February 24, 2014
Neurodegenerative diseases are often a result of protein deposits, which can damage nerve cells. In the case of Parkinson’s, deposits of aggregated synuclein proteins become visible in the brain when viewed under the microscope. Precursors of these deposits, the so-called oligomers, are highly neurotoxic. For humans, this can result in muscle tremors, movement disorders and muscular rigidity. These disease-causing protein deposits, which are caused by so called prion proteins, are also found with the Creutzfeld-Jakob disease. Working in teams, both Armin Giese, research group leader at the Center for Neuropathology and Prion Research at the Ludwig-Maximilians-Universität and Christian Griesinger, Director at the Max Planck Institute for Biophysical Chemistry, Department of NMR-based Structural Biology have developed a drug candidate that has prevented the formation of the toxic oligomeric forms of protein, thereby delaying the progression of neuronal damage to an extent previously unattained and thus prolonging the disease-free phase.
A new feature is that the active substance called anle138b targets directly and specifically oligomer forms of protein. Thus, anle138b prevents the aggregation and formation of new, disease-relevant oligomer protein forms. The synthesized drug, which was administered with food to the test mice, is tolerated very well in therapeutic doses and effectively penetrates the blood-brain barrier, thereby reaching high levels in the brain with a small dosage. The pre-clinical study found that mice exposed to anle138b had significantly longer survival times and were able to display better motor coordination than their untreated siblings.
In the future, diseases such as Parkinson’s could possibly be slowed down or even stopped with anle138b as disease-related processes are directly inhibited. However, the new substance is not only effective for diseases such as Parkinson’s. Positive research results show that anle138b is effective with Creutzfeld-Jakob by effectively inhibiting the aggregation of pathogenic protein deposits, and treated mice survive significantly longer. Likewise, the results in mouse models for Alzheimer’s are encouraging. Managing Director Torsten Matthias says that “due to the study results so far, the MODAG GmbH seriously gives me hope that with this new substance we will be able to offer much-needed help for Parkinson’s and Alzheimer’s to patients with an early stage and safe therapy.”
The underlying basic technology was patented as a joint invention of the Ludwig-Maximilians-Universität and the MPI for Biophysical Chemistry, and was exclusively licensed by MODAG GmbH. “We are pleased that we could help to bundle the research competencies of the Ludwig-Maximilians-Universität München and the Max Planck Institute for Biophysical Chemistry in the field of drug research in a targeted way into this promising spin-off company,” says Peer Biskup, Managing Director of the Bayerische Patentallianz, the patent marketing agency of the Bavarian universities and universities of applied sciences. “In addition to outstanding research skills, MODAG also has the necessary development skills and extensive business know-how,” adds Astrid Giegold, Start-up & Portfolio Manager at Max Planck Innovation, the technology transfer organization of the Max Planck Society.