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Gene therapy – The future of our health

June 2019

Gene therapy
Vigorous research into gene therapy is now underway across the world

Gene therapy is hugely exciting. Whether it will fulfil its promise and in future years produce terrific treatments for many health problems we don’t know but at the moment, although still in its early stages, the results are very encouraging.

At the moment gene therapy is being used only in what are normally referred to as experimental techniques to treat medical problems where there is no other cure.

There are various approaches to gene therapy and these can include replacing a mutated gene that causes disease with a healthy copy of the gene; introducing a new gene into the body to specifically fight the disease; and inactivating a mutated gene that is not functioning properly and causing the problems.

Research is ongoing with vigour...there have recently been over 80 clinical trials taking place in the UK related to gene therapy. Commercial sponsors including companies from overseas are now involved in helping to finance the trials, and if you want to know more about the trials that have been taking place, a good source is ct.catapult.org.uk/cell-and-gene-therapy.

But while it is all very new, certain gene therapy has already received the okay to be used here in the UK, albeit still under the umbrella of “experimental techniques”. 

The EU regulators have recently given approval for Luxturna. This is a gene therapy to treat a rare form of retinal blindness. The treatment involves injecting a corrective copy of a specific gene directly into the retinal cells of the affected eye. Another approved treatment, this time authorised by NHS England, involves harvesting the immune T cells from the patient, modifying them to attack cancer cells, and then reintroducing them back into the patient. It has been approved for use to treat a common form of childhood cancer.

This really is the tip of a very big iceberg. Many trials are going on across the world with varying successes; the downside is that, and even with early success, there is going to be a very long lead in time before the treatments can be introduced into the UK for general use.

For instance, a small group of patients with advance Parkinson’s disease have been treated with special genes being inserted into a small area of the brain, enabling the brain cells to start making dopamine again. This has worked well and gave all of the patients in the trial improved muscle and movement control.  However, it will be a long time before sufficient clinical trialling has been undertaken and this treatment can be approved for wide use.

Controlling the spread of malaria is another genetic engineering project which has had some good success. Researchers at London’s Imperial College used a gene editing tool to block reproduction in the malarial carrying mosquitoes and this has the potential to greatly reduce and even eradicate these malarial carrying insects. However, it will be many years before all the early tests have been completed and genetically altered female mosquitoes can be let into the wild.

Another aspect is to transfer the natural immunity that some people have to certain diseases into another person through gene technology. This has already been done when stem cells from a donor with natural immunity to HIV was given to an HIV patient. This stopped the virus from attaching to the patient’s immune system and the improvements were profound.

It is all a very new approach to medicine and our overall health. The frustrating aspect is that while things are progressing fast, they may not be quite fast enough for those currently suffering from a severe medical problem.  But as more and more money is being invested in this sort of medical research, who knows what the future might bring.

There is a lot of information on the internet about gene therapy including:

learn.genetics.utah.edu/genetherapy
esgct.eu/gene-and-cell-therapy-glossary

 

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