Proof of Concept 2024 Grant recipients: Giulio Cossu for Ospedale San Raffaele

Dilated cardiomyopathy (DCM) is the second most common cause of heart failure and is currently treated with medications aimed at delaying the need for a heart transplant.

Although numerous treatment attempts using stem cells, extracellular vesicles, or viral vectors are underway, none of these strategies have so far demonstrated the desired efficacy.

Thanks to a previous ERC grant, Professor Cossu and his team have developed transplantable cells with unique characteristics: they are immortal and universal, as they do not trigger an immune response. However, a simple extension of this methodology is not feasible, as the existence of resident cardiac stem cells remains controversial, and cell therapies for the heart have so far yielded disappointing results.

To overcome these limitations, Cossu's team aims to produce immortal cardiac cells that are invisible to the immune system directly from the heart. These cells will then be converted into cardiomyocytes through the in vitro expression of cardiac transcription factors.

Since the conversion takes about two weeks, the project plans to introduce these cells into the heart via cardiac catheterization, allowing them to stabilize and differentiate in vivo directly in the damaged areas.

Professor Cossu is in a privileged position to test the feasibility of this innovative strategy, which could pave the way for a new clinical protocol. This approach not only promises to revolutionize therapies for DCM but also exemplifies the transformative potential of cutting-edge research in addressing unmet clinical needs.

Professor Cossu states: "I was very pleased to learn about the funding and I am grateful to the European Commission, and to the ERC in particular, as much of my work has been supported by them for several decades.

The particular significance of this award lies in the possibility of treating dilated cardiomyopathy in Duchenne muscular dystrophy, my long-standing research field, but also in expanding beyond muscular dystrophy to the broader field of dilated cardiomyopathies—diseases affecting thousands of patients who could benefit from an innovative therapy like the one proposed here."

Giulio Cossu is a Professor of Regenerative Medicine at the University of Manchester and a Group Leader at IRCCS Ospedale San Raffaele in Milan. Previously, he was a professor at University College London (UCL), Director of the Division of Regenerative Medicine at San Raffaele, and a Professor of Histology at the Universities of Milan and Rome.

Cossu is renowned for his contributions to the development of skeletal muscle and for his pioneering work in regenerative medicine, using stem cells to treat muscular dystrophy.