Mutant PLN protein is the major cause for hereditary heart failure in the northern parts of the Netherlands. It is often lethal and there is currently no proven therapy available. A novel therapy can lower PLN protein levels in a laboratory setting. We will further develop this drug utilizing relevant disease models, including explanted human heart tissues. Finally, we will identify patients at need for this novel therapy. This knowledge will allow us to design a first in human clinical trial.
Novel therapies that are successful in the lab often fail in clinical trials. This is in part because laboratory models do not accurately reflect the complex human physiology. We will utilize two innovative disease models consisting of engineered human cardiac tissue and actual patient heart tissue. These later tissues are obtained from waste material during surgery for a mechanical left ventricular assist device or heart transplantation. Data obtained from these models will better inform us on effects to be expected in patients.
Thereby, this project will show us the effects of the novel therapy in relevant human disease models. Additionally, it will inform us which patients, at what point in their disease course, will be the highest urgency to receive such treatment.
This collaboration project between the UMCG and AstraZeneca is co-financed by the Ministry of Economic Affairs and Climate Policy by means of the PPP-allowance made available by the Top Sector Life Sciences & Health to stimulate public-private partnerships.
