Development of drugs for the treatment of Huntington’s and Alzheimer's

GO-Bio 1 – Prof. Dr. Erich Wanker – Max Delbrück Center for Molecular Medicine (MDC), Berlin

Liegende Glasflasche, aus der Pillen herausfallen

Hannes Eichinger -

Recipient: Max Delbrück Center for Molecular Medicine (MDC), Berlin
Funding: GO-Bio Phase I (01.01.2007 - 30.04.2011, 3.134.946 Euro)


Proteins are the molecular workhorses of the body and are able to take on a wide variety of functions. In order to become active, however, the molecular chains must first fold into a three-dimensional form. If this highly complex process is disrupted, it can result in aggregations and abnormal deposits of protein molecules, which are a cause of a number of different disorders of the nervous system. In recent years, Erich Wanker has demonstrated that, as well as Alzheimer's, the inherited condition of Huntington’s disease also traces back to these anomalous aggregations, resulting in the malfunction of important nerve cells. This has given rise to the idea of targeting the protein folding process as part of comprehensive treatment for nervous diseases.

In previous work, funded among others by the BMBF BioFuture Competition, Wanker developed a high-throughput method that was used in the identification of around 20 potential active substances in several chemical classes. These were selected from a total of 184,000 substances. In the animal model, the identified substances effectively prevented protein misfolding in a number of different ways. Wanker is planning to use the GO-Bio funding to filter out promising candidates for application in the treatment of Huntington’s and Alzheimer's. After three years, it is intended to found the company Amylocure, which will carry out initial pharmaceutical testing on humans. This will be accompanied by the development of a diagnostic procedure for Alzheimer's.