Somatic gene therapy research: Is it the future?

Updated: Jun 27

By Freya Malalagama





There are around 367,000 new cancer cases in the UK every year, that's around 1,000 every day, around 1 in every 2,500 babies are born with cystic fibrosis. In 2017, 93,385 people living with diagnosed HIV infection received HIV care in the UK. For people suffering with debilitating diseases, the disease which they suffer with can cause grief and declining mental health for them and their family members. Now imagine a world in which gene therapy research is successful; a world where disease does not dictate a person’s quality of life or hinder their opportunities.

‘Gene therapy’’ refers to the experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient's cells instead of using drugs or surgery. ‘Germline gene therapy’ is when DNA is transferred into the cells that produce reproductive cells, eggs or sperm, in the body, in comparison to ‘somatic gene therapy’, which involves the placing a human gene into a living person's somatic cells. The latter cells do not produce the eggs and sperm and somatic cell gene therapy would aim to cure a disease only in the patient, not in the patient's descendants.

It is crucial to recognise the difference between the two therapies, as I will be focused on the effects of increased funding towards somatic gene therapy, as opposed to germline.


Utilising NHS funding towards longer term solutions that will be able to aid long-term debilitating diseases such as cancer, cystic fibrosis and AIDS, and could prevent pressure on the NHS in urgent care. This is more effective than focusing the funding towards short-term solutions that simply deal with the problem at face value. Furthermore, by investing into gene therapy research, we may be able to not just ease diseases, but cure them at their most basic level.


Don’t just take my words for granted, see the effects of somatic cell therapy for yourself. Rhys Evans, a UK toddler who suffered from an inherited immune system disorder, underwent gene therapy and recovered. Two years before this, a team of French scientists reported the successful use of gene therapy to treat two boys affected by X-linked severe combined immune deficiency -- the same disorder that affected Rhys Evans. A new study published by the team from the Necker Hospital for Sick Children in Paris reports that two boys, as well as two other patients, are continuing to live normal, healthy lives. These children could be the first of many patients to be permanently cured by gene therapy.


Now, we have just talked about the most gripping, personal, life-changing aspects to gene therapy research. This is a success, not just felt by the immediate circle of people who actually suffer from this disease, but by potentially everyone in Britain, as we are all connected to one another via the NHS


Some may argue that these life-changing solutions may not be worth investing the cost and the time, when we could be targeting the NHS’ funding towards other purposes. However, the future is closer than you think.


For instance, take cancer. Perhaps one of the strongest driving forces behind the propulsion of gene therapy. The origin of gene therapy for cancer, which is Gendicine was manufactured in 2003 for head and neck cancer. When it was found that almost 50% of tumours have a mutated p53 gene, it triggered the idea that if an intact unmutated p53 gene could be delivered to the cancer cells, it could repair DNA damage. The outcome of its first successful clinical trials led to an astonishing 90-96% rate of total response, which far exceeded the success rate of standard cancer treatment. Following the success of this therapy, an astounding 30,000 people have benefited from this therapy. These trials have already begun to make an impact and this, not only, demonstrates a proper use of funding, but also show our proposal is effective and has the potential to save lives. For people like Ryhs Evans, this research makes all the difference.


To conclude, what are the advantages of increasing the NHS funding towards somatic gene therapy research? Firstly, those who suffer from life-changing diseases will finally be able to experience an increased quality of life and normality. Secondly, the NHS will be able to provide a better level of care towards patients, due to reduced pressure on the NHS. Last but not least, increasing funding, as proposed by our team, will be able to accelerate the rate of success of gene therapy so that the future success of gene therapy can be shared. However, balancing the unknown side-effects, moral dilemmas and economical crisis that the ongoing pandemic has placed upon us is a tough challenge, which could put a hindrance on gene therapy breakthroughs in the near future.


In the words of the Aneurin Bevan, ‘No society can call itself civilised if a sick person is denied medical aid because of lack of means’. These were the very words that built the foundation of the NHS we know today. Through somatic therapy, we have a gateway to offer a lifetime cure to those suffering with debilitating illnesses. However, the outcome of gene therapy research will only be apparent to us once we are able to navigate the full potentials of gene therapies, which could take years.


RESOURCES:


[1]: Biological significance of c-erbB family oncogenes in head and neck cancer. (n.d.). PubMed. Retrieved 22 April 2021, from https://pubmed.ncbi.nlm.nih.gov/15785872/


[2]: Entering the Modern Era of Gene Therapy. (n.d.). PubMed. Retrieved 22 April 2021, from https://pubmed.ncbi.nlm.nih.gov/30477394/