The Applied Stem Cell Technologies research group, led by Prof. Robert Passier and part of the University of Twente’s Department of Bioengineering Technologies, has received funding for BEAT-PLN. This research programme aims to accelerate the development of new treatments for PLN-R14del cardiomyopathy. This inherited heart muscle disease is caused by a mutation in the phospholamban (PLN) gene and can lead to severe cardiac arrhythmias, heart failure and sudden cardiac death. Within BEAT-PLN, the University of Twente is collaborating with PLaN Therapeutics on a project with a total value of €900,000.
Within BEAT-PLN, the University of Twente and PLaN Therapeutics are developing an advanced heart-on-a-chip platform based on human pluripotent stem cell-derived cardiomyocytes from patients carrying the PLN-R14del mutation. This fully human, animal-free model system makes it possible to closely mimic the disease and investigate both the underlying disease mechanisms and the effects of potential treatments in human heart tissue.
Faster route to new treatments
PLN-R14del cardiomyopathy is a rare inherited heart muscle disease that originated in the northern Netherlands. It is believed that the genetic mutation first occurred around 700 years ago in a Frisian ancestor. All carriers of the PLN mutation are descendants of this common ancestor. Many PLN carriers still live in the northern Netherlands. As a result of migration and emigration, smaller numbers of carriers can also be found elsewhere in the Netherlands and abroad (see the PLN website for more information).
Cardiac arrhythmias, heart failure and sudden cardiac death
The disease can lead to severe cardiac arrhythmias, heart failure and sudden cardiac death. Currently, medication is available only to manage symptoms; there is no therapy that addresses the underlying genetic cause. In addition, animal models and traditional cell models only partially reproduce the disease, making it difficult to predict how effective new therapies will be in patients. Better human disease models are therefore essential for the development of new treatments.
New programme
By combining innovative therapy development with advanced human disease models, BEAT-PLN aims to accelerate the development of new treatments for PLN-R14del cardiomyopathy. The programme will increase our understanding of the disease and support the development and evaluation of potential treatments. In doing so, the partners are taking an important step towards new treatment options for people affected by this inherited heart muscle disease.
Funding was awarded through the Health Holland Organ-on-a-Chip call. This support enables the partners to pursue several innovative research lines in parallel and further explore new technologies for treatment development.
The collaborative project HH-PPS-25083-OoC is co-funded through a PPP Allowance made available by Health Holland to stimulate public-private partnerships.
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