Medical researchers from UNSW have developed a new way to predict whether patients with the blood condition myelodysplastic syndrome (MDS) will respond to treatment.
More than 1400 people in Australia are diagnosed with the disease each year, which is characterised by impaired peripheral blood cell production and abnormal bone marrow.
MDS is much more common in older individuals and, if left untreated, the prognosis can be very poor.
Bone marrow transplantation could cure MDS but as an invasive procedure is often not tolerated by older people.
Instead, the best alternative is the drug azacitidine (AZA), which can improve blood production and decrease the likelihood of MDS progressing into leukemia.
“About half of MDS patients will not respond to AZA, with few alternatives for those who fail treatment,” says UNSW Research Fellow Dr Ashwin Unnikrishnan.
“Also, it takes about four to six months before we can tell if someone is responding to AZA treatment, at which point months of futile treatment has been given to those who are resistant to it.”
To tackle this issue, UNSW Medicine researchers led by Dr Unnikrishnan and Professor John Pimanda set out to develop a way to measure AZA in patients receiving the treatment, to predict their response, and building on their recent discovery that increased cell cycle quiescence of blood cells is a characteristic of patients who fail to respond to AZA treatment.
“These findings are really exciting, because AZA-MS now allows us to identify early on which patients will not respond to AZA,” Dr Unnikrishnan said.
In early 2018, the AZA-MS method, along with another related test, will be used in a UNSW clinical trial led by Professor Pimanda, together with the pharmaceutical company Celgene and multiple hospitals in New South Wales.
“One of the benefits of using the test in this trial is that it will help determine if people respond to AZA tablets, compared to the currently used injectable form,” says Professor Pimanda.
“The hope is that the AZA tablets will be effective for people who would otherwise not respond to AZA, and that the overall response to AZA improves,” Dr Unnikrishnan said.
“The clinical trial will also allow us to collect samples that will enable us to answer long-term questions aimed at improving treatment in MDS.
“Altogether, we are hoping to fundamentally improve treatment for MDS patients, with the test as a big step towards personalised therapy.”