© 2018 by UFFI United for Fighting Ichthyosis - Amici per la Pelle ONLUS. 

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The Enzyme Replacement Therapy

A breakthrough was achieved that can change lives, not just for better, but that actually cures the illness!

 

A specialists team led by Professor Heiko Traupe, brought together the University of Munster and the Leibniz Institute for Molecular Pharmacology developing and lab testing what is called the enzyme replacement theraphy to treat TGM1 deficiency Lamellar Ichthyosis.

 

​WHAT IS THIS ENZYME REPLACEMENT THERAPY AND WHAT DOES IT DO?

The latest nano-technology is developed and synthesized in a cream, a molecular vehicle capable of placing the enzyme (TGM1) in the exact spot where it’s missing thus reinstating all skin malfunction: basically curing the desease.  The treatment would mainly consist of a lifelong yet periodic application of the sophisticated cream, (perhaps once or twice a week), which could replace the missing enzyme thus resulting in the normalization of the skin! 

WHAT HAS OUR MEDICAL TEAM ACHIEVED SO FAR?

  • They have developed the experimental basis for an enzyme cream replacing the missing enzyme;

  • This has already resulted in the healing of massive scaling in a mouse model where transplants of human ichthyosis skin were treated therefore, at the moment, we have a  liposomal formulation that encapsulates the enzyme in an aqueous solution. At the moment this cannot yet be tested directly on humans which is why we need urgent donations to continue this development until human testing is possible;

  • Awarded Drug of the year 2015 by Leibniz Research Alliance;

  • Received official recognition by an orphan drug designation of the EU in 2013.

READ THE FULL STUDY

WHAT NEEDS TO BE DONE

Immediate Action: 

 

1. Development of cost-efficient production system for the enzyme;

2. Further improvement of liposomal formulation;

3. Development of a definite hydrogel as a prototype;

Estimated time for steps 1, 2 and 3: 12 to 15 months

Estimated costs: 2 million euros.

 

4. Afterwards a devoted biotec company or a pharmaceutical company should hopefully continue the development;