Researchers to develop vital steroid treatment for babies

A European Union grant, awarded to Sheffield University and Sheffield Teaching Hospitals NHS Foundation Trust, will go towards developing a new neonatal formulation of the steroid hydrocortisone.

Cortisol has a vital role in fighting infections, or other sources of stress such as extreme exercise or surgery.

But approximately one in every 12,000 children is born with congenital adrenal hyperplasia (CAH), a rare genetic disorder that blocks the adrenal glands’ ability to produce cortisol, and there is no steroid designed especially for babies with which to treat them.

Professor Richard Ross, from Sheffield University’s medical school, said: “Adrenal insufficiency is a serious condition resulting from a lack of stress hormone cortisol, and untreated patients will die from an adrenal crisis.

“Currently there is no licensed preparation to treat neonates and infants with adrenal insufficiency, and parents have to use crushed adult tablets, which have associated risks of over- and under-treatment.

“Under-treatment can be potentially lethal, while over-treatment results in growth failure and obesity.”

Martin Whitaker, from the university, said that the funding will, for the first time, “allow the possibility to develop an effective paediatric medicine for the rare disease adrenal insufficiency.”