Thalassemia–Reactive oxygen (T-REX)

Development of novel reliable biomarker to investigate the clinical potential of oxidant-ektacytometry for assessing drug efficacy in thalassemia patients

In this project, RRM will collaborate with medical partners University Medical Centre Utrecht, Erasmus Medical Centre and University of Toronto to develop a novel oxidant-ektacytometry biomarker for monitoring the response of red blood cells to oxidative stress in thalassemia patients. 

Thalassemia is a hereditary red blood cell disorder affecting 1-5% of the global population. Current treatments are invasive, requiring lifelong blood transfusions and medication, with up to 80% of patients not responding to certain therapies. Existing biomarkers lack reliability and only reflect long-term treatment effects. As migration from high-incidence regions increases, the medical care costs for thalassemia patients in developed countries are expected to rise, necessitating innovative solutions for more effective and personalized treatment. 

The approach aims to develop the specific oxidant biomarker, optimal test conditions, as well as the verification of oxidant ektacytometry on a large cohort of thalassemia patients. This approach will gain insight into the mechanisms of the novel biomarker that may become an important readout instrument for centers treating thalassemia patients worldwide, and to be incorporated in future clinical trials as surrogate endpoint. This approach will enable more precise monitoring of treatment efficacy and allow for tailored treatment plans. The innovative solution has the potential to improve patient outcomes, reduce healthcare costs, and advance personalized medicine not only for thalassemia but also for other disorders characterized by increased oxidative stress. 

The deliverables include a Standard Operating Procedure (SOP), detailed medical patient descriptions correlating with oxidant-ektacytometer results, a comprehensive understanding of outcome parameters, scientific publications, and feedback from conferences and workshops with thalassemia opinion leaders to enhance clinical practice and research in this area. Tailoring the treatment approach to patient needs will also incur cost savings for the society, protect patients from side effects while saving healthcare costs, and will provide a large step forward into personalised medicine. 

Paper on Red Blood Cell Deformability Demonstrated with the Oxygenscan from RR Mechatronics 

Figure 1: Blood of thalassemia

 

Summary
The consortium aims to develop a novel oxidant-ektacytometry biomarker for monitoring clinical severity and treatment efficacy in Thalassemia patients. A novel technology for measuring red blood cell deformability changes under oxidative stress will be created, exploring its potential for evaluating drug efficacy in patients.
Technology Readiness Level (TRL)
3 - 6
Time period
24 months
Partners
UMC logo
Erasmus mc logo
University of Toronto logo