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Pioneering research into rare neonatal diseases

4th March 2013


Groundbreaking research into rare neonatal diseases, conducted by the University of Sheffield, is set to improve the treatment of babies who lack the stress hormone cortisol.

A European Union grant, awarded to the University of Sheffield and Sheffield Teaching Hospitals NHS Foundation Trust with pharmaceutical Diurnal Ltd and partners, will assist the development of a novel neonatal formulation of hydrocortisone – a steroid drug that is essential for the survival of babies who lack the hormone cortisol.

Known as the stress hormone, cortisol is secreted from the adrenal gland in the body - without it infants cannot survive. Cortisol has a critical role in combating infections or other sources of physical stress such as extreme exercise or surgery.

The most common cause of adrenal insufficiency in neonates is congenital adrenal hyperplasia (CAH). Approximately 1 in every 12,000 children is born with CAH which is a rare genetic disorder that blocks the adrenal glands' ability to synthesise cortisol.

Due to numerous ethical and practical challenges of testing medications, newborns, neonates and infants are often thought to be at a disadvantage when it comes to new medical treatment, because they have to be treated with drugs that were developed for adults.

Professor Richard Ross from the University of Sheffield'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."

The European collaborative project Treatment of Adrenal Insufficiency in Neonates and Infants (TAIN) – Development of Hydrocortisone Preparation for the Treatment of Adrenal Insufficiency in Neonates and Infants, began in December 2011 and involves European leaders in neonatology, paediatric pharmacology and clinical trial methodology, together with a number of small and medium enterprises.

Professor Ross added: "The TAIN project will deliver the first licensed preparation specifically designed for parents to treat their babies with ease of administration, taste masking and appropriately tailored doses.

"As physicians, we are increasingly aware that treatments given as early in life as possible can have an important impact on development and health later in life. TAIN aims to optimise treatment for babies with adrenal insufficiency, and will strengthen paediatric drug evaluation across Europe by building a network of units experienced in clinical research delivering appropriate formulations for neonates and infants."

TAIN coordinator, Martin Whitaker from the University of Sheffield, said: "The funding of the TAIN programme by the European Commission will, for the first time, allow the possibility to develop an effective paediatric medicine for the rare disease adrenal insufficiency.

"The outcomes of TAIN will make a real and significant difference to the loves of young children suffering from this disease, who currently have to rely on unlicensed, sub-optimal medications.

The TAIN project also includes a consortium to monitor all ethical aspects of the research, to ensure that the resulting product will be safe and efficacious for use in children from birth.




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