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The goal is a pill to treat Huntington’s disease

In a significant advancement for medical science, the Lundbeck Foundation has awarded a Frontier Grant of DKK 4.3 million to Associate Professor Ulf Andersson Vang Ørom at the Department of Molecular Biology and Genetics, Aarhus University. This grant aims to facilitate Ørom's ambitious project to develop a groundbreaking medicine for Huntington's disease, a rare and currently incurable neurological disorder.

Huntington's disease, affecting the central nervous system, manifests through symptoms such as involuntary movements, depression, and cognitive difficulties. Current medications can only marginally manage these symptoms without halting the disease's progression. Ørom's innovative approach, which also involves collaboration with Veronica Brito from the University of Barcelona, focuses on RNA-targeted treatments. This novel method aims to block the genetic error responsible for Huntington's disease from causing harmful protein aggregation in cells, a process leading to cell death.



Ørom, an expert in RNA Biology and Innovation, expressed his long-standing interest in this project, noting the challenges faced due to its radical approach. The Lundbeck Foundation, through its Frontier Grants introduced in 2022, seeks to support such innovative research with the potential to develop new treatment forms.

Paul E.G. Kristjansen, MD, PhD, Senior Scientific Director at the Lundbeck Foundation, emphasized the significance of Ørom's project, acknowledging the risks involved in pioneering research but highlighting its necessity for medical advancement.

The Lundbeck Foundation Frontier Grants aim to mature projects to a stage where they can attract investors and train researchers in entrepreneurship to transition lab research into market-ready products. The Foundation, known for its active role in healthcare and philanthropy, annually awards over DKK 500m in grants, focusing significantly on brain research.

Ørom's research breakthrough involves the identification of unique modifications in the diseased mRNA of mice, differentiating it from healthy mRNA. These modifications could allow for targeted medication that selectively eliminates the pathogenic mRNA without affecting healthy cells, a method distinct from previous trials that impacted both healthy and diseased mRNA.

The research team will use the grant to validate these findings in human brain tissue and to demonstrate the efficacy of RNA treatment in targeting diseased mRNA. Successful implementation of this project could not only lead to effective treatment for Huntington's disease, potentially in tablet form, but also pave the way for RNA-based medicines for other diseases, including certain cancers.

As Ørom points out, the complexities and potential of RNA are only beginning to be understood, opening new avenues for medical research and treatment. This project represents a significant step forward in the quest to combat neurological disorders and potentially other diseases through targeted RNA therapy.

For more information on the Lundbeck Foundation and its initiatives, visit www.lundbeckfonden.com.

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