1. ITV Report

Study suggests epilepsy drug can be used to treat form of dwarfism

A drug used to treat conditions such as epilepsy has been shown in lab tests to significantly improve bone growth impaired by a form of dwarfism.

The research team, which includes scientists at Newcastle University, discovered in lab and mouse studies that the drug carbamazepine, already approved for treating conditions such as epilepsy and bi-polar disease, can significantly reduce the effects of MCDS - a genetic condition which affects bone growth.

Carbamazepine. Credit: Newcastle University

MCDS leads to skeletal dysplasia, commonly referred to as dwarfism, where patients are often short in stature with unusual limb proportions.

There is no current treatment.

Newcastle University said that human trials will take place by the end of the year

Human trials will be the next step. Credit: ITV News

The team which has made the breakthrough are from Newcastle University’s Institute of Genetic Medicine, The University of Manchester and Murdoch Children’s Research Institute in Australia.

Their findings have been published in The Journal of Clinical Investigation.

Scientists at Newcastle University worked with counterparts in Manchester. Credit: ITV News

"The concept of going so quickly from pre-clinical data to orphan drug designation to a clinical trial is incredible.

“It exemplifies the power of drug repurposing for rare disease: there has been no involvement of big pharma and this inexpensive drug has had a great safety record since the 1950s.”

– Michael Briggs, Professor of Skeletal Genetics, Newcastle University

"Carbamazepine is an inexpensive drug which has been used to treat conditions such as epilepsy and bi-polar disease for decades.

“So the possibility that it may be effective in MCDS is exciting and needs to explored further.

“But clearly, the next stage is to test it in humans.”

– Professor Ray Boot-Handford, University of Manchester