New research into rare but serious childhood muscle disease
A young Bath medic has been awarded a prestigious year-long fellowship to find out more about a rare but serious muscle disease affecting children and teenagers.
Dr Harsha Gunawardena from the Royal National Hospital for Rheumatic Diseases has been awarded a one-year £76,000 Barbara Ansell Fellowship by leading medical research charity the Arthritis Research Campaign (arc) to study the disease, called juvenile dermatomyositis (JDM).
JDM, an inflammatory form of muscle disease, affects only a few hundred youngsters in the UK and is a chronic (ie incurable) condition which can also affect the skin, joints and internal organs.
Patients often develop extreme tiredness and muscle fatigue, becoming weak and unable to lead a normal life. Drugs and physiotherapy can help, but despite ongoing national research, little is still known about why and how the disease develops.
Bath is a leading centre for research into JDM, with young patients from the South West receiving specialist clinical care at the nearby Bristol Royal Children’s Hospital.
Dr Gunawardena, a specialist registrar, is planning to find out more about how abnormal mechanics of the immune system may play a major role in the disease’s development.
Some preliminary work from the Bath group found that specific autoantibodies associated with specific features may be markers (ie predictors) of more severe disease.
Dr Gunawardena aims to take the research forward by investigating the importance of these autoantibodies in all JDM patients recruited on a national registry of all 300 patients in the UK, held at the Institute of Child Health in London. “We aim to investigate the relationship of these different autoantibodies to each other in JDM, and using some exciting new lab techniques we might also find further new antibodies that are clinically important,” he says.
“If we can start testing for these autoantibodies routinely, then in future we will be able to predict how the disease will pan out, to guide the doctor in prescribing appropriate treatment. So it has huge clinical application.”
