Cheaper and improved treatment may be on the way to patients with the metabolic disorders called lysosomal storage diseases. Weihua Tian, a post-doc, and Zhang Yang, an associate professor at the DNRF’s Copenhagen Center for Glycomics (CCG) at the University of Copenhagen, headed a research team from CCG, USA and the CCG spinoff company GlycoDisplay Aps. Together, the researchers have developed a sugar structure that gives hope for better and cheaper treatment of rare lysosomal diseases such as Fabry’s disease. The study was recently published in Nature Communications.
Researchers from the DNRF Center of Excellence CCG at the University of Copenhagen, in collaboration with the company GlycoDisplay – a spinoff company from CCG – and American researchers, have developed a new sugar design that may improve the treatment of the so-called lysosomal diseases, also known as genetic metabolic diseases. The newly developed sugar structure was initially targeted to one of the rare metabolic diseases called Fabry’s disease, but the researchers also intend to use it to develop improved and cheaper treatment methods for all lysosomal diseases.
“Each lysosomal disease is caused by a shortage of one particular enzyme, and now we have been able to increase the effect of one of them, namely, the one missing in Fabry’s disease. We believe the new design concept can be transferred to other enzymes in the future, in principle making it possible to treat all lysosomal diseases,” said first author of the study, post-doc Weihua Tian from the Copenhagen Center for Glycomics.
Current method is demanding and imperfect
Lysosomal diseases are characterized by a shortage of a particular lysosomal enzyme responsible for metabolizing waste products in the body. Shortage of a lysosomal enzyme causes waste products to accumulate in the organs, which may lead to organ failure.
The current treatment method for Fabry’s disease and other lysosomal diseases occurs by so-called replacement therapy, where the patient is given frequent injections of the missing enzyme. But the method is demanding and among the most expensive treatments.
The biggest problem with the current treatment method, however, is that the enzyme rarely reaches all organs, and the treatment is only partially effective. Some patients cannot be treated at all. This means, for instance, that men with Fabry’s disease often suffer from heart and kidney problems, even though they are in treatment, and thus, men suffering from Fabry’s disease have a life expectancy of less than 60 years. The newly developed sugar structure makes it easier to target the enzymes to the organs.
“The injected enzymes are decorated with sugar structures that determine how long the enzymes circulate in the body and to which organs they are targeted. Using gene editing technologies, we have been able to modify these sugar structures, prolonging the circulation time of the enzymes and ensuring better targeting to the organs that need them,” said Claus Kristensen, CEO of GlycoDisplay.