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ALPHA-MAN

Prof. Paul Saftig

Scientific Coordinator of ALPHA-MAN, based at Christian-Albrechts University Kiel, Germany

Research field

Lysosomal Storage Disorders

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Research Profile

Who?

"It is fascinating to drive projects from the discovery of basic cellular processes to the development of drugs for clinical use", says Paul Safig, Professor of Biochemistry at the Christian Albrechts University of Kiel. The team of Paul Saftig contributed to a better understanding of cellular compartments called ‘lysosomes’.

Why?

Alpha-mannosidosis is a rare genetic disease with severe consequences. Patients suffer from mental retardation, skeletal changes, hearing loss and recurrent infections. Many patients die during early childhood. A therapy initiated shortly after birth may dramatically improve the life expectancy and quality of life of patients.

What?

Professor Saftig explains: “Lysosomes ensure that waste is removed from our body cells. In lysosomal storage disorders (LSDs) such as alpha-mannosidosis, this process malfunctions. As a result, toxic substances accumulate in cells. The ALPHA-MAN project aims to develop an effective drug therapy for alpha-mannosidosis.

How?

“In our two previous European projects ‘EURAMAN’ and ‘HUE-MAN’, we succeeded in producing the enzyme that is lacking in alpha-mannosidosis patients. We used recombinant technology to do this. The ALPHA-MAN consortium will now perform clinical trials to test the recombinant enzyme as a therapy for alpha-mannosidosis patients."

From basic science to therapy for a lysosomal storage disorder

In the Picture
In the Picture
ALPHA-MAN project website HUE-MAN project website International Society for Mannosidosis & Related Diseases CLEAR (another European project about lysosomal storage disorders) In Focus article 'Rare disease, common struggle' PDF versionPDF version
Image credits

Background image: ALPHA-MAN

Portrait of the project coordinator: ALPHA-MAN

Timeline (in chronological order): ALPHA-MAN