New drug could change lives
QUEENSLAND patients with haemochromatosis, Australia's most common inherited disorder, are among the first in the world to trial a potentially life-changing new medication.
Haemochromatosis, which affects an estimated one in 200 caucasians, is an iron overload disorder that can lead to scarring of the liver, an increased risk of liver cancer, diabetes, arthritis and ultimately, an early death unless treated.
University of Queensland liver expert Darrell Crawford said haemochromatosis was traditionally treated by removing blood from the body three to four times a year - a process similar to blood donation.
Without blood removal, a process known medically as therapeutic venesection, Prof Crawford said a patient's organs, including their heart and pancreas, could suffer serious damage from a build-up of iron.
But some patients have problems receiving the treatment because of issues surrounding access to their veins.
"Many patients don't fully comply with the venesection regimen," Prof Crawford said. "Having a treatment alternative would offer more choice."
He said Brisbane's Greenslopes Private Hospital was trialling a synthetic hepcidin, a protein that regulates iron absorption in the gut, which is deficient in most types of hereditary haemochromatosis.
Hepcidin, developed in the US, is injected once a week, similar to how diabetic patients administer insulin.
Prof Crawford, the director of the Gallipoli Medical Research Foundation, which is running the trial, said if the medication proved successful, it could make a big difference.
He said the disorder tended to be underdiagnosed and urged people to see a general practitioner if they had a family history of the condition.
Symptoms include constant fatigue and joint pain.
"Early diagnosis and intervention restores life expectancy to normal for patients with haemochromatosis," Prof Crawford said.
Patients wanting to take part in the Gallipo drug trial should log on to gallipoliresearch.com.au.