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LEIBNIZ-DRUG OF THE YEAR: HOPE FOR PATIENTS HAVING A SKIN DISEASE

2015-04-22 / Award for a novel therapeutic approach for ichthyosis, a rare skin disease for which no therapy is available so far. The Leibniz Research Alliance "Bioactive Compounds and Biotechnology" recognizes the achievements of Margitta Dathe’s group at the Leibniz-Institut für Molekulare Pharmakologie (FMP) for the development of a drug application form that has received an orphan drug status from the European Commission in 2013.

In humans being afflicted with a congenital ichthyosis the natural balance between cell growth and cornification is disturbed. Patients suffer from an extreme increase of the upper most skin layer, which becomes visible as dark-brown lamellar scaling. Affected persons spend 1 to 2 hours each day just for their skin care, moreover, they suffer from inflammation and are prone to infections, can hardly perspire and often are afflicted with bullying and stigmatization, from childhood onwards. Ichthyosis is a general umbrella term since the disease is caused by several genetic defects most of them being very rare. Some patients lack the enzyme transglutaminase 1 (TG1). This enzyme acts as a natural glue and enables proteins to crosslink within the upper skin layers thus preventing water loss and dehydration. The now awarded drug application form restores precisely this function.

Several years ago, Heiko Traupe and Karin Aufenvenne, ichthyosis experts at the department of dermatology of the university hospital Münster, were looking for a way to replace the lacking enzyme in patients. They contacted Margitta Dathe who is heading the “peptide-lipid interaction group” at the Leibniz-Institut für Molekulare Pharmakologie (FMP). Together with her group she developed liposomal carriers for recombinant human TG1. The challenge was to achieve an uptake of the enzyme to its site of action within certain skin cells, namely keratinocytes. Equipped with a special peptide the liposomal carriers were able to deliver TG1 into these cells.

The overall efficacy of the enzyme replacement therapy has been demonstrated in the meantime in cooperation with Fernando Larcher’ group at the Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas in Madrid and Ingrid Hausser-Siller at the department of pathology of the University Hospital Heidelberg. In 2013 the therapy has received an orphan-drug status from the European Commission, which guaranties a simplified and cost-reduced procedure for drug approval as well as exclusivity for ten years after market authorization in the member states of the European Union.

Currently, discussions are ongoing with a company of the Pierre Fabre Laboratory (Pierre Fabre Orphan Dermatology) in order to initiate further pre-clinical and early clinical studies.
The liposomal based drug for enzyme replacement therapy of ichthyosis patients has been awarded “Drug of the Year 2015” by the Leibniz Research Alliance "Bioactive Compounds and Biotechnology". Involving 17 institutions, this alliance bundles the Leibniz Association's broadly-based research on molecules with biological effects. Margitta Dathe and her colleagues Heiko Nikolenko and Katrin Jordan received the award. The award includes a prize worth EUR 2,000 and will be handed over on April 27 on the occasion of the LEIBNIZ MEETING ON BIOACTIVE COMPOUNDS in Hamburg.

Original publications:
Apolipoprotein E peptide-modified colloidal carriers: the design determines the mechanism of uptake in vascular endothelial cells.
Leupold E, Nikolenko H, Dathe M.
Biochim Biophys Acta. 2009 Feb;1788(2):442-9.
Topical enzyme-replacement therapy restores transglutaminase 1 activity and corrects architecture of transglutaminase-1-deficient skin grafts.
Aufenvenne K, Larcher F, Hausser I, Duarte B, Oji V, Nikolenko H, Del Rio M, Dathe M, Traupe H.
Am J Hum Genet. 2013 Oct 3;93(4):620-30.

About the Leibniz-Institut für Molekulare Pharmakologie (FMP)
The Leibniz-Institut für Molekulare Pharmakologie (FMP) is part of the Forschungsverbund Berlin e.V. (FVB), who legally represents eight non-university research institutes - members of the Leibniz Association - in Berlin. The institutions pursue common interests within the framework of a single legal entity while maintaining their scientific autonomy. More than 1,500 employees work within the research association. The eight institutes were founded in 1992 and emerged from former institutes of the GDR Academy of Sciences.

About Pierre Fabre
Pierre Fabre is the 3rd largest French pharmaceutical group and the 2nd largest dermo-cosmetics laboratory in the world. In 2014, its sales reached €2.1 Billion, with Dermo-Cosmetics revenues accounting for 55% and international sales for 55%. Founded and its headquarters still based in the South-west of France, Pierre Fabre currently has branches in 44 countries and distribution agreements in over 130 countries. Covering a continuum of healthcare products, from prescription drugs and consumer health care products (OTC, oral care, natural health : Elgydium, Eludril, Drill, Naturactive) to dermo-cosmetics (Avène, Klorane, Ducray, René Furterer, A-Derma, Galénic), Pierre Fabre Laboratories employ over 10,000 people worldwide.  A unique example in the industrial universe in France, the Pierre Fabre Laboratories belong to a government recognized public-interest foundation. To find out more, please visit www.pierre-fabre.com.

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