RARE-NL: LUMC joins consortium to find treatments for rare diseases
RARE-NL, a new collaboration between university hospitals, hopes to find treatments for rare diseases. Professor Teun van Gelder is representing the LUMC in the initiative.
Around 36 million people in the EU suffer from rare diseases for which no treatment is available. This is mainly because of the high cost of drug development or conflicting interests among businesses, academic institutions and government.
The RARE-NL consortium will try to develop therapies or drugs for these diseases nonetheless. They hope to do so by creating public-private partnerships that will either look at whether existing drugs would prove effective for particular rare diseases (drug repurposing) or develop new treatments for such diseases.
RARE-NL is a hub within Future Affordable Sustainable Therapy Development, (FAST), the national expertise centre focusing on innovation in drug development. Teun van Gelder, Professor of Clinical Pharmacology and lead of the Leiden interdisciplinary research programme Leiden interdisciplinary research programme Translational Drug Discovery and Development, is representing the LUMC in the project.
RARE-NL is a national initiative launched by the LUMC (NFKC), Radboudumc (Therapy accelerator), Amsterdam UMC (Medicijn voor de Maatschappij) and FAST. The consortium is inviting all UMCs to join.
Katwijk Disease
The consortium members still need to decide which specific diseases RARE-NL will focus on. ‘These could include the rare, genetic brain disorder HCHWA-D, also known as Katwijk Disease’, says Van Gelder.
We know where in the DNA the defect can be found that affects the cerebral blood vessels in this genetic disease, and that proteins accumulate in the blood vessel walls.
An effective drug that addresses the cause of the problem is sorely needed. The pharmaceutical industry is not particularly interested because the condition is rare, but Leiden researchers (Expertise Centre for Hereditary Cerebrovascular Diseases) are now taking the lead.