University research team develops new therapy to help children with rare hormone deficiency
Professor of Endocrinology at Sheffield leads the spin-out venture Diurnal. A NEW therapy has been developed by researchers at the University of Sheffield to help children suffering from chronic hormone deficiency.
About 4,000 children under the age of six are estimated to be affected by a potentially fatal adrenal insufficiency, with the most common form congenital adrenal hyperplasia (CAH) caused by a lack of the stress hormone cortisol. Children must take daily replacements from birth, with parents having to crush adult tablets to measure an accurate dosage.
A replacement therapy, developed by University of Sheffield spin-out company Diurnal, has been granted market authorisation, which allows for a child-size prescription.
Richard Ross, Professor of Endocrinology at the University of Sheffield and chief scientific officer of Diurnal, said: “Cortisol is an essential stress hormone, and without it, if you have a stressful event, such as an infection or a trauma you will die from an adrenal crisis. Administering adult tablets to babies and young children is a real challenge for parents who have to crush the tablets and then suspend them in water.
“It is incredibly hard to get an accurate dose which can lead to children having too little steroid, which causes an adrenal crisis, or too much steroid, which causes a number of long-term problems including thin bones, thin skin and hypertension.”
Sue Elford, the Living with CAH support group’s chairwoman, said the new therapy, called Alkindi, could make a huge difference, and said: “My son was diagnosed with CAH at two weeks old.
“It was so scary desperately trying to crush up the steroid tablets and measuring the right amount.
“This new therapy will ensure that the dosage of each tablet is specific for individuals and dramatically reduce the long-term effects of too much steroid.”