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Marjo van der Knaap: restoring the beauty of the brain

Neurology is on the brink of great changes in the development of therapeutic strategies, says Marjo van der Knaap, professor of child neurology at the VU medical centre. She works hard to develop treatments for diseases that affect the young brain.

Ever since she first encountered an MRI scanner, it was clear that Marjo van der Knaap would use it. She explains: ‘In 1985 we got the first scanner, and I immediately liked it. I am moved by the beauty of things: paintings, sculptures, and certainly the detailed images of the brain that we were able to make with magnetic resonance imaging.’ The nature of neurology also fits her personality, van der Knaap adds, ‘I like to know a lot, to analyse the problem. The localisation of neurological diseases is a puzzle I like to solve.’

Combining neuroscience with clinical practice, van der Knaap provides her little patients with cutting edge medical advice. Her specialty is a group of diseases that affect the white matter of the brain. All white matter disorders are rare, but because there are so many disorders, their total incidence is not so low, with between a hundred and two hundred new patients in the Netherlands per year. They have a profound impact on the development of the child and may culminate in death at a very young age. Van der Knaap developed an MRI pattern recognition system to distinguish different diseases of the white matter based on the MRI-scan of the brain, improving the possibilities for early diagnosis and better prognosis in living children. This also led her to the discovery of multiple new white matter diseases. Together with doctor Jan Pronk, a geneticist at the VUmc, she identified the five genes responsible for one of those conditions, called vanishing white matter disease. Over the years, she and her group have identified several additional genes for different white matter disorders.

No cure is available for this and many other neurological diseases at present. However, in the near future, treatment options will expand, says van der Knaap. ‘Neurologists are widely regarded as specialists who are very good at diagnosing diseases of the nervous system, but have little to offer in terms of treatment options. I think that paradigm is about to shift. Medical interventions for diseases like Parkinson’s are getting better, and within the next decades we will see a whole range of new treatments for neurological diseases being developed. Medicine is moving towards smarter and more effective treatments, also in my field.’

Van der Knaap is determined to investigate the possibilities of new therapies, for example stem cell therapy for vanishing white matter disease. Winning the Spinoza Award in 2008 only encouraged her to expand the scope of her research program, and the Neuroscience Campus helps her to make those promises come true. ‘Ten years ago my group joined the CNCR, which resulted in many instances of fruitful collaboration with other, more fundamental, neuroscientists. It is very rewarding to work closely together with specialists of closely related research areas. We offer them new questions and insights, and they provide us with new tools and ways to think about the brain.’

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