A potential new application for the drug Sulforadex – a treatment for Noonan syndrome

An investigation of the potential therapeutic effects of Sulforadex in Noonan syndrome

Noonan syndrome is a genetic disorder that affects up to 1 in 1,000 births and is known to cause heart conditions, learning problems and short stature. In half of people with Noonan syndrome, the genetic cause is a mutation in a gene called Shp2. This mutation causes an enzyme to be dangerously overactive. In 2013 the BHF funded Professor Eaton’s team to find the targets of a new drug called Sulforadex, which is being tested in prostate cancer drug trials. They found that Sulforadex inhibits the activity of the Shp2 enzyme. Now Professor Eaton and his team want to see whether Sulforadex could be used as a treatment for Noonan syndrome. In this latest project, the team will test this theory using Sulforadex and a similar compound. They will treat the mice with Noonan syndrome with these medicines and see if they can improve their growth and heart function. If successful, this work could lead to clinical trials of the drugs in humans, and result in a new treatment for Noonan syndrome.