Drugs Screening & Therapy Design
Program Committee
Peter Heutink (program leader)
Guus Smit (program leader)
Marjo van der Knaap
Adriaan Lammertsma
Huibert Mansvelder
Matthijs Verhage
Drugs Screening and Therapy Design
The aims of this advanced technology program are two fold: The first aim is to make optimal use of our growing potential in cellomics facilities, which currently include cell culture robots and automated fluorescence time lapse microscopy tools that allow for medium throughput screening of genetically modified cell-lines. The unique asset of this technology is that the Neuroscience Campus can offer new bioassays by taking gene polymorphisms or haplotypes selected in for instance Genome Wide Analyses (GWA) and model these directly at the cellular level to test the effect of genetic variation or mutations on for instance neuroregeneration. Since this technology is automated and executed in mutliwell plates, it allows for the focused testing of (small molecule) compound libraries (in collaboration with for instance partners from TiPharma. The second aim is to set up a so-called stem-cell facility, and to implement and further investigate to potential of stem-cell therapy (first in mutant mouse models of Childhood White Matter Disorders and in the long run in human patients).
The program’s collaborative research projects concern screening of potential therapeutic compounds in view of neurodegenerative disease (grey matter) as well as while matter disorders. In particular through bundling of the newest insights into the genetic vulnerability toward grey or white matter disorders in cellular assays and combining these with drug (design) screening, will help us to achieve a number of truly translated objectives. Second, in particular for the devastating (and Mendelian genetic) child neurology disorders (but potentially in the long run also for more common diseases including MS) stem-cell therapy may be a serious option when it comes to curing the diseases. The Spinoza price to Marjo van der Knaap (2008) will partly be used for this purpose.
Finally, most recently there was the Prinses Beatrix price for Peter Heutink. The group of Heutink supported by this price in coming year will identify new genetic risk-factors for both Mendelian and multifactorial forms of PD and use these, and known risk factors, for the development of new therapeutic approaches. Heutink will use a multidisciplinary approach to confirm and characterize the biological relevance of risk factors and to identify all functionally interacting genes in the affected pathway by using a high throughput cell biology (cellomics) approach. The characterization of gene networks instead of single genes will help to identify those genes in the affected gene network that can best serve as lead targets for development of new therapeutic approaches. As Heutink claimed at this time of the award: "This price is a fantastic boost for the Drugs Screening and Therapy research program of the Neuroscience Campus Amsterdam. I am very happy with this award and with the funding for our cellomics research activities".
