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. Journalists and other media 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 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 individuals who choose to receive gene and cell therapies are viewed.
If you are providing information about gene and cell therapies to 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 accurate and balanced information to your audience.
Gene and cell therapy: the basics
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. It is important to be clear in how you are using a particular term; the disparity between the technical and colloquial use of a term may lead to misinterpretation.
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.
You can find an overview below of the reasons why some conditions are better suited to treatment by gene and cell therapies than others.
How do gene and cell therapies reach the clinic?
To be approved for clinicalal use, new therapies must go through the clinical trials process and regulatory approval process. After a therapy has been approved, national or regional healthcare providers must arrange a purschase/reimbursement scheme with the therapy manufacturers in order to provide it through their clinics.
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. You can read about the factors affecting the availability of approved therapies, including reimbursement of therapy manufacturers, below.
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.
These are therapies which have not gone through the clinical trials process. The manufacturers and practitioners have not had to prove to any regulatory body that the therapy is scientifically sound. These therapies can be offered by taking advantage of loopholes, providing misleading or partial information, or simply continuing to offer a therapy which does not meet regulatory standards. In some cases this may be technically legal, but they do not undergo the same safe-guarding as regulated therapies.
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 use these terms accurately, and make your audiences aware of the nuances within 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 the ethical and social issues aurrounding gene and cell therapies in our 'Ethics and Society' section.
A therapy which has received regulatory approval, and been made available at a regional level, may still prove difficult for some patients to access. Many societies already experience inequality of healthcare, due to financial or social circumstances. A therapy provided free-of-charge through a national health service can still incur heavy costs related to travel, care, and 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 completed on issues of healthcare equity in gene and cell therapy specifically.
It is difficult to discuss the topic of gene and cell therapy without addressing some of the ethical questions. We would encourage you to bear in mind that your framing may affect how your audience thinks of these therapies, and of the people receiving them.
Specific information and expertise
If you are interested in finding about the use or investigation of gene and cell therapies to treat a particular condition, we recommend that you start with our condition-specific factsheets. We are constantly adding new topics to our factsheets.
You can find information about ongoing clinical trials in Europe on the European Clinical Trials Register.
You may be able to find a national registry of clinical trials on either your national health provider’s website, or a government website.
You can find information and case studies about the development of gene and cell therapies in our 'Reseach Pathways' section.
We do not have the capacity to put you in direct contact with experts in the field for comment or interview. Our factsheets and Spotlight on Research pages may be a useful starting point for identifying scientific experts, and national support agencies may be able to recommend individuals with lived expertise.
Some media depictions present gene and cell therapies as a ‘magic bullet’ which will eventually be used to cure all diseases and injuries. Your audience may believe, consciously or unconsciously, that gene and cell therapies are a ‘better’ way to treat disease and injury. It is also important to keep in mind that all healthcare decisions are deeply personal, and must be considered on an individual basis. The risks or side effects deemed acceptable at a regulatory level may not be acceptable to each individual with the condition, depending on their priorities. Individuals who feel that their condition is well-managed with traditional therapies, and who are satisfied with their quality of life, may prefer to continue this approach than undergo an irreversible therapy.
We advise patients to consider the following questions when speaking to their healthcare provider, to ensure that they make a decision which aligns with their own healthcare priorities. They may offer a useful perspective on the individual decision-making process.