Dutch universities and university medical centers (UMCs) hold a wealth of high-level knowledge and expertise in therapy development. At the same time, many patients are still waiting for effective treatments. What can RARE-NL do to help unlock and apply this academic expertise to bring new therapies to patients? We spoke with Prof. Dr. Teun van Gelder, Professor of Clinical Pharmacology and board member of the Dutch National Pharmaceutical Knowledge Centre (NFKC).

Van Gelder is a physician (internist-nephrologist) and clinical pharmacologist. In late 2019, he moved from Erasmus MC to Leiden University Medical Center (LUMC), where he now works on academic drug development. “I came to Leiden because they were investing in Academic Pharma – stimulating drug development within academia, either independently or in collaboration with the pharmaceutical industry. That seemed like a great challenge, and it still is,” he says. In addition to his research and teaching roles, Van Gelder continues to see patients, with outpatient clinics for kidney transplant recipients and for patients with complex pharmacological issues.

Building a Knowledge Hub

LUMC and Leiden University have jointly prioritized academic therapy development. They found they already had considerable expertise in-house, and the social relevance of this field was decisive in setting it as a strategic focus. Van Gelder: “We basically cover the full spectrum of drug development here – from molecular discovery at the Leiden Institute of Chemistry (LIC), to pharmaceutical sciences and clinical pharmacy, including our own GMP production facility, all the way to studies in healthy volunteers and patients at CHDR.”

To support these efforts, the Dutch National Pharmaceutical Knowledge Centre (NFKC) was established. In December 2020, the foundation received funding from the Ministry of Health (VWS) to develop regulatory dossiers for several different drugs. “These are diverse compounds at various stages of development,” Van Gelder explains. “By going through the full process – from preclinical and clinical research to submitting a marketing authorization application to the European Medicines Agency (EMA) – we gain valuable experience in navigating regulatory pathways.”

Examples in Practice

One compound currently being prepared for regulatory submission by the NFKC is a modified version of doxorubicin, a chemotherapy drug known for its serious side effects, particularly cardiac toxicity. Leiden-based Professor of Chemical Immunology Prof. Dr. Sjaak Neefjes has spent years developing and testing chemical variants of doxorubicin. One of them, dimethyldoxorubicin, shows a promising efficacy profile and is significantly less harmful to the heart.

Another example is a treatment for Lambert-Eaton myasthenic syndrome (LEMS), a rare neurological disorder caused by an autoimmune attack on calcium channels in the neuromuscular junction. The LUMC pharmacy has long been producing a slow-release formulation of amifampridine to reduce symptoms. “We are now seeking official registration – if only to prevent a commercial entity from taking the academic know-how, registering the product, and selling it at many times the current cost,” says Van Gelder.

A Complex Regulatory Landscape

According to Van Gelder, working through the regulatory requirements raises many difficult questions. “It’s a complex field with a steep learning curve,” he says. “Anyone who’s dealt with a major pharmaceutical company knows how many departments they have. When things get complicated, they show up with ten specialists – one for toxicology, one for preclinical studies, one for clinical trials, and so on. And if they don’t know something, they call in one of their other 50 colleagues. If we, in academia, want to do what pharma companies do – actually bring new treatments to market – we need access to that same breadth of expertise.”

Unlocking Expertise Across Institutions

To develop new treatments, you need more than scientific knowledge alone – regulatory experience, intellectual property management, and market access expertise are also essential.
Van Gelder sees a clear role for RARE-NL in connecting these areas of expertise: “I know most Dutch universities and UMCs are engaged in therapy development. The level of visibility and resources may vary, but overall, there’s a great deal of expertise spread throughout the country. The challenge is: how do we find it? By bringing people and knowledge together through RARE-NL, it becomes easier to identify the right expert for a given problem. I have high expectations – especially for all the therapies and indications that pharmaceutical companies tend to overlook because they’re not profitable or don’t fit their strategy.”

Affordable and Accessible Treatments

Pooling academic strengths can make therapy development more cost-effective, which could ultimately improve access and affordability. Van Gelder: “As academic centers, we don’t need to satisfy shareholders or generate profit. Depending on the production scale, we can manufacture treatments ourselves or partner with companies. I believe every situation requires a tailored approach – one that prioritizes patient need over profit.”

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