The Nijmegen Therapy Accelerator for Rare Diseases supports researchers in developing new treatments for rare conditions. Prof. Dr. Dirk Lefeber (Radboudumc Nijmegen) shares the vision behind this initiative:
“In recent years, much effort has gone into improving care and diagnostics. Now it’s time to accelerate therapy development.”

“Roughly one million people in the Netherlands live with a rare disease, which can have a profound impact on quality of life and even life expectancy,” says Lefeber. “When we look at rare diseases as a collective, we’re dealing with a major societal issue. That’s why we want to take a similarly unified approach in research. It means applying lessons learned in the development of one therapy to other rare diseases. And that calls for collaboration – here in Nijmegen and across the country.”

The Value of Collaboration

In recent years, significant progress has been made in organizing care for people with rare diseases – in part due to the efforts of the Dutch Federation of University Medical Centers (NFU). Within UMCs and some general hospitals, centers of expertise have been established for the diagnosis and treatment of rare diseases, often embedded in European networks of clinicians and researchers.
Lefeber: “My own research focuses on rare metabolic disorders related to sugar metabolism. Thanks in part to genetic research, we’ve made great strides in diagnostics and have identified and described several new diseases. Now the time has come to strengthen and speed up the development of effective treatments. That includes drug repurposing, DNA- or RNA-based therapies, and cell therapies. Collaboration is crucial in all of these areas because there are always commonalities. By sharing expertise, we can move much faster from creative idea to clinical application – and do so in a socially responsible way.”

Joining Forces

Lefeber and his colleagues in Nijmegen have already experienced the added value of working together:
“At Radboudumc, we saw that we’d had success in a number of therapy development efforts: dendritic cell vaccination for rare cancers, RNA therapy in ophthalmology, and n=1 trials in neurology. There were many examples of isolated successes. That made us ask: wouldn’t it be better to bring this all together to accelerate and strengthen the process? We received funding from the government to do just that – and that’s how the Therapy Accelerator for Rare Diseases was born.”

The program currently focuses on two key areas: therapy pathways and learning communities.
The first serves as an advisory function for researchers looking to turn an idea into a concrete treatment. Drawing from existing experiences and case studies, the team is developing a step-by-step framework for therapy development. The goal is to systematically gather and make available expertise across the entire development process – “with the end goal in mind.”

The learning communities focus on collaborative topics such as drug repurposing.
Lefeber: “These groups aim to share knowledge, develop education programs, and connect people within and beyond the UMC – including, for example, our regional investment fund.” Currently, they are exploring which topics would benefit most from this community-based approach.

According to Lefeber, collaboration is also crucial when it comes to methodological challenges:
“A key question in preclinical research is the predictive value of model systems. Whether you’re working with human organ-on-a-chip models or animal models, you always need systems to test potential treatments. It’s also relevant when deciding on the most suitable delivery method for gene therapy or oligonucleotide-based treatments. By looking at existing successful therapies, you can choose more effective delivery strategies early on – and avoid, for instance, using a vector that’s under a costly commercial patent. These are precisely the types of questions we should tackle not just within one UMC, but nationwide. The value of a particular approach becomes much clearer when we pool everything that’s happening across the Netherlands. Each UMC has dozens of research groups developing models and testing interventions – we can learn a great deal from each other.”

The Role of RARE-NL

When asked what he envisions for the coming years, Lefeber says:
“I hope RARE-NL becomes the platform that provides clear, protocol-based guidance for research into therapy development for rare diseases – always with social responsibility in mind. For instance, we could formalize our knowledge about developing RNA or gene therapies into clear guidance documents. Medicijn voor de Maatschappij is already working on a pilot project to improve access to orphan drugs through the Orphan Drug Access Protocol. We also hope to bring together existing research projects and secure larger grants for key themes. Ultimately, our joint initiative aims to ensure that more effective and safe treatments become available for patients with rare diseases.”

A version of this interview was previously published on the FAST website in the lead-up to the launch of RARE-NL.