How brain disorder models are like the Night Watch
Professor of Human Genetics Willeke van Roon will give her inaugural lecture on Monday 28 March entitled: ‘Translational research, where small parts make the bigger picture.’ She will emphasise how university medical centres should take responsibility for finding treatments for very rare diseases.
Willeke van Roon will start her inaugural lecture with an unusual reference: to the Night Watch by Rembrandt. She believes this painting has many similarities with her work. ‘The longer you look at this big painting, the more details you see. Like the girl with the chicken hanging from her belt. Its claws refer to the Kloveniers, the name of the civic guard in the painting. Together, all these small parts make up the bigger picture.’
Van Roon compares the Night Watch to the brain disorder models she is working on. She focuses on disorders that are characterised by proteins that clump together in cells. ‘In the simple models, all these disorders look similar. But if you look longer and at more complex models, you start to see many more differences and the diseases turn out to be much more complicated.’
Sketch beneath the paint
Protein accumulation was first thought to be the cause of Huntington’s disease, for example. But revisiting this model with the latest techniques showed that it is the phase before protein accumulation that is most harmful. ‘Again, I see a parallel here with the Night Watch. They’ve now used the latest technology to reveal the first sketch visible. That makes you look differently at the existing painting, or in this case disease model.’
Van Roon will then take her audience from brain cells to the families of people with hereditary brain disorders. ‘Doctors often send the patient home because they can’t do anything else for them, but the patient’s family isn’t about to accept that.’ These patients with no hope of treatment inspired Van Roon and her fellow professor Annemiek Aartsma-Rus to set up the Dutch Center for RNA Therapeutics.
The centre focuses on the development of RNA therapies that make the proteins less harmful or restore the missing protein. This will not cure the disease, but it will slow it down. ‘At the centre, we want to develop tailor-made RNA therapies for patients with a genetic disorder. As sometimes only one patient is known to have a particular disease, companies aren’t interested in developing a therapy.’
Leaving the lab
Van Roon therefore contends that university medical centres should take responsibility for finding treatments for these very rare diseases. ‘Traditional drug development doesn’t suit this group of disorders,’ says Van Roon. She is working on several RNA therapies for rare diseases in her laboratory. ‘We still have plenty of research to do in the lab. But in the next few years, I also hope to leave the lab with a number of therapies and actually treat patients.’
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