Research, projects

MISSION OF THE FIGHT

Currently available drugs and therapies are not completely effective because they can not overcome the blood-brain barrier (BBB) that surrounding and protecting the brain prevents their absorption and pharmacological action in the brain. Therefore, the main goal of the specialized research centres taking part to this research program is to develop innovative therapeutic strategies to overcome the blood-brain barrier and permit drug delivery directly to the brain.

In particular, individual sub-projects have the following objectives:

Project 1  Ingeborg Krägeloh-Mann Tübingen Germany

To make an epidemiological survey and description of the natural course of metachromatic leukodystrophy manifesting during childhood and adolescence.

Project 2  Hans Joachim Galla / Münster / Germany

To set up in vitro experiments in order to characterize the receptors and the mechanism responsible for endothelial cells absorption and effective transfer to the brain of ASA enzyme.

Project 3  David Begley / Londra / UK

To use mice animal models for testing the effectiveness of the recombinant human enzyme ASA (rhASA) combined with nano particles vector and verifying its ability to cross the BBB and successfully reach the brain.

Project 4  Ingolf Blasig / Berlino / Germany

To set up procedures for in vitro preparation of ASA enzyme and test its effectiveness in combination with fusion peptides that should facilitate its passage trough the BBB.

Project 5  Jörg Kreuter  Francoforte / Germany
                  Cooperation Partner Svetlana Gelperina, Nanosystem LTD, Mosca Russia

To develop and employ nanoparticles allowing ASA enzyme to bypass the BBB. To test diverse nano particles made of different polymeric materials and verify the efficiency and biocompatibility.

     Project 6   Volkmar Gieselmann / Bonn/ Germany

To research and develop, by mean of transgenic mice, tools that allow the inactivation of the cerebroside transferase enzyme (CST) useful for the substrate reduction therapy.

     Project 7   Maurizio Scarpa / Padova e Angelo Luigi Vescovi /    Milano  Italy

To use neural stem cell as treatment for MLD.

     Project 8   Alessandra Biffi / Milano / Italy

To use haematopoietic stem cell gene therapy for metachromatic leukodystrophy.