Better Tech, More Collaboration Helps Drop Barriers for Gene Therapy

January 24, 2017 | ProgressTH 

Imagine being able to replace any section of your DNA precisely to cure genetic conditions, regenerate damaged or aging body parts, or train your immune system to recognize and eliminate otherwise incurable diseases. A technique called gene therapy is making this possible. 

However, gene therapy is still a cutting edge technique. With a few exceptions aside, gene therapy is still being explored mainly in animal models and clinical trials. But to even attempt it several years ago, researchers would have to be lucky enough to work at one of a handful of premier medical facilities around the world. 

Today, this is different. Research institutes all around the world are now increasingly gaining access to the technology and techniques required to study and apply gene therapy on their own, including here in Thailand.

We visited the Institute of Personalized Genomics and Gene Therapy at the Faculty of Medicine Siriraj Hospital, Mahidol University and spoke with Associate Professor Dr. Manop Pithukpakorn to learn about the current state of gene therapy in general, and how recent trends in technology and cooperation between researchers have helped remove barriers to the progress of this powerful technique.

We also learned about what precision medicine may help achieve before the promise of gene therapy is fully realized.

We Still Have Much to Learn 

Dr. Manop stressed that despite many promising results, there is still a lot of work to do. While some conditions are considerably easier to treat with gene therapy, others are at the moment quite dangerous to even attempt. Clinical trials throughout the years have failed, sometimes even claiming lives because of powerful and unpredictable immune reactions or the development of cancer years later after seemingly positive results.

Despite this, Dr. Manop revealed that dropping costs for technology and younger doctors more keen on collaboration have helped to make gene therapy research significantly more accessible to a greater number of researchers around the world. 

He is also hopeful that genetic research in general will add to the emerging field of precision medicine, creating for cancer patients the same hope now enjoyed by many HIV patients. 

While HIV still cannot be cured, with constant treatment, they can expect to live relatively normal lives and reach a normal life expectancy.  In many cases, an HIV patient's prognosis is more positive than that many cancer patients'. But with improved medication targeting cancer on a genetic level, before gene therapy is perfected, cancer patients may well live relatively normal lives in much the same way. 

What's Left to Do 

Dr. Manop agreed that public awareness regarding precision medicine and gene therapy is still very low. The difficultly stems from medical staff lacking both the time and the expertise to effectively communicate their work's significance to the public, and traditional media neither understanding their work, nor having any interest in it to begin with.

Raising awareness for the potential of this technology as well as expanding the number of young people entering professions essential to expanding research will help significantly in making future breakthroughs at a faster pace. 

Dr. Manop also said that improving genetic sequencing and analysis techniques as well as more efficient ways of delivering corrective genes to human cells will help make gene therapy more reliable, more effective, and safer.

Researchers at Mahidol University have already used gene therapy to treat and effectively cure 4-5 patients suffering from Thalassemia, an inherited blood disorder that can result in the abnormal formation of hemoglobin. 

Dr. Manop stressed that the long-term effects of this otherwise amazing result remain to be seen and that doctors prefer to use traditional treatments and would only recommend gene therapy at the moment if all other alternatives have been exhausted.

Still, the fact that gene therapy has reached Thailand and is being applied both in research and in trials show the power of improving technology and its ability to empower people who possess the skills and the desire to use it when it comes within reach.

Does a future where gene therapy becomes routine and conditions humanity has wrestled with for ages become as treatable as a common infection? It depends on people becoming inspired, informed, and involved with the work of doctors like Dr. Manop and the talented team he is a part of. 

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