Funding transnational collaborative research through joint transnational calls is one of the major objectives of E-Rare. This is the most important and effective joint activity to enhance the cooperation between European scientists working on rare diseases and thus reducing the fragmentation of research in this field. E-Rare launches calls on a yearly basis. The topic and eligibility criteria are specified every year and therefore may vary from one call to the other.

Inhibition of Rho Kinase (ROCK) with Fasudil as disease-modifying treatment for ALS

Project Coordinator

University Medicine Göttingen


Markus Weber Kantonsspital St. Gallen St. Gallen, Switzerland
William Camu Centre hospitalier universitaire Gui de Chauliac Montpellier, France

In this study, the investigators will determine whether the drug Fasudil holds promise for treating ALS. Motor neurons are the cells, which primarily degenerate in ALS and this degeneration results in progressive paralysis and finally death of patients by respiratory failure. Fasudil is a small-molecule drug that is approved and marketed in Japan for the treatment of hemorrhagic stroke.

Using an ALS mouse model scientists from independent groups have discovered that Fasudil prolongs the life span and improves motor function. Interestingly, Fasudil not only diminished the death of motor neurons, but also influenced the behaviour of surrounding non-neuronal cells and protected neurons from stress signals. Fasudil also increased the regenerative outgrowth of nerve cells. Similar findings were also reproduced in other animal models. Since Fasudil has a safe and well-described side-effects profile, the investigators now decided to move into the clinic and evaluate Fasudil in human ALS patients.

In the current study the safety of Fasudil at two different dosages will be tested and initial information on the drug’s potential efficacy will be obtained. Furthermore, biomaterials will be collected in order to enlarge the biosample collection of human ALS patients, which will be made available to other researchers. Centers in Germany, France, Switzerland, Poland, and the US will participate in this trial.

If Fasudil proves to be effective in this clinical trial, a larger international phase III trial could be envisaged, which ultimately may lead to an effective disease-modifying therapy for this fatal disease.

E-Rare 2012 - Created by Toussaint Biger