Treatment
Treatment remains largely supportive. The behavioral disturbances of MPS-III respond poorly to medication. If an early diagnosis is made, bone marrow replacement may be beneficial. Although the missing enzyme can be manufactured and given intravenously, it cannot penetrate the blood–brain barrier and therefore cannot treat the neurological manifestations of the disease.
Along with many other lysosomal storage diseases, MPS-III exists as a model of a monogenetic disease involving the central nervous system. Several promising therapies are in development. Gene therapy in particular is under Phase I/II clinical trial in France since October of 2011 under the leadership of Paris-based biotechnology company Lysogene . Other potential therapies include chemical modification of deficient enzymes to allow them to penetrate the blood–brain barrier, stabilisation of abnormal but active enzyme to prevent its degradation, and implantation of stem cells strongly expressing the missing enzyme. For any future treatment to be successful, it must be administered as early as possible. Currently MPS-III is mainly diagnosed clinically, by which stage it is probably too late for any treatment to be very effective. Neonatal screening programs would provide the earliest possible diagnosis.
The group of Grzegorz Węgrzyn, from the Department of Molecular Biology at the University of Gdansk, Poland, has found that the flavonoid genistein decreases the pathological accumulation of glycosaminoglycans in Sanfilippo syndrome. In vitro, animal studies and clinical experiments suggest that the symptoms of the disease may be alleviated by an adequate dose of genistein. Despite its reported beneficial properties, Genistein also has toxic side effects.
A couple of US-based groups have recently been organized to speed the development of new treatments for Sanfilippo syndrome.
Read more about this topic: Sanfilippo Syndrome
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