This week I attended the British Society of Gene and Cell Therapy annual conference in Cardiff. The aim of the conference was to showcase some of the amazing research going on in the field of gene and cell therapy (GCT) and to allow scientists to come together and explore the field further. In this post I will give a summary of some the extraordinary research going on in the UK and discuss some of the challenges the field is still facing. If these challenges can be overcome, GCT has the potential to change the face of medicine as we know it.
GCT is at the forefront of medical research, exploring ways to cure diseases by the therapeutic application of gene editing or genetically enhanced cells. Traditional, small molecule medicines aim to treat diseases by targeting either the cause or the general symptoms of an illness. This is often achieved with synthetic drugs in a ‘one drug fits all’ kind of manor. Although effective in many instances, this approach can fail to account for the rather crucial differences between patients such as age, sex, race or specific disease cause. GCTs offer a more personalised approach to medicine and can be engineered to target the specific cause of a patient’s disease. By tailoring the treatment to the individual disease, defect or patient, GCTs have the potential to be more effective, less toxic and even to provide a long-term treatment for patients who can’t be treated with traditional small molecule medicines.
One of the most exciting applications of GCT is the use of genetically engineered T cells to treat cancer. T cells are an important component of your immune system that naturally find and destroy any cancerous cells within your body. Many established cancers have developed ways to either hide from these T cells or to suppress their natural killing properties thereby allowing the cancer to evade your immune system. Typical cancer treatments include chemo- and radio-therapies which are highly toxic and destructive to cancerous cells. Unfortunately these therapies are also damaging to healthy cells making them very harsh on already extremely sick patients. Scientists are currently working on a new approach to treat cancer by training the body’s own immune system to find it and destroy it. A common method is to remove T cells from the body and engineer them to be to be more effective at recognising, attacking and destroying tumours. This can be done by giving them receptors that recognise the cancer, improving their longevity and making them more aggressive. The T cells can also be designed to be better at homing to tumours. Although it’s still early days, clinical trials have reported some T cell therapies leading to the complete remission of cancer in multiple patients.
GCTs have also been used to successfully treat patients with Haemophilia. Haemophilia is a blood disorder that prevents the normal clotting of blood. The two main types of Haemophilia, Haemophilia A and B, are caused by a mutation in the gene for Factor IIIV or Factor IX respectively. These genes encode important clotting factors that are required to stop bleeding and allow wounds to heal. Severe haemophiliacs have to inject themselves with the missing factor numerous times a week and yet still have a very high risk of fatal bleeds. A recent clinical trial involved the delivery of correct copies of the Factor IX gene to haemophilia B patients. The gene was delivered to the patient’s cells using a virus which was engineered to carry the Factor IX gene. Once inside the cell the gene was then used by the cell’s own machinery to generate the missing protein. After treatment with the virus, all of the patient’s Factor IX levels increased leading in most cases to a ‘long-lasting amelioration of bleeding episodes’. Following the success of this study, researchers are now looking at adapting this technique for the treatment of haemophilia A.
These are just two examples of the amazing work being undertaken in the field but GCTs are also being developed for wide variety of other diseases including cystic fibrosis, macular degeneration and Parkinson’s disease to name but a few. Recent years have also seen the first GCTs becoming licenced for use in the EU such as GSK’s Strimvelis which treats a particular form of severe combined immunodeficiency, ADA-SCID.
Despite all these major advancements, there is still a long way to go before GCTs can become routine treatments. One of the major issues highlighted at the conference was the inability of hospital pharmacies to safely deliver these therapies to their patients. The handling of GCTs requires specialist equipment and training that many hospital pharmacy departments are yet to acquire. Although at present there are very few GCTs on the market, as the number of GCT clinical trials increases, more work needs to be put into providing suitable facilities for their administration.
Another obstacle facing the field, is the regulation and licencing of GCTs. As these are relatively new medicines, there is little frame work to follow on what safety studies and standardised models need to be presented to the regulatory bodies to get these drugs licenced. In my opinion, this is where the big players in the field need to come together to ensure that the correct documentation and guidelines are in place to speed up licencing, drive down costs and get these drugs to the patients that need them the most.
The final challenge in my eyes, is to raise the profile and public opinion of GCT. Thanks to the speed and ease of social media, ‘news’ disseminates across the world rapidly regardless of its factual accuracy or integrity. People love controversy and this means that when treatments go wrong (whether these treatments are scientifically sound or not) the story spreads like wildfire leading to mistrust and scepticism of the field. A perfect example of this was the recent news story of patients going blind after taking part in a ‘cutting-edge stem cell trial’. This story first appeared on my Twitter feed back in March and within hours could be seen across numerous social media platforms inciting comments such as the one below:
‘The American consumer should be more skeptical and realize that “science” is often wrong and professionals are not always to be trusted.’
Upon further reading it emerged that was this not a legitimate trial and was in fact carried out by a cowboy ‘clinic’ looking to take advantage of patients who had run out of treatment options but unfortunately that’s not the message that was splayed across our screens.
Although there are many obstacles to overcome, the future of GCT is certainly a bright one with much promise for providing effective, long –term treatments for a whole plethora of different diseases. As the field matures and more therapies make it to market we certainly have the potential to change the face of medicine as we know it.
I would like to conclude by saying a big thank you to the British Society of Gene and Cell Therapy and all of the presenters at #BSGCT2017 for a truly inspiring conference and a great 3 days in Cardiff. Here’s to next year and all the amazing advancements still to come!