First-ever International TREATWolfram Trials take place at our Clinical Research Facility: Finding a cure for Wolfram Syndrome
This 'Global Wolfram Syndrome Awareness Day 2021', Professor Tim Barrett, Consultant in Paediatric Endocrinology and Diabetes, and Wolfram Syndrome (WS) Principal Investigators Dr Ben Wright and Dr Renuka Dias share with us their insights into the TREATWolfram Trial taking place at our Birmingham Children's Hospital and the Queen Elizabeth Hospital Birmingham.
Wolfram Syndrome (WS) is a rare inherited condition affecting only one in 500,000 people across the globe. The life-limiting condition has a number of gruelling primary symptoms referred to as 'DIDMOAD', meaning - Diabetes Insipidus (DI), Diabetes Mellitus (DM), Optic Atrophy (OA) the irreversible deterioration of the eyes, and Deafness (D). Further symptoms may also include bladder and bowel dysfunction, and problems with the inner ears and areas of the brain that affect eye movement and balance. Sadly, the average life expectancy of those with the condition is shortened significantly. The illness is often confused in its early stage, as common diabetes – until other symptoms related to the disorder begin to surface.
The Clinical Research team's therapeutic advancements in rare diseases, throughout the TREATWolfram Intervention demonstrates the potential to develop an 'orphan drug' - a rare treatment medication, used to slow or treat the illness; prolonging the life expectancy and quality of life for patients with Wolfram Syndrome. The international trial is an ongoing medical and began in January 2019, due to end in 2024 - with approximately fifty-nine patient participants recruited so far, including 12 at our Children's Hospital. The trial is seeking an additional six participants by the end of October; we would like to raise awareness and share education on the syndrome to ensure those with the illness can seek diagnosis and treatment at a quicker rate.
The trial expands across countries such as Spain, Poland, and France, meaning that this is the first international trial of its kind. The trial underwent a series of logistical challenges throughout Brexit, and the COVID-19 Pandemic - as the expenses for international shipping of medical treatment rose. We'd like to say a big 'thank you' to the Medical Research Council (MRC) for providing additional funds needed to ensure that the trial continued – and our fantastic clinical trials unit, particularly trial leaders Darren Barton and Sabrina Cronier.
Professor Tim Barrett said:
"Without the support from the Research Teams (nurses, doctors and other health professionals) at BWC and QEH, we would not have achieved such fantastic recruitment for this trial. I'd also like to add a massive 'thank you' to all of the TREATWolfram Group, including National Co-ordinating Centres and each site's clinical and non-clinical team members. We're proud of all of those involved across all disciplines and would like to give a special thanks to Wolfram Syndrome UK for supporting our participants during their visits with us."
The main trial international site is based in Birmingham, as our Children's Hospital treats 90 percent of all children with this neurodegenerative condition – pioneering the first and only potential cure for Wolfram Syndrome. Renuka Dias, a Paediatric Endocrinology and Diabetes Consultant at the Hospital for six years, and the Principal Investigator for our Children's Hospital TREATWolfram Trial; alongside Dr Ben Wright, Neurology Consultant at the Queen Elizabeth Hospital of four years, and Principal Investigator shared their thoughts on Global WS Awareness Day.
Dr Renuka Dias said:
"We are lucky here at Birmingham Children's Hospital to be able to offer a multi-disciplinary clinic supporting families with young people diagnosed with Wolfram Syndrome. Raising awareness of this incredibly challenging condition is key to helping individuals and the wider Wolfram Syndrome community. In this, we are also well supported by WS UK.
I also want to thank all the young people and their families who have continued to take part in the TREATWolfram trial throughout the COVID-19 pandemic to help Improve our understanding and treatment options for this rare disease."
Dr Ben Wright also added:
"Raising awareness of rare conditions, like Wolfram syndrome, is important as this will allow people to be diagnosed earlier and be referred to specialist teams sooner. The adult Wolfram service is comprised of a number of clinicians from different specialist areas to provide a joint, comprehensive assessment of people with Wolfram syndrome.
I am immensely impressed and humbled by those people who have taken part in the TreatWolfram clinical trial, especially during COVID-19 restrictions, as without them, we would not be able to investigate whether treatments of this syndrome can be realised."
We'd like to express our admiration for the bravery of those with Wolfram Syndrome – and say a big 'thank you' to all of those involved in making that intervention a possibility to uncover the cure for this challenging and life-limiting illness.