Abu Dhabi: There was more good news recently on the potential of gene therapy after doctors in the US cured 15 patients with a fatal genetic brain disease by using gene therapy. Despite the success, don’t expect to see gene therapy being used in UAE hospitals anytime soon, as doctors continue to assess its long-term viability and safety.
The process of gene therapy sounds very simple on paper — doctors insert normal or corrective genes into a person’s body to help treat a genetic disorder, the normal genes essentially replace the genes that are not functioning properly.
At present, there are several diseases that cannot be cured by medicine or surgeries, as the disorder lies in the person’s genetics, and it is here where gene therapy does its work with the corrective genes.
Getting all of this done however, is highly complicated, as doctors use viruses as the vector of getting the normal genes into a person which, can lead to possible harmful side effects.
“It is a very delicate process to perform gene therapy, you need to have a very good infrastructure in place, this includes having the specialists, the laboratories, and facilities,” said Dr Fady Georges Hachem, who specialises in obstetrics and gynaecology at Burjeel Hospital.
“The principle of gene therapy is that if you have any genetic defect, and the chronic disease is related to the mutation of the gene, then gene therapy can help by replacing the defective gene with the new one,” he added, explaining how gene therapy could cure multiple types of diseases that are linked with genetic mutations.
Dr Hachem acknowledged the risks associated with the procedure.
“When patients go on gene therapy trials the doctors tell them there can be some complications, nothing is ever 100 per cent guaranteed, because with this technique we are introducing a virus into the person.
“The virus is the best way to introduce the gene into the body because it has a high activity of replication of the gene that you are introducing, but the complication and possible side effect is that you don’t know if the virus will have a mutation and create a new disease, so we have to study this factor and see the results on the trial patients over time,” he added.
“The good news is that there have been successful trials, for example with some forms of neuromuscular diseases, so progress is being made and I do think gene therapy will eventually be introduced to the market one day,” he said.
Exactly how long could it be before it does get introduced?
Dr Hachem said he believed that it would be within the next 10 years.
“I think that maybe in 5 years, maximum 10, we will start to see gene therapy being used. A lot of it is also down to the advancement of technology, we are able to do things today as doctors that we couldn’t 10 or 15 years ago, I follow a lot of the medical publications and really every month there is always something new happening in a positive way,” he said.
Dr Hachem said that he was also confident in the UAE’s capacity to carry out gene therapy when it eventually gets introduced to the hospitals.
“I think we are best placed for it, gene therapy requires the technology, human resources, and money, and if you look at the medical sector in the UAE we have all of that.
“The UAE is very well placed to be an innovator in this field, and with strong collaborations and partnerships with other countries, the UAE can really become a leader in the Middle East when it comes to this gene therapy,” he added.
