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UAE: Toddler gets new lease of life after rare disease treatment

Turkish patient is first to be brought to Dubai to receive therapy for genetic disorder

A Turkish child suffering from a rare genetic disorder was treated successfully through gene therapy in Dubai and is recovering fast in his home country.

The case highlights how Dubai is fast becoming the destination of choice in the Middle East for treatment of genetic disorders through gene therapy.

Fourteen-month-old Arel was suffering from spinal muscular atrophy (SMA), a condition where motor cells die with each passing day. Arel was administered SMA gene therapy at Medcare Women and Children Hospital on December 9, 2021, and the hospital monitored his condition for the next few months to ensure success of the treatment.

Dr Vivek Mundada, the consultant pediatric neurologist who treated Arel said: “Currently, the baby is showing signs of recovery and is back home in Turkey. We are glad to see that he is doing well and is improving with time. Without the gene therapy, his health would have worsened.”

Arel’s mother Dilek Ozbek said that they had approached several hospitals in different countries to get Arel treated before they decided on Dubai. “Many countries could not carry out Arel’s treatment due to lack of expertise in tracheostomy (an air passage to help you breathe when the usual route for breathing is somehow blocked or reduced), but Dubai had the facility, and I had faith in the doctor,” she said.

Arel’s case was complicated since he was on a ventilator in Turkey. When the family flew down to Dubai, doctors faced many challenges. “We had to look at several aspects and not only the medical ones. We also had to arrange a Turkish translator to understand the case and its complexity,” said Dr Mundada. “Arel underwent a clinical check up by different specialists for almost 2-3 weeks to ensure he was stable enough to get gene therapy.”

Since staying long term in Dubai would have been very expensive, Arel had to be discharged from hospital after three months. However, the hospital staff asked them to be in touch with the team via WhatsApp or Zoom. “I am in touch with the mother and his doctors in Turkey to overlook the medicines given by the local paediatric neurologist,” said Dr Mundada.

“Arel is doing fine now and his mother keeps sending his videos and updates to us,” said Dr Mundada.

Medcare is a pioneer in the field of gene therapy to treat children affected with SMA and where they require complex care such as providing air passage via tracheostomy. The hospital has administered the gene therapy to over 23 kids with SMA and these cases have come from Sharjah and some from Ethiopia and Nepal.

Dr Mundada said SMA is a genetic disorder in which muscles become weak and degenerate due to a defective gene.

In gene therapy a normal gene replaces the defective gene, and it is used to treat patients with genetic disorders who harbour a defective gene that hampers the formation of a protein essential for normal functioning. By replacing the defective gene, normal protein is formed which leads to improvement in the condition of the patient.

Dr Mundada said that SMA is a genetic disorder where both the parents are unaffected carriers of a defective SMN1 gene and if their child inherits it, there is no normal gene to compensate for the disease.

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Types of SMAs

There are three types of SMAs in children with varying severity. Type 1 SMA presents in first six months of life and is the most severe with death likely to occur by 2 years of age. Type 2 is less severe, but the children have lot of morbidities, while Type 3 is the least severe with normal survival, but walking difficulties. The defective gene is not always inherited from parents; it can occur spontaneously as well.