Back to birth to beat blood disorders

09 April 2015
Related topics
Research to improve the lives of patients suffering from devastating blood diseases has received a $5.4 million boost from the Wellcome Trust.
Dr Ian Street in the laboratory
Dr Ian Street is the principal investigator on the
project and chief scientific officer at the
Cancer Therapeutics CRC.

The Wellcome Trust Seeding Drug Discovery Award funding was awarded to the Cancer Therapeutics CRC, an Australian drug discovery and development company of which the Walter and Eliza Hall Institute is a partner. The Wellcome Trust Seeding Drug Discovery Award is designed specifically to help applicants take later stage projects towards clinical trials.

The Cancer Therapeutics CRC is working closely with partners at the Walter and Eliza Hall Institute and Monash University to develop drugs from one of its discovery programs that could help patients suffering from the blood disorders thalassemia and sickle cell disease.

Thalassemia and sickle cell disease are disorders of the oxygen-carrying protein haemoglobin. They are the commonest genetic diseases worldwide, affecting millions of people globally, and consuming 30 per cent of the donated blood supply for treatment.

The Cancer Therapeutics CRC has led development of novel drugs that aim to treat these diseases by switching on the form of haemoglobin – foetal haemoglobin – normally only used by babies before birth.

This foetal haemoglobin can replace the damaged adult haemoglobin and allow patients to live normal lives, without regular blood transfusions or damage to vital organs. The drugs being developed by the Cancer Therapeutics CRC are underpinned by more than 20 years of research led by Professor Stephen Jane from Monash University and the Alfred Hospital.

Walter and Eliza Hall Institute researcher Dr Ian Street is chief scientific officer for the Cancer Therapeutics CRC and principal investigator on the project.

“The Wellcome Trust award allows us to test our drugs in this important area in addition to maintaining our focus on early stage cancer,” Dr Street said. “The plan we have agreed with Wellcome will advance the project rapidly towards the first clinical trials here in Australia.”

The Cancer Therapeutics CRC is headquartered in Melbourne and funded through the Australian Government CRC Program. The major focus of CTx is developing cancer drugs but the potential benefit for these other major diseases could not be ignored so CTx turned to the Wellcome Trust for assistance.

Dr Warwick Tong, chief executive officer of the Cancer Therapeutics CRC, said if the project progressed successfully the first trials in humans should be starting in about three years.

“This is another example where, by working under the collaborative framework supported by CRC Program funding, we can take superb Australian research and translate it into new treatments with the potential to change the lives of patients across the world,” Dr Tong said.

Further information

Liz Williams
Media and Publications Manager
M: +61 428 034 089
E: williams@wehi.edu.au

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