Gene therapy a medical breakthrough in the making

While it can help treat and prevent genetic disorders, there is a long way to go to prove its effectiveness and safety

20:50 July 14, 2017

Abu Dhabi: Inherent genetic diseases could soon be cured and treated by gene therapy which, if successful, could prove to be among the greatest medical breakthroughs of our time, but only time will tell if it comes to that.

Gene therapy is the process whereby doctors use corrective or normal genes to treat a genetic disorder. Genetic disorders and diseases are usually caused by genes that are not functioning properly — a mutation — and as such, with gene therapy, doctors will insert the corrective gene into the person’s blood cell, which will then replace the gene that does not function properly.

The process itself is highly complicated, with doctors inserting the normal genes into the patient’s cells through a virus and, as a result of this method, there are potential harmful side effects.

Experimental tests that have applied gene therapy to patients suffering from incurable diseases have so far been positive, and more recently, an advisory panel from the Food and Drug Administration in the US approved the use of gene therapy for leukaemia treatment. Despite all of this, it will be a long time until this type of gene therapy is used in the UAE, with many years more worth of studies, tests, and monitoring having to be conducted before a full approval is given by medical authorities around the world.

“Even though it’s an experimental technique currently tested mainly to treat diseases that have no other cures, it has a great potential to be a promising treatment option in the future,” said Dr Krishnamurthy Subbian, laboratory director, Medeor 24x7 Hospital.

“Gene therapy is tested for treating inherited disorders, some types of cancers and certain infections but its application will increase in the future,” he added.

Dr Subbian did, however, cautioned that despite the justified enthusiasm for gene therapy, the road ahead was still long, with many hurdles to overcome.

“Significant challenges to implementing the technique remain. These include ensuring efficient delivery of therapeutic nucleic acids to target cells, limiting unintended effects, cost and complexity of treatment regimens,” he said.

Listing some of the side effects that could occur, Dr Subbian said that there were many.

“Your body’s immune system may see the newly introduced viruses as intruders and attack them. This may cause inflammation and, in severe cases, organ failure.

“Targeting the wrong cells. Because viruses can affect more than one type of cells, it’s possible that the altered viruses may infect additional cells — not just the targeted cells containing mutated genes. If this happens, healthy cells may be damaged, causing other illness or diseases, such as cancer,” he added.

Dr Norbert W. Dreier, consultant – oncology, Burjeel Hospital, shared the same opinion, and said that the use of gene therapy still had to be studied and tested before it could be introduced to the UAE.

“At present we still don’t know the full long-term side effects of gene therapy, if you are using vectors like viruses to change the genetic materials of a person, then we don’t know if this will lead to another effect — there are a lot of questions that still need be answered,” he said.

“We need to prove the positive effects [of gene therapy], compare it with and look at the side effects — before all of this is done, we cannot just start using it on the patients; there has to be the proven studies before we give this kind of treatment,” he added.

Dr Dreier did, however, say that if gene therapy proved its medical worthiness, it could become a big game changer.

“The potential is huge, I do think we will see a lot of new development in the near future,” he said.