There is an urgent need for better treatment for myelodysplastic syndrome (MDS), a type of blood cancer, and new research published in prestigious scientific journal Nature Communications suggests a breakthrough could be coming. Researchers at the University of Heidelberg in Germany have reported results claimed to be “the best we have ever observed in our pre-clinical models of MDS with primary patient samples.”
As a peer-reviewed journal by the top medical researchers around the world, the findings are significant for cancer research globally. The article details a pre-clinical collaboration with the University of Heidelberg investigating the role of lysyl oxidase enzymes in MDS and the effect of combining 5-azacytidine (5-AZA) with PXS-5505, a pan-lysyl oxidase inhibitor discovered and owned by Australian biotech company Pharmaxis (ASX: PXS).
The current standard of care for MDS are hypomethylating agents like 5-AZA but the team at University of Heidelberg were taken aback by the impressive results when combining 5-AZA with PXS-5505.
The combination showed formation of red blood cells from bone marrow taken from patients is significantly restored when treated with PXS-5505 plus 5-AZA in 20/31 cases (65%) versus 9/31 cases (29%) treated with 5-AZA alone.
Co-lead researcher Professor Daniel Nowak said, “This study is one of the first published showing that re-modelling the extracellular matrix and bone marrow microenvironment can induce outstanding improvements of haematopoiesis in these diseases.
“The results of PXS-5505 in combination with 5-AZA are the best we have ever observed in our pre-clinical models of MDS with primary patient samples.
“The significant boost in erythropoiesis achieved by adding PXS-5505, allied to its favourable safety profile makes the combination of 5-AZA + PXS-5505 interesting for both high and low risk MDS as well as chronic myelomonocytic leukemia, myelofibrosis and low blast acute myeloid leukemia, filling a significant gap in the current treatment landscape of these diseases.”
MDS comprise a group of blood cancers that share clinical and pathologic features with acute myeloid leukemia (AML), a type of cancer in which the bone marrow makes a large number of abnormal blood cells. Patients with MDS are at risk for symptomatic anaemia, infection, bleeding, and transformation to AML.
MDS occurs most commonly in older adults with an annual incidence reported to be 75 cases per 100,000 people. Approximately 50% of MDS patients initially respond to the current standard of care treatment but subsequent relapse is almost certain, highlighting an urgent need to improve the current standard of care.
As impressive as the MDS research data is, it is likely to come as no surprise to the team at Pharmaxis which already has PXS-5505 deep into the path for commercialisation with human patients nearing the completion of a Phase 2 clinical trial.
For those trials, PXS-5505 is being used to treat patients suffering from myelofibrosis (bone marrow cancer) with interim data highly encouraging, showing the drug’s ability to improve bone marrow fibrosis scores as well as stable or improved platelet or haemoglobin scores. This trial is now over 80% recruited and Pharmaxis has scheduled a review meeting with the Food and Drug Administration (FDA) to discuss the data from this study and validate its path to FDA approval.
The full peer-reviewed journal article is available in Nature Communications here.
Alfred Chan is a Business Reporter at The Sentiment specialising in ASX-listed small cap companies, a bloodstock enthusiast and former equities analyst.
Latest posts by Alfred Chan (see all)
- Harris Technology to expand refurbished tech division amid rising demand from cost-conscious Australians - April 30, 2025
- Harris Technology secures major investment from Taiwan’s FSP Technology at 100% premium - March 10, 2025
- ARC Funds acquires 30% of auzbiz Capital as latest direct-to-investor marketing venture - October 8, 2024
*Owners of this website are shareholders in a company mentioned in this article and have been engaged by them to assist in investor communications
Leave a Comment
You must be logged in to post a comment.