Precision medicine in the UAE is set to change lives

Every parent wants the best for their child, particularly when it comes to their health. For families confronted by spinal muscular atrophy – a rare but debilitating genetic disorder – the list of symptoms makes for grim reading indeed.

According to the US Muscular Dystrophy Association, infants with SMA type 0 are often born severely weak. Such babies can have facial paralysis and congenital heart defects. Heartbreakingly, some patients can die from respiratory failure by six months of age, others within their first month. Older sufferers can have severe disability as motor neurons – nerve cells that carry signals from the brain to the muscles – are affected, causing weakness in the limbs and other problems, such as difficulty breathing or swallowing.

Compounding the cruelty of this illness is the cost of treating it. The best remedy for SMA is gene therapy but this cutting-edge medicine is beyond the reach of most. One drug, Zolgensma, has been described as the world’s most expensive medicine, costing about $2 million for each patient. Although this situation won’t change overnight, news from the UAE this week gives a glimmer of hope.

Dubai’s Medcare Royal Specialty Hospital has announced it will become one of the first centres outside the US to offer groundbreaking gene therapy for SMA, with one neurologist telling The National that this drug’s approval is the first step to making the Emirates a global hub for the treatment. Earlier this month the Emirates Drug Establishment approved Itvisma for the UAE, making the country just the second in the world after the US to give the drug the green light.

Although this is good news for SMA patients, it has wider implications for medicine in the Middle East. The UAE’s approval of Itvisma could spur other countries into speeding up their approval processes for similar treatments as well as encouraging more screening. Having more regional hubs specialising in SMA treatment will increase collaboration and research between hospitals and universities, building on homegrown scientific expertise and reducing reliance on foreign medical centres.

That would be good for future SMA strategies; as more people come to the Emirates for treatment, clinicians will gain more experience, techniques will be refined and procedures streamlined. With time, it is possible that case studies in the UAE could help to change the cost profile of drugs such as Itvisma, bringing it within reach of more patients. Having patients from the Middle East and surrounding region would give greater insights into the DNA from this part of the world.

Already, work by UAE-based specialists on SMA has led to healthcare advancements. Doctors from the Genomics Centre of Excellence at Al Jalila Children’s Specialty Hospital in Dubai have collaborated with US biotech company Asuragen on the development and optimisation of a cost-effective SMA test for newborns. Experts at Al Jalila have also developed another test to detect many genetic variants linked to SMA.

Despite such progress, a long road lies ahead. However, with its advanced healthcare system and international transport links, the UAE looks set to drive research and treatment for conditions such as SMA in the years ahead. But perhaps the most important thing such progress offers is not a quick fix but hope that, with time, no disease will be beyond treatment.