Breast cancer drug repurposed for congenital hyperinsulinism

Checking a baby's glucose levels

In a world first, Sidra Medicine, a member of Qatar Foundation, has pioneered a new treatment protocol for a young infant with congenital hyperinsulinism. The findings were published in the New England Journal of Medicine.

As the patient wasn’t responding to conventional medical therapies, the Sidra Medicine team trialled a new treatment protocol using Alpelisib. Within six weeks, the patient started to show a vast improvement in her blood sugar levels.

The treatment decision followed previous case reports where adult patients with breast cancer, being treated with the same drug, were shown to have experienced high blood glucose profile.

Alpelisib is a protein-targeted medication that is used for treating breast cancer and overgrowth disorders in children. The medication regulates cell growth, proliferation and development and insulin signalling. One of its common adverse effects is increased glycogenolysis and gluconeogenesis, resulting in patients taking the drug experiencing hyperglycaemia.

The treatment protocol was implemented by a team of endocrinologists, led by Professor Khalid Hussain, Division Chief of Endocrinology at Sidra Medicine.

“For most children, hypoglycaemia cannot be controlled with conventional medications and pancreatectomy remains the only option. This is a major procedure and the children then have to live with lifelong diabetes mellitus and pancreatic exocrine insufficiency,” said Prof Hussain.

“We will now be trialling this method in other paediatric patients with the same condition, who do not respond to conventional medical therapies. We call upon the wider medical community to conduct further clinical trials as the more research done in this field can help find better, safer and faster treatment methods for patients with congenital hyperinsulinism. This will radically change the way these patients are managed and could benefit thousands of children around the world.”

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