New Drug For Bone Disease Babies

A new drug could help save the lives of babies dying from a rare genetic bone disease, experts have revealed.

Children with severe hypophosphatasia, which affects just one in a million people, began being treated with the drug three years ago after it was developed by bone research scientists in the USA and Canada.

The results of the trial, showing that the asfotase alfa drug had been successful, were published in the medical journal The New England Journal of Medicine this week.

Experts from The Children’s Hospital, Sheffield, and the University of Sheffield, the largest site in the worldwide trial, said it was “crucial” the drug was licensed as soon as possible.

Eleven babies were treated during the trial, with three of those, from Germany, Greece and Northern Ireland, being given the drug in Sheffield.

The children were injected with the drug three times a week and clear results emerged after around three to six months, experts said.

Nine of the babies completed the trial.

Sufferers of severe hypophosphatasia, which is thought to affect just 35 babies worldwide, normally die after the age of six months and children struggle to walk or use their limbs properly, are very weak and in pain. Less severe forms are seen in older children and sometimes in adults.

Professor Nicholas Bishop, an expert in metabolic bone disease at Sheffield Children’s NHS Foundation Trust and The University of Sheffield, said: “The long-term effects of this drug are not yet known but the results we’ve had so far on this trial are undeniable. It’s saved and changed the lives of nine children who would otherwise have died after just a few months.”

Prof Bishop, who has been developing Sheffield’s bone service to become the leading European centre in the field for the last 13 years, added: “The trial has been a fantastic success and it’s crucial we now get the drug licensed as soon as possible and begin helping children born with this disease.”