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Dubai is conducting clinical trials for rare diseases with no cure, cancer
The carefully monitored clinical trials offer patients with rare genetic and complex diseases access to innovative treatments before they become widely available
- PUBLISHED: Wed 11 Feb 2026, 11:58 AM
An ambitious programme by Dubai Health is trialling investigational treatments for rare diseases, some with no known regulatory-approved disease-modifying therapies yet.
At a time when patients with potentially fatal conditions have to wait for a long time to get access to new approved treatments, the initiative is offering early access to promising therapies through carefully monitored clinical trials.
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Led by Dr Alshafi Mohammad, Clinical Trials Office at Dubai Health, the programme offers hope for many families, especially those with children suffering from rare genetic disorders. For them, this could mean the difference between years of uncertainty and a new shot at life.
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“We are studying novel treatments for complex conditions such as cancer, rare diseases, metabolic disorders and neurodegenerative conditions,” said Dr Alshafi. He was speaking to Khaleej Times on the sidelines of the World Health Expo (WHX) which began at the Dubai Exhibition Centre (DEC) on Monday.
Improve quality
He explained that many of these treatments can be lifesaving and may greatly improve a patient’s quality of life — especially for those with rare diseases where options are limited or regulatory-approved disease — modifying therapies do not exist yet.
“Normally, patients have to wait five to 10 years for new treatments to be studied and approved,” he said. “Through clinical trials conducted at Dubai Health, our patients can access these treatments many years in advance.”
This means patients in Dubai, across the UAE, and even in nearby countries have the opportunity to receive promising therapies long before they become widely available. “Our motto is changing lives through clinical trials,” he added.
Focus on rare conditions
Dubai Health is currently running trials for several rare conditions. These include Microvillus Inclusion Disease (MVID), a serious congenital intestinal disorder that affects children, and Primary Hyperoxaluria, a metabolic disorder that can lead to kidney failure.
Another major focus is ENPP1 deficiency, a rare inherited genetic disorder. “There is no regulatory-approved disease-modifying therapies yet,” said Dr. Alshafi. “Through clinical trials, children are gaining access to novel therapies. Lives are being changed.”
He described the ENPP1 study as one of the most promising trial underway. “If there are no approved treatments available and we are bringing hope to patients, that is a huge emphasis for us.”
The programme also includes trials in cancer, including early breast cancer, as well as blood disorders such as thalassemia.
Early access
While many trials in the region are late-stage studies, Dubai Health is now conducting early-phase trials, including Phase Two protocols.
“This is very important because we can study how well treatments are working for our patients and determine the right dose at an early stage,” he said.
Some of the treatments being tested include RNAi therapies, biologics and monoclonal antibodies, an advanced forms of therapeutic approaches that target diseases more precisely.
Patients are usually introduced to trials during their regular hospital visits. Doctors explain the study in detail, including benefits and risks, before patients decide whether to take part.
“With our specialised pediatric facilities and wide range of adult services, we are uniquely positioned to conduct these trials,” Dr Alshafi said. “Ultimately, it is about giving patients new hope and advancing health for humanity.”
