unexpectedly emotive. We encourage you to bear in mind that your framing may affect how your learners think of these therapies, and of the people receiving them. It may also be a personally affecting topic for some students.
Even when a therapy has received regulatory approval, and been made available, they may still be difficult to access for a variety of reasons. Many societies already experience inequality of healthcare, due to financial or social circumstances. A therapy which is provided through a national health service can still incur heavy costs related to travel, care, time taken off work, to name a few.
Healthcare campaigners have expressed concern that, while advanced therapies can dramatically improve the lives of those receiving them, they carry the risk of all new technologies: that they will amplify existing systemic inequalities. As these therapies are relatively new, very little research has been conducted on the issues of healthcare equity.
When raising these topics in class and allowing students to discuss them, you should remind your students that these techniques and therapies are intended to improve the lives of people with limited options, and encourage them to exercise empathy rather than treating the subjects as simply abstract ideas.
Educational materials
You can find educational resources developed by resreachers and education professionals in our Educational Resources Directory.
Our Educational Resources Directory includes activities, lesson plans,and online courses for learners of all ages.
If you are interested in finding about gene and cell therapies for a particular condition, we recommend that you start with our condition-specific factsheetsm which you will find under 'Conditions' in the navigation bar. We are constantly adding new topics to our factsheets.