With announcement of high-profile clinical trials of innovative therapies
Abu Dhabi: In the advent of International Thalassemia Day, an occasion devoted to raising awareness about the serious health implications of the blood disorder, Burjeel Medical City (BMC), Abu Dhabi’s leading medical facility and subsidiary of VPS Healthcare Group, is celebrating its achievements and planned stellar contributions to the field.
The hospital is preparing to begin two Phase 3 clinical trials of a new, first-in-class enzyme-activating drug which has direct effects on survival of red blood cells and already demonstrated potential in earlier trials for transforming the treatment of thalassemia by ameliorating the hallmark of the disease – chronic hemolytic anemia. One trial will be conducted in transfusion-independent patients with the aim of increasing hemoglobin level and improving quality of life, and the other trial will be conducted on transfusion-dependent patients with the aim of decreasing transfusion requirement and thus decreasing the burden of the disease on the patient and healthcare system.
BMC will enlist the expertise of top thalassemia medical experts in their healthcare network, who are global key opinion leaders in their own right, to conduct the trials. BMCs state of the art technology will remain key for the success of the trials’ requirements of short and long-term assessments and monitoring.
Dr. Khaled Musallam, Group Chief Research Officer at VPS Healthcare and Principal Investigator on the trials, said: “The management landscape for patients with thalassemia is swiftly evolving, with several novel therapies already in advanced stages of development. There are several unmet needs with current conventional therapies and the only path forward to address those and improve patient outcomes is to bring discoveries at the bench to the patient’s bedside through collaborative research.”
BMC’s recent collaboration agreement with HH Sultan Bin Khalifa Al Nahyan Humanitarian & Scientific Foundation also marks a tremendous move towards improving healthcare access in the field. BMC offers the most modern and advanced treatments in the UAE for thalassemia patients, such as bone marrow transplantation, making them the most suitable provider for the Foundation to refer patients for treatment.
Mr. John Sunil, CEO of Burjeel Hospitals in Abu Dhabi, said: “We are always ready to bring on board cutting-edge technologies and attract talented physicians and scientists to our medical facilities if it means we can offer state of the art treatment options for patients. This becomes more imperative for rare diseases which are often left with poor access to optimal management and chronic holistic care.”
Thalassaemia is an inherited blood disorder which affects the production of hemoglobin in the bloodstream, impacting the ability of red blood cells to efficiently carry oxygen around the body. Thalassemia is a lifelong and a far more morbid disease than common acquired anemias like iron deficiency. Although considered a rare disorder, they are among the most common genetic diseases worldwide and they are particularly highly prevalent in the Middle East region. Patients suffer an array of serious clinical complications due to their severe anemia and many require regular monthly transfusions for survival.
With the Thalassaemia International Federations theme of “Be aware. Share. Care: Working with the global community as one to improve thalassaemia knowledge”, this year’s International Thalassaemia Day aims to shed light on the awareness aspect of thalassemia, initiating educational campaigns around the topic amongst the public, as well as targeting stakeholders involved in providing care to thalassemia patients to maximize the facilitation of advanced solutions in medical care. Advances in management of thalassemia would have significant benefits for the public health sector overall, as well as for patients and their families.