I study a rare disease called Pantothenate-Kinase-Associated-Neurodegeneration, also known by the abbreviation PKAN. The disease is caused by a defect in the so-called pantothenate kinase 2 enzyme. This enzyme is needed to make coenzyme A from vitamin B5. Coenzyme A is important for many metabolic processes in our body. A malfunction of the pantothenate kinase 2 enzyme could lead to problems in the brain. There is no proper treatment yet against PKAN and patients often die at a young age.
During the research (Coenzyme A metabolism in health and disease - Sibon Group), we found that fruit flies with PKAN would become less ill and live longer if they were provided a substance derived from vitamin B via their food. To explore this further, we had to make this substance suitable for testing in a clinical research with PKAN-patients. This sounds easier than it is: the execution takes a long time and is very complex.
As a fundamental researcher, I work in a laboratory: I do not have the knowledge or experience to develop a product suitable for critically ill patients. Moreover, we have the ambitious goal to make our product available for all PKAN-patients worldwide. It was, however, quite a challenge to finance this project. Any ‘standard investors’ were out of the question as our project is not patentable.
The Innovation Center did not just help, but even ensured that every step outside of the laboratory became possible. The center was crucial in finding the right companies and facilities to help us create the vitamin B5-derived substance that met all requirements (of purity, reproducibility, safety). Thanks to their help, the product was eventually approved by the medical ethical board so we could use it for clinical research with PKAN patients.
The Innovation Center also helped with identifying financing methods, explaining regulations and much more. Thanks to our collaboration with them, we were able to succeed in starting up a clinical research with the product we invented!
The Innovation Center did not just help, but even ensured that every step outside the laboratory became possible. Thanks to our collaboration with them, we were able to succeed in starting up a clinical research with the product we invented.
