Back to birth to beat blood disorders

$5.4 million funding will allow Monash researchers to develop novel approaches to treat major blood disorders, by switching on a form of haemoglobin normally only used by babies before birth.

The Drug Discovery Award funding from the Wellcome Trust, a global charitable foundation dedicated to improving health, will see the University’s Faculty of Medicine, Nursing and Health Sciences work in partnership with Cancer Therapeutics CRC (CTx), a leading Australian drug discovery and development company, and The Walter and Eliza Hall Institute of Medical Research (WEHI).

It’s hoped that developing novel new drugs will lead to improvements to the lives of patients suffering from the devastating blood diseases thalassemia and sickle cell disease. Disorders of the oxygen carrying protein haemoglobin are the most common genetic diseases worldwide, affecting millions of the global population, and consuming 30 per cent of the donated blood supply.

The work builds on more than 20 years of research by CTx on ‘foetal haemoglobin’, which has the potential to replace damaged adult haemoglobin, allowing patients to live normal lives, without regular blood transfusions or damage to vital organs. The work is being led by Professor Stephen Jane, Head of the Central Clinical School at Monash University and Director of Research at the Alfred Hospital.

Professor Jane said the Wellcome Trust’s Seeding Drug Discovery Award is designed specifically to help researchers take later stage projects towards clinical trials.

“The CTx partnership provided the necessary translational expertise needed to turn our medical and scientific research into novel treatments, and enabled the project to reach a stage where it was ready to progress into clinical development,” he said.

“The Wellcome Trust award allows us to test our drugs in this important area in addition to maintaining our focus on early stage cancer,” he said.

Dr Ian Street, Chief Scientific Officer for CTx and Principal Investigator for the project, said the collaborative framework will drive forward Australian research translated into new treatments with the potential to change the lives of patients across the world.

“The plan we have agreed with the Wellcome Trust will advance the project rapidly towards the first clinical trials here in Australia,” he said.

(Source: Monash University)